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Clinical Trials


Study Title:

DUAL-1: Digital Ulcers with macitentan in systemic sclerosis. Prospective, randomized, placebo-controlled, double-blind, multicenter, parallel group study to assess the efficacy, safety and tolerability of macitentan in patients with ischemic digital ulcers associated with systemic sclerosis

IRB Number: HUM00057376  

Study Team:

Elena Schiopu, MD, Rheumatology, Internal Medicine, University of Michigan Scleroderma Program

Dinesh Khanna, MD, MS, Rheumatology, Internal Medicine, University of Michigan Scleroderma Program

Study Purpose:

The purpose of this clinical research study is to evaluate the effectiveness and safety of macitentan to reduce the number of new digital ulcers associated with scleroderma. This study will evaluate the effect of macitentan therapy on the development of new digital ulcers and improvement on hand function.

Who can take part in this study?

Males and females greater than 18 years of age who have been diagnosed with systemic sclerosis (SSc) who currently have a finger ulcer. You should not or may not be eligible to participate in the study if you have other medical conditions such as pulmonary arterial hypertension (a type of high blood pressure in the lungs), be pregnant or fail to practice acceptable birth control, have severe diarrhea, anemia (low levels of iron in your blood), liver or kidney disease, arthritis, any form of tobacco or nicotine use in the last 6 months, or have low blood pressure.  You may not be eligible to participate if you are on or have received certain medications, or have received certain treatments for your finger ulcers.  Your doctor will review this with you.

Study Procedures

Visit 1 (Screening): Pre-Study Evaluation 

To participate in the study there are certain requirements that must be met. The information from the tests, procedures, history taking and physical examinations that take place during a screening visit will determine if you qualify for the study. The study will be fully explained to you and you will be asked to sign the informed consent prior to any study procedure being performed. 

The procedures to be performed during this initial visit include, but are not limited to, a physical examination, including blood pressure, heart rate, height and body weight, a complete medical history and a blood sample (approximately 5 teaspoons) to be taken for laboratory evaluations.  You will be asked to fill out a questionnaire to assess your level of pain associated with all your digital ulcers.

Your doctor will ask you to perform a test that checks the function of your heart (electrocardiogram).

If you are a woman who is able to have children, a blood pregnancy test will be done at this visit, as you are not eligible to participate if you are pregnant.

If you do not have digital ulcers at this visit that could qualify for study participation, your doctor may reconsider later your possible participation to the study if you develop new digital ulcers that could qualify for study participation.

If you meet the study requirements, and if you decide to participate in the study, your doctor will schedule the next visit.

Visit 2 (Randomization)

If you are eligible to participate you will come back within 2 weeks of Visit 1 (screening). The exams at this visit include assessment of your digital ulcers, any complications from your digital ulcers, blood pressure, heart rate, body weight and any changes in your medication. A blood sample (not exceeding 2 teaspoons) will be taken for laboratory evaluations (specifically to measure antibodies or proteins in your blood, produced by your body’s immune system).  You will be asked to fill out questionnaires to assess your health, activities of daily living and work productivity, level of pain associated with all your digital ulcers, and your overall assessment of your digital ulcers. In addition, if you are a woman who is able to have children, a urine pregnancy test will be done if this visit is conducted more than 5 days after Visit 1, to ensure that you are not pregnant prior to receiving study drug.

After these procedures and assessments are completed, and if you meet the final criteria for inclusion into the study, you will be given the study medication, which can be either macitentan (3 or 10 mg) or placebo. You will receive a bottle of medication containing 36 tablets. You must take 1 tablet every morning. It is important that you take your study medication as instructed and, during your next visit, return your study drug bottle (even if it is empty) with all unused study drug tablets.

You will also be provided with a diary, in order to record all the pain medications you may be taking including all local pain medications you may be applying on the digital ulcers in between office visits, as well as changes in these medications during this trial. You will be requested to bring this diary with you at all visits to discuss with your doctor.

Four weeks after your Visit 3 you will return to the doctor's office. If the results from the screening and randomization assessments show that you do not meet all the study requirements, it may, depending on the reason, be possible to repeat the screening and randomization visit (re-screening). Re-screening is allowed only once.

Visits 3, 4 and 5 (Weeks 4, 8 and 12)<

At these visits your doctor will evaluate your general health (blood pressure, pulse rate and weight) and concomitant medications. Your digital ulcers will be examined and documented, as well as any complications from your digital ulcers. You will be asked to fill out a questionnaire to assess your level of pain associated with all your digital ulcers and your overall assessment of your digital ulcers. You will also be asked to complete a questionnaire on activities of daily living. In addition to these assessments, at Week 8 (Visit 4), you will be asked to complete one health assessment questionnaire and another questionnaire on work productivity.

Blood (up to 5 teaspoons depending on the visit) will be drawn for laboratory tests (hemoglobin and liver tests), and for women of childbearing potential only, a pregnancy test to verify the absence of a pregnancy. Your doctor will also ask you questions about how you feel and have been feeling in order to investigate any arising side effects and to evaluate your response to treatment.

At each of these visits, you will need to return your study drug bottle (even if it is empty) with all unused study drug tablets. The doctor or nurse will give you a new bottle of medication containing 36 tablets.

Visit 6 (Week 16)

Four weeks after your Visit 5 you will return to the doctor's office. At this visit, you will need to return your study drug bottle (even if it is empty) with all unused study drug tablets. The doctor or nurse will give you three new bottles of medication containing 36 tablets each. Your doctor will perform a physical examination and evaluate your general health (blood pressure, pulse rate and weight) and concomitant medications. Your doctor will also perform a test that checks the function of your heart (electrocardiogram).

Your ulcers will be examined and documented, as well as any complications from your digital ulcers. You will be asked to fill out questionnaires to assess your health, activities of daily living and work productivity, level of pain associated with all your digital ulcers, and your overall assessment of your digital ulcers. 

Blood (up to 5 teaspoons) will be drawn for laboratory tests and, for women of childbearing potential only, a pregnancy test to verify the absence of a pregnancy.  Your doctor will also ask you questions about how you feel and have been feeling in order to investigate any arising side effects and to evaluate your response to treatment.

If your doctor or yourself decides on withdrawal from the study prior to Week 16 (for whatever reason), you will be asked to perform the assessments planned for Visit 6 at the time you withdraw from the study. Your doctor will also follow you 30 days after you stopped the study medication to perform an overall assessment of your digital ulcers and any associated complications, to collect information on any illness (including side effect) you may experience, as well as medications you are taking for your digital ulcers and systemic sclerosis, to perform additional blood testing and, for women who are able to have children,  verify the absence of pregnancy. Additionally, you will continue to be followed every 3 months thereafter until end-of-study through Visits a, b, c…as described in the relevant section.

Visits 7, 8, 9… (visits every month for laboratory tests and visits every 3 months for other evaluations)

There are two types of recurring visits during this period:

Monthly Visits:

These visits will occur every month, in order to perform laboratory tests (hemoglobin and liver tests) and, for women of childbearing potential only, a pregnancy test to verify the absence of a pregnancy.

Quarterly Visits:

These visits will occur every three months. At these visits your doctor will evaluate your general health (blood pressure, pulse rate and weight) and concomitant medications. Your ulcers will be examined and documented, as well as any complications from your digital ulcers.  

You will be asked to fill out questionnaires to assess your activities of daily living and work productivity during your first 2 quarterly visits. Blood (up to 5 teaspoons depending on the visit) will be drawn for laboratory tests: hemoglobin and liver tests and, for women of childbearing potential only, a pregnancy test to verify the absence of a pregnancy.  Your doctor will also ask you questions about how you feel and have been feeling in order to investigate any arising side effects and to evaluate your response to treatment.

At each of these visits, you will need to return your study drug bottle (even if it is empty) with all unused study drug tablets. The doctor or nurse will give you three new bottles of medication containing 36 tablets each.

End-of-Treatment (EOT) Visit

EOT will occur when the last patient enrolled in this study, has completed the second (16-week) interval as previously described.

At this visit, you will need to return your study drug bottle (even if it is empty) with all unused study drug tablets. Your doctor will perform a physical examination and evaluate your general health (blood pressure, pulse rate and weight) and concomitant medications. Your doctor will also perform a test that checks the function of your heart (electrocardiogram).

Your ulcers will be examined and documented, as well as any complications from your digital ulcers. You will be asked to fill out questionnaires to assess your health, activities of daily living and work productivity. 

Blood (up to 5 teaspoons) will be drawn for laboratory tests (blood chemistry and hematology) and, for women of childbearing potential only, a pregnancy test to verify the absence of a pregnancy. Your doctor will also ask you questions about how you feel and have been feeling in order to investigate any arising side effects and to evaluate your response to treatment.

During this visit, all study medication and bottle(s) will be returned and there will be no further medication dispensed.

If your doctor or yourself decides on withdrawal from the study after Week 16 (and completion of Visit 6), but prior to EOT (for whatever reason), you will be asked to perform the assessments planned for the EOT visit at the time you withdraw from the study. Your doctor will also schedule you 30 days after you stopped the study medication to perform an overall assessment of your digital ulcers and any associated complications, collect information on any illness (including side effect) you may experience, as well as medications you are taking for your digital ulcers and systemic sclerosis. He or she will also perform additional blood testing (liver tests) and, for women who are able to have children, verify the absence of pregnancy. Thereafter, you will continue to be followed every 3 months until end-of-study through Visits a, b, c…as described in the relevant section.

Study Coordinator:
Linda Briggs, PhD, MPT

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Drive
PO Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone:
(866) 628-9200

Study Status:
Currently Enrolling Patients
Informed consent required


Study Title:

Patient Reported Outcomes Measurement Information System (PROMIS) Network Study: Development and Initial Validation of PROMIS GI Symptoms Scale

IRB Number: HUM00052942

Study Team:

Dinesh Khanna, MD, MS, Rheumatology, Internal Medicine, University of Michigan.

Kristine Phillips, MD, PhD, Rheumatology, Internal Medicine, University of Michigan

Elena Schiopu, MD, Rheumatology, Internal Medicine, University of Michigan

Mona Trivedi, MD, Rheumatology, Internal Medicine, University of Michigan

Study Purpose:

Gastrointestinal tract (GIT) disorders cause a number of disruptive symptoms in millions of individuals on a daily basis. In 2004 alone, there were more than 72 million medical office visits for some type of GI disorder. Organs of the gastrointestinal tract include the mouth, throat, stomach, intestines, and anus. GIT disorders can be severely debilitating and even life threatening. Some problems associated with GIT may include heartburn, loss of voice or hoarseness, ulcers (open sores), difficulty swallowing, constipation, diarrhea, malabsorption (impaired absorption of nutrients from the GI tract), diminished peristalsis (decreased in the wavelike motion in the muscles of the intestines), and the inability to control your bowel movements.

Our group is participating in the development of a new tool to help us assess the severity of your gastro-intestinal symptoms and the effect on your physical and mental well-being.

The purpose of the study is to validate this instrument for gastro-intestinal care and future clinical trials focusing on improving associated symptoms.

Who can take part in this study?

If you meet the following criteria you may be able to take place in the study:
  1. If you have been diagnosed by a physician with a GI illness, or you have volunteered to be a control
  2. If you are 18 years or older
  3. If you are willing and able to give informed consent
  4. If you can speak, read, and understand English

Study Procedures

In this study you will be asked to fill out a series of questionnaires.  These questionnaires will ask you questions about symptoms of GI illnesses like bloating, nausea, vomiting, constipation and diarrhea.  There will also be general questions about pain levels and emotions.  All participants will be asked to fill out the first set of questionnaires.  Of those, a smaller group will be asked to complete a second set of questionnaires 2-6 months later.

Study Coordinator:
Julie Konkle, BSN, RN, CCRP

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Drive
PO Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone:
(866) 628-9200

Study Status:
Currently Enrolling Patients
Informed consent required


Study Title:

A clinical trial of N-Acetyl Cysteine (NAC) for Connective Tissue Disease-associated with Interstitial Lung Disease (CTD-ILD)

IRB Number: HUM00049434

Study Team:

Principal Investigator:
Trivedi, Mona, MD, Department of Internal Medicine, Division of Rheumatology, Scleroderma Program, University of Michigan

Coordinating Investigators:
Phillips, Kristine, MD, PhD,  Department of Internal Medicine, Division of Rheumatology, University of Michigan, Scleroderma Program

Khanna, Dinesh, MD, MS, Department of Internal Medicine, Division of Rheumatology, University of Michigan, Scleroderma Program

Study Purpose:

The primary purpose of this study is to evaluate the safety and effectiveness of N-acetyl cysteine (NAC), an antioxidant, for connective tissue disease associated with interstitial lung disease (CTD-ILD).  It is currently used for rescue treatment in acetaminophen overdose and prevention of kidney damage caused by contrast material used in radiology imaging studies.  This study is an off-label use of NAC.  NAC is considered a nutritional supplement, so it is not FDA-regulated or approved as a drug; as such, the efficacy and safety of the product are unknown.  This means the drug is not approved for use with patients with your disease and is considered experimental for your disease condition. 

Who can take part in this study?

Males and females ages 18 and above, who are English speaking and diagnosed with CTD-ILD.  Connective tissue diseases may include rheumatoid arthritis, myositis, scleroderma, or Sjogren’s syndrome.  Participants should be able to return to clinic for follow up every three months.  Women of childbearing potential must be willing to use a reliable form of medically acceptable contraception and have a negative pregnancy test confirmed at baseline before starting NAC as per standard of care. Women who are surgically sterile or have been post-menopausal for at least 2 years are not considered to be of child-bearing potential.  You will not be allowed to smoke tobacco or take nitroglycerin while you are participating in this study.

Study Procedures

After you sign this informed consent, if you meet all the study entry criteria you will return to the study center for five more visits.

At your baseline and follow up visits you will have a physical exam by a member of the study team, answer some questions about your health and medical history, have blood drawn for your baseline blood count, liver, and kidney function, and have a pregnancy test, if applicable.  You will be asked to complete six questionnaires which assess your disease symptoms and overall health status.  You will also have blood drawn at these visits for analyzing specific proteins that may be affected by NAC.  No genetic testing will be done on your blood samples.  These blood samples drawn at your study visit will be stored with your medical registration number.  You will also have pulmonary function testing done at each visit.   

You will receive NAC for the entire duration of the study.  The medication will be given to you at your baseline visit and again at each follow up visit.  The medication is in the form of a pill and will be taken three times a day every day.

You will need to bring your study drug and containers to each visit so the study team can check your supply.

Study Coordinator:
Julie Konkle, BSN, RN, CCRP

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Drive
PO Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone:
(866) 628-9200

Study Status:
Currently Enrolling Patients
Informed consent required


Study Title:

Scleroderma Registry

IRB Number: HUM 00049896

Study Team:

Principal Investigator:
Khanna, Dinesh; Department of Internal Medicine, Division of Rheumatology, Scleroderma Program, University of Michigan

Co-Investigators:
Schiopu, Elena, MD; Department of Internal Medicine, Division of Rheumatology, Scleroderma Program, University of Michigan

Phillips, Kristine, MD, PhD; Department of Internal Medicine, Division of Rheumatology, Scleroderma Program, University of Michigan

Trivedi, Mona, MD; Department of Internal Medicine, Division of Rheumatology, Scleroderma Program, University of Michigan

Study Purpose:

Scleroderma can affect patients in several ways which include activities of daily living, quality of life, and increased healthcare costs. The purpose of this project is to take a closer look at scleroderma patients’ clinical, radiological, and laboratory information associated with routine medical care, and to obtain biological specimens for testing. With your permission, we will also be storing some of these specimens so that future testing may be done.  In addition, we hope to explore the impact of scleroderma on patients’ daily lives and medical care through various questionnaires to be filled out by study participants.

By looking at all of the areas mentioned in the previous paragraph, we hope to find out information about scleroderma that will help us to treat  future patients, develop new treatments and work toward a cure.

Who can take part in this study?

Patients with a diagnosis of scleroderma who are over 18 years old.

Study Procedures:

If you agree to participate, you will be asked to do the following once a year:

1) Allow us to record data related to your condition from your medical records

If you have scleroderma, we will record information about your disease that has been previously observed or tested by your doctor.  For example, if you have had a pulmonary function test we may record the results of that test in our study records so that we are able to use that information to figure out more about your disease and how it changes over time. 

2) Fill out questionnaires relating to your mental and physical health.

We will have you fill out questionnaires at each visit during the study.  If there isn’t time for you to fill them out during your visit, we will give you a stamped addressed envelope and just ask you to mail them back to us.  

3) Skin Biopsies

If you have scleroderma and agree to having optional skin biopsies, we will take up to four small (4mm) punch biopsies (4mm is about the size of the eraser of a pencil). Up to two of these biopsies may be from skin on your arm that is affected by SSc (usually the forearm) and up to two may be taken from an unaffected area of your skin (usually the upper arm).
The procedure for a punch biopsy is relatively simple and should not take very long. You will first receive a shot of numbing medicine.  Next, a small, sharp tool that looks like a tiny cookie cutter (punch) will be placed over the area of skin to be removed.  The tool will be pushed down, and then slowly rotated to remove a circular piece of skin. The skin sample will be lifted up with a tool called a forceps or a needle and then will be cut from the tissue below with sterile scissors. This is a small tissue sample and stitches are rarely needed. Pressure will then be applied to the site until the bleeding stops. The wound will then be closed with special tape (Steri-strips), and covered with a bandage or sterile dressing. You will feel a stinging pain when the numbing medicine is injected but you should not feel anything during the biopsy. There is a risk of a small scar resulting from the skin biopsy.

 
Punch biopsy procedure
4.) Optional additional samples
If you have a bronchoscopy or biopsy of your stomach lining as a part of routine care, we would like to be able to collect any extra fluid from the bronchoscopy and/or any extra tissue from the biopsy for additional scleroderma research.  This is optional.

Study Contact Information:
Monika Benedict-Blue, BSW, CCRP
Linda Briggs, PhD, MPT, CCRP

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Drive
Lobby M, Ste 2500
Ann Arbor, MI  48106

Telephone:
(866) 628-9200

Study Status:
Currently Enrolling Patients
Informed consent required


Study Title:

Vascular Biomarker in patients with Systemic Sclerosis and Pulmonary Hypertension

IRB Number: HUM00049138

Study Team:

Coordinating Investigators:
Schiopu, Elena, MD, Department of Internal Medicine, Division of Rheumatology, University of Michigan, Scleroderma Program

Khanna, Dinesh, MD MS, Department of Internal Medicine, Division of Rheumatology, University of Michigan, Scleroderma Program

Phillips, Kristine, MD, PhD,  Department of Internal Medicine, Division of Rheumatology, University of Michigan, Scleroderma Program

Trivedi, Mona, MD, Department of Internal Medicine, Division of Rheumatology, University of Michigan, Scleroderma Program

Study Purpose:

Scleroderma is an autoimmune disease of the connective tissue.  Autoimmune diseases are illnesses that occur when the body's tissues are attacked by its own immune system. Scleroderma is characterized by the formation of scar tissue (fibrosis) in the skin and organs of the body.  Scleroderma can cause Pulmonary hypertension (PAH) which is increased blood pressure in the lungs caused by a thickening of the blood vessels.  The current “gold standard” test for PAH is the right heart catherization which is an invasive and costly procedure.  Because of this the test is not often used for screening of PAH but more so for confirming the diagnosis.  The aim of this study is to check if certain markers/substances found in blood and skin are present in patients with or without PAH and if these markers/substances fluctuate over time with changes in the severity of the disease and lung involvement in scleroderma. 

Who can take part in this study?

Persons who are 18 years of age and older, can read and write English, and fulfill the American College of Rheumatology criteria for systemic sclerosis.

Study Procedures:

There will be 7 visits where we will collect information from you.  Your participation in this study will take place during 7 of your routine clinical visits.  Information will be collected from you at start of study (baseline), and again at about months 6 and 12 and then yearly for up to 5 years.  At each of these visits the following will be done:

1). Fill out Questionnaires:

    You will be asked to complete several questionnaires.  One questionnaire is called the Health Assessment Questionnaire Disability Index (HAQ DI) and it will ask you questions about your disease and how it affects your daily living.  Another questionnaire will ask you questions whether you get short of breath and what makes you short of breath (these are called the UCSD Dyspnea Questionnaire.

    2). Allow us to record data related to your condition from your medical records

    The study team will also collect information about you by reviewing your medical chart at each visit about tests and procedures your doctor ordered.  These tests and procedures are part of your standard of care treatment and would be done even if you were not in this study. 

    3). Give a blood sample

    In addition to the regular blood tests, blood samples would be collected and stored for biomarker testing.  Biomarkers are laboratory tests used to better understand the disease process in scleroderma and other related diseases by testing proteins or substances in your body to measure the progress of a disease or the effects of a drug.   We will collect blood for biomarker testing at the baseline visit (first visit), 6 months, 12 months and then yearly for up to 5 years.   Approximately 30 ml of blood will be collected at each visit.  This blood will be taken from a vein in your arm using standard blood drawing procedures.  Blood samples for biomarker testing will be collected in tubes labeled with a study number only (no identifying information).   You will only be identified by your study number. 

    4). Skin Biopsies

    Skin biopsies are optional and are only performed once which is at your baseline visit.  These are 4mm punch biopsies (4mm is about the size of the eraser of a pencil). Up to two of these biopsies will be from skin on your arm (usually the forearm). 

    The procedure for a punch biopsy is relatively simple and should not take very long. You will first receive a shot of numbing medicine.  Next, a small, sharp tool that looks like a cookie cutter (punch) will be placed over the area of skin to be removed.  The tool will be pushed down, and then slowly rotated to remove a circular piece of skin. The skin sample will be lifted up with a tool called a forceps or a needle and then will be cut from the tissue below with sterile scissors. This is a small tissue sample and stitches are rarely needed. Pressure will then be applied to the site until the bleeding stops. The wound will then be closed with special tape (Steri-strips), and covered with a bandage or sterile dressing. You will feel a stinging pain when the numbing medicine is injected but you should not feel anything during the biopsy. There is a risk of a small scar resulting from the skin biopsy.

Study Coordinator:
Terry Thielan, LPN

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Drive
PO Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone:
(866) 628-9200

Study Status:
Currently Enrolling Patients
Informed consent required

 

Study Title:

ReCoRD – A database of patients with an autoimmune or rheumatologic disorder

IRB Number: HUM00036513

Study Team:

Principal Investigator:
Phillips, Kristine, MD, PhD, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Co-Investigators:
Schiopu, Elena, MD, Department of Internal Medicine, Division of Rheumatology, University of Michigan, Scleroderma Program

Khanna, Dinesh, MD MS, Department of Internal Medicine, Division of Rheumatology, University of Michigan, Scleroderma Program

Purpose:

The study doctor is creating a research database that will allow analysis of the medical record information of patients diagnosed with an autoimmune or rheumatologic disorder.  This will also permit review of the medical information to identify patients who may be eligible for participation in future research studies.

Who can take part in this study?

Males and females 18 years or older who may have an autoimmune, rheumatologic, or arthritis disease.

Study Procedures:

If you agree to take part in the project, we will gather information about you to be entered into the projects’ computer database. The type of information may include birth date, gender, race, ethnic background, family medical history, date of diagnosis, type of disease, organ involvement, results of blood tests, x-rays, and any other special diagnostic tests that you may have had.  This information will be obtained from your medical records and from questionnaires that you will complete. This project will require no new or additional tests.

Patients will see a study team member once at baseline.  Follow up questionnaires may be given during a regular clinical visit or mailed to you at month 6.  If possible, you will be seen by a study team member at one of your regular clinical visits or in the Michigan Clinical Research Unit or mailed questionnaires once a year for 3 years.  If you agree at each yearly visit you will have your blood drawn. .  Approximately 30 mL or about 3 tablespoons of blood is collected at each blood draw.  At the baseline, and year 1 to year 3 visits, patients will be asked to complete 11 questionnaires related to their symptoms and physical functioning.  Patients will also be advised that they may be contacted in the future to inquire about other studies in which they may be interested in participating.

The medical information stored in the Record-A Database may be shared with other research investigators who are not affiliated with U of M.  However, before any of these investigators are granted permission to use information in the database they must obtain approval to do so.

If during the course of your participation in this study you undergo a procedure to obtain a surgical biopsy or extra tissue is obtained during routine medical care, any extra samples that are available will be evaluated and stored for future studies. It is only if you agree that you will be asked to provide a sample of blood. 

Study Coordinator:
Linda Briggs, PhD, MPT

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Drive
PO Box 481, Lobby M, Suite 2500
Ann Arbor, MI  48106

Telephone:
(866) 628-9200

Study Status:
Currently Enrolling Patients
Informed consent required


Study Title:

A Phase 2a Randomized Double-Blinded, Placebo and Active Controlled Two Cohort Two Doses Cross-Over Multi-Center Clinical Study to Assess Efficacy of a Once Daily Administration of a Phosphodiesterase 5 Inhibitor (PF‑00489791) for the Treatment of Vasospasm in Primary and Secondary Raynaud’s Phenomenon, A7331010 (VAMPS

IRB Number: HUM00041158

Study Team:

Principal Investigator:
Schiopu, Elena, MD, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Purpose:

Study purpose: You are being asked to take part in a research study sponsored by Pfizer because you have Raynaud’s Phenomenon (RP), either primary (PRP)or secondary (SRP). For unknown reasons, blood vessels in your hands and maybe feet get smaller and the blood flow is restricted at certain times as in cold weather or when you are under stress.  PF-00489791 is an oral medication which works by inhibiting phosphodiesterase 5 (PDE-5) which is a chemical substance found in the body. This medication helps to make narrow blood vessels wider, so that blood can flow to the right places.

This study is being done to find out if PF-00489791 can be a safe and effective treatment for RP. PF‑00489791 is investigational and is not approved for sale to treat RP.  In addition, this study will evaluate how the medicine that is given in this study is absorbed and distributed, broken down and eliminated.  So far, approximately 250 adults have been given PF-00489791 in other studies.

Because this is a research study, PF-00489791 will be given to you only during this study and not after the study is over. If the decision is made to continue to develop this drug after the study is over, you may be allowed to enroll in another study but we cannot guarantee that a study will be available to you or when that may occur.

Because we do not know if PF-00489791 is effective in RP, two doses of PF-00489791 (4 and 20 mg) will be tested in this study along with placebo (pill that contains no active drug). You will receive both treatments (placebo and either the 4 or 20 mg dose) during the course of the study.  You will be randomly (like flipping a coin) assigned to a PF-00489791 dose. There is a 50% chance of receiving either the 4 or 20 mg PF-00489791 dose.  The study medication and placebo look identical so you will not know what you are taking.  Neither you nor your study doctor will know what dose of study medication you are receiving.  You doctor can get this information quickly if it is needed.  Certain other medications will not be allowed during the study and your doctor will discuss this with you.


Who can take part in this study?

You will be considered for this study if you have scleroderma, have active Raynaud’s Phenomenon and are between 18 and 65 years of age.

You must also be a non-smoker (smoke free for at least 3 months), free of alcoholism/drug abuse for at least 5 years, have no severe heart, liver, kidney, lung, etc. disease, or an unstable blood pressure.  Because some of the blood tests require that you fast for up to 8 hours, subjects with diabetes unable to tolerate this fasting may not participate. You should also discuss any eye disorders you may have, such as retinopathy, as there have been rare instances of visual disturbance while taking this medication. If you are pregnant or nursing, plan to get pregnant or are unwilling to use an acceptable form of birth control (See section on contraception on page 8) you may not participate in this study.  In addition to the above, there are several medications that you will not be allowed to take (from 2 weeks prior to beginning the study through the end of the study):

  • Potent cytochrome P450 3A4 inhibitors eg, itraconazole, erythromycin,
    ketoconazole, or protease inhibitors;
  • Nitrates or nitric oxide donors;
  • Ritonavir or Nicorandil;
  • Theophylline; pentoxifylline
  • Alpha blockers;
  • Iloprost;
  • Bosentan;
  • ACE-I or angiotensin receptor inhibitor;
  • Corticosteroids (unless on a stable dose of less than or equal to 10 mg of
  • prednisone a day or equivalent for at least 3 months);
  • Aspirin (except 81 mg per day or low-dose per local regulations);
  • Dipyridamole;/li>
  • Other antiplatelet agents (eg, Ticlopidine, Clopidogrel);/li>
  • phosphodiesterase inhibitors(eg, sildenafil, tadalafil, vardenafil)

Study Procedures:

If you agree to take part in the study and you continue to meet study requirements, you will be asked to come to the clinic every two weeks, a total of 9 times over a maximum of 120 days.  During 4 of the visits (Days 0, 14, 42, and 56), you will stay in the office for 4-6 hours so the doctor can assess your response to the study drug.  While you are in the study, you will have to complete a daily paper diary that will collect information about the number of RP attacks, how long they lasted, your Raynaud’s Condition score, pain due to your Raynaud’s and when you are taking your study medication.  This diary is returned at each visit.

Study Coordinator:
Julie Konkle, RN

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Drive
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone:
(866) 628-9200
(734) 763-3110

Study Status:

This study has been completed.


Study Title:

A Phase 1, Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Tolerability of MEDI-551 in Scleroderma

IRB Number:  HUM00031527

Study Team:

Principal Investigator:
Schiopu, Elena, MD, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Purpose:

Scleroderma is an autoimmune disease of the connective tissue. Autoimmune diseases are illnesses that happen when the body's tissues are attacked by the body’s own immune (protective) system. As a result, scar tissue (fibrosis) is formed in the skin and organs of the body. Scleroderma affects different people in different ways, but some of the symptoms that people with scleroderma can have include lung and breathing problems, thickening of areas of the skin, high blood pressure, kidney problems, poor digestion, joint pain, and poor blood circulation to the hands.

Current treatments for scleroderma are mainly drugs that change the immune system such as prednisone, mycophenolate mofentil, methotrexate, and cyclophosphamide.  Overall, these treatments are not very helpful in treating scleroderma so it is important to research new treatments that might be more effective. 

The purpose of this study is to see if a new drug, MEDI-551, is safe when given through a small tube inserted into a vein (an IV) of people who have scleroderma.  This new drug works by blocking Cluster of differentiation 19 (called CD19), a protein that is located on a type of white blood cell called a B cell.

In this study, we will also explore whether and how MEDI-551 leads to changes in scleroderma disease activity and skin thickening. The information learned in this study may be helpful in the further development of MEDI-551 for the treatment of scleroderma.

Who can take part in this study?

Both men and women can be in this study. Subjects in this study must be at least 18 years of age and have scleroderma, including moderate skin thickening in at least one place on the body such as the arms, legs, or trunk that is suitable for a skin biopsy. A skin biopsy is when a tiny piece of skin is removed from your lesion for testing. There will also be additional questions asked and testing done to make sure that you continue to qualify for the study.

Female subjects must be unable to become pregnant or must agree to avoid pregnancy from the time informed consent is signed through the end of the study. Female subjects who are sexually active must use 2 effective methods of avoiding pregnancy (including oral, transdermal, or implanted contraceptives, intrauterine device, female condom with spermicide, diaphragm with spermicide, cervical cap, or use of a condom with spermicide by the sexual partner) unless they are surgically sterile (eg, have had your “tubes tied” or had a hysterectomy) or at least 2 years after menopause. Female subjects must not be pregnant at the time of entry into the study. Male subjects must use 2 effective methods of avoiding pregnancy (condom with spermicide or abstinence) from the day of the first dose of study drug until the end of the study.  If the study drug is discontinued, male and female subjects must continue to use 2 effective methods of birth control for a period of 75 days (about 5 half-lives) following the last dose.

Study Procedures:

If you are in this study you will get either one dose or 5 doses of either MEDI-551 or placebo (dummy drug). If you are in a group that gets one dose, you will get it within 3 weeks after you sign the informed consent form, and you will return to the clinic for follow-up 2 days, 1 week, 2 weeks, 1 month, 2 months, and 3 months after the dose. If you are in a group that gets 5 doses, you will get the first dose within 3 weeks after you sign the informed consent form, return for follow-up 2 days and again 1 week after the first dose, and then get another dose 2 weeks, 1 month, 2 months, and 3 months after the first dose, for a total of 5 doses. You will return to the clinic for follow-up 2 days, 2 weeks, 1 month, 2 months, and 3 months (the last visit and the end of the study) after the last dose. If you are in a group that gets one dose, the study will last 3 months total.  If you are in a group that gets 5 doses, the study will last 6 months total. In the event that blood test results at the end of the study show that your numbers of B cells in your blood are low, you will be asked to return to the site for a blood test monthly for 3 months and every 3 months thereafter for 1 year, or until your B cell numbers have started to come back.

Unless you are the single subject in Group 1, there is one chance in 5 that you will get the placebo instead of MEDI-551. Unless you are the single subject in Group 1, neither you nor your doctor will know which you are getting.  If you are the single subject in Group 1, you will receive MEDI-551.

Study drug (MEDI-551 or placebo) will be given to you through the vein by IV infusion over at least 60 minutes in the lower dose groups and 120 minutes in the higher dose groups. You will also be asked not to take other experimental drugs, vaccines, or certain other treatments during the entire study from the time you sign the informed consent until the final visit, unless your doctor considers it medically necessary. Furthermore, if study drug is discontinued, you will be asked not to receive active vaccination within 75 days (5 half-lives) of last dosing.  If you decide at any time during the study that you do not want to receive any more doses of study drug, you will still be asked to return to the clinic for the scheduled tests to be performed.

If you are female and able to become pregnant, you will have blood and urine pregnancy tests done before your first dose of study drug and urine pregnancy tests before each later dose of study drug. You will not be able to enter the study or get additional doses of study drug if the pregnancy test is positive.

Optional DNA analysis
If you agree to take part in this study, you will be asked to sign a separate consent form for DNA analysis.  The DNA analysis is a genetic test. This test is entirely voluntary and optional. You do not have to take part in this substudy to be in the main study MI-CP200.

Study Contact Information

Study Coordinator:
Monika Benedict-Blue, BSW or Leah Kramer, BS

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Dr.
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI  48106
Telephone: (866) 628-9200

Study Status:

Currently Enrolling Patients
Informed consent required

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Study Title:

Digital Ischemic Lesions in Scleroderma Treated with Oral Treprostinil Diethanolamine:  An Open-Label Multicenter Extension Study (TDE-DU-202/DISTOL-EXT)

IRB Number:  HUM00031212

Study Team:

Principal Investigator:
Schiopu, Elena, MD, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Purpose:

Systemic sclerosis, also called scleroderma, is an autoimmune disease of the connective tissue.  Autoimmune diseases occur when the body's immune system attacks its own tissues.  Systemic sclerosis is a widespread connective tissue disease that involves changes in the skin, blood vessels, muscles, and internal organs. 

Treprostinil diethanolamine (UT-15C SR) is an investigational drug for the treatment of digital ulcers caused by systemic sclerosis or scleroderma.  An investigational drug is one that has not been approved by the Food and Drug Administration (FDA) or other agency as a prescription or over-the-counter drug.  This study drug is contained in a tablet that is taken by mouth and is released over an extended period of time (sustained release, or SR).  The purpose of this research study is to provide, or continue to provide, treprostinil diethanolamine (UT-15C SR) treatment for scleroderma patients completing the DISTOL-1 study (protocol TDE-DU-201) or the DISTOL-2 study (protocol TDE-DU-301) and to collect safety data over the duration of treatment.

Who can take part in this study?

Males and females greater than 18 years of age who have been diagnosed with systemic sclerosis (SSc) who currently have a finger ulcer. You should not or may not be eligible to participate in the study if you have other medical conditions such as pulmonary arterial hypertension (a type of high blood pressure in the lungs), be pregnant or fail to practice acceptable birth control, have severe diarrhea, anemia (low levels of iron in your blood), liver or kidney disease, arthritis, any form of tobacco or nicotine use, or have low blood pressure.  You may not be eligible to participate if you are on or have received certain medications, or have received certain treatments for your finger ulcers. Your doctor will review this with you.

Scleroderma patients that have completed the DISTOL-1 study (protocol TDE-DU-201) or the DISTOL-2 study (protocol TDE-DU-301).  Patients who stopped participation prior to completion of DISTOL-1 or DISTOL-2 may not enroll in this extension study.

Study Procedures:

If you choose to participate in this study, you will be required to visit the Michigan Clinical Research Unit up to 12 times over a -four year period.  You will begin treatment with UT-15C at 0.25 mg twice a day (with your morning and evening meals), and will work with your doctor to increase your dose until you reach the dose at which your doctor wishes you to remain.  This dose may be the same dose at which you were treated in the DISTOL-1 or DISTOL-2 study, or a different dose.  Though you may have reached an acceptable dose level on the DISTOL-1 or DISTOL-2 study, we will not know whether you were assigned to active treatment (UT-15C) or to placebo when you enter this continuation study.  Therefore, for your safety and comfort, all patients will begin with a low dose of UT-15C and increase their dose slowly over time to a dose of at least 1 mg, twice a day.

Study Contact Information

Study Coordinator:
Theresa Thielan, LPN or Julie Konkle, RN

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Dr.
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI  48106
Telephone: (866) 628-9200

Study Status:

This study is closed.

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Study Title:

Glomerular Filtration Rate in Scleroderma (DISTOL – GFR)Associated with: Protocol TDE-DU-201: DISTOL-1 Digital Ischemic Lesions in Scleroderma Treated with Oral Treprostinil Diethanolamine: A randomized, double-blind, placebo-controlled, multicenter study (DISTOL-1)

IRB Number: HUM00036731

Study Team:

Co-Investigators:
Phillips, Kristine, MD, PhD, University of Michigan, Department of Internal Medicine, Division of Rheumatology, Scleroderma Program

Principal Investigator:
Schiopu, Elena, MD, University of Michigan, Department of Internal Medicine, Division of Rheumatology, Scleroderma Program

Crystal Gadegbeku M.D., F.A.H.A., University of Michigan, Department of Internal Meidicine, Division of Nephrology

Purpose:

Systemic sclerosis, also called, is an autoimmune disease of the connective tissue.  Autoimmune diseases are diseases that occur when the body's immune system attacks its own tissues.  Systemic sclerosis is a widespread connective tissue disease that involves changes in the skin, blood vessels, muscles, and internal organs.  Kidney involvement is a potentially life-threatening complication for scleroderma patients.

Treprostinil diethanolamine (UT-15C SR) is an investigational drug for the treatment of digital ulcers caused by systemic sclerosis or scleroderma.  In addition, we would like to see what effect this drug has on the kidneys.  An investigational drug is one that has not been approved by the Food and Drug Administration (FDA) or other agency as a prescription or over-the-counter drug.  This study drug is contained in a tablet that is taken by mouth and is released over an extended period of time (sustained release, or SR).

The purpose of this research study is to determine if treprostinil diethanolamine (UT-15C SR) is effective at improving the blood flow to your kidneys, as part of a clinical trial of treprostinil diethanolamine for treatment of digital ulcers.

Who can take part in this study?

Only subjects who meet eligibility requirements, give their voluntary consent and are enrolled in Protocol TDE-DU -201 (the study for digital ulcers – DISTOL 1) may participate in this part of the study.   

If you have any condition, such as known allergy to iodine or contrast material, kidney problems, other chronic conditions or abnormal laboratory test results that, in the opinion of the investigator, pose an added risk to your health, you will not be able to participate in this study.  Also, if you have a health condition or an abnormal laboratory test result that may affect the outcome of the trial, you will not be enrolled.
Breastfeeding and/or pregnant women are also excluded.

Up to 25 participants will be enrolled in this study, all at the University of Michigan.

Study Procedures:

After enrolling in the digital ulcer study, the staff will determine if you would be a good candidate for the kidney function study.  Once enrolled in the study, assessments (blood draw, urine specimen, vital signs and physical exam) will be conducted at the Baseline, Week 5 and Week 20 clinic visits.

At Baseline, you will go to a special laboratory used for measuring kidney function.  About a teaspoonful of a contrast dye will be given through your IV.  This dye, called iohexol, completely passes through the body in the urine a few hours after it is given. Mild nausea has been reported to be associated with the use of the iohexol.

5 mL of Iohexol (»3200 mg determined by weight) will be administered intravenously as a single injection as per previously validated technique and 1 mL blood samples will be taken at 5, 10, 15, 180, 240 and 300 minutes.

Your participation from baseline until study completion will last 20 weeks (140 days). During the study you will come to the hospital for 3 visits to have the the kidney test. These visits, done at baseline, week 5 and week 20, may take up to to 6 hours, and will be done along with the visits for the DISTOL 1 study that occur on the same dates. You will have 3 additional visits for the DISTOL 1 study which is explained in the consent for DISTOL 1.  There will be no additional visits for this study.

Once you have completed the week 20 visit, your participation in the study will be complete.

Study Contact Information:

Study Coordinator:
Terry Thielan, LPN or Linda Briggs, PhD, MPT

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Dr.
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI  48106
Telephone: (866) 628-9200

Study Status:

This study is closed.

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Study Title:

A two-stage prospective observational cohort study in scleroderma patients to evaluate screening tests and the incidence of pulmonary arterial hypertension (PAH) and pulmonary hypertension (DETECT)

IRB Number:   HUM00021876

Study Team:

Principal Investigator:
Phillips, Kristine, MD, PhD, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Co-Investigators:
McLaughlin, Vallerie, M.D., University of Michigan, Internal Medicine, Cardiology

Rubenfire, Melvyn, M.D., FACP, FACC, FACCP Professor, Department of Internal Medicine Director, Cardiovascular Medicine at Domino's Farms

Purpose:

Pulmonary hypertension (PAH) is increased blood pressure in the lungs from scleroderma, caused by the thickening of the blood vessels.   It is one of the more serious complications in scleroderma.  Patients with early PAH may have little warning before they develop severe shortness of breath.
The aims of this research study are (1) to evaluate different screening methods to predict and confirm Pulmonary Hypertension (PH) in scleroderma patients and (2) to evaluate the incidence of PH (i.e. number of new cases per year) in scleroderma patients.

  1. Evaluation of screening methods

There is a need to determine a reliable, convenient and cost-efficient screening method for PH.  The current “gold standard” test for PH is the right heart catheterization (RHC), an invasive and costly procedure. Due to its invasiveness RHC is only used for confirmation but not for screening of PH.

Several potential screening methods have been proposed for the early detection of PH, but proof of their value is currently limited.  This study will investigate the value of the following methods for early detection of PH in patients with scleroderma.

  • Electrocardiography (ECHO) Also known as a cardiac ultrasound, this test uses ultrasound to create two-dimensional images of your heart.
  • Electrocardiography (ECG): An electrocardiogram (EKG or ECG) is a test that records the electrical activity of your heart and checks for problems with your heart.

  • Pulmonary Function Testing (PFT): These tests determine how much air your lungs can hold, how quickly you can move air in and out of your lungs, and how well your lungs add oxygen to and remove carbon dioxide from your blood.
  • 6 Minute Walk Test (6MWT)/Borg Dyspnea: In this test you will be asked to walk for 6 minutes. The distance walked and the degree of oxygen saturation during exercise (using a simple patch taped to the forehead) will be measured.  In this test you will also be asked to rate your breathing discomfort on a scale from 0 to 10.
  • Blood Tests – Approximately 5 teaspoons will be collected to check for different substances in your blood that are thought to contribute to the progression of pulmonary hypertension.
  • Right Heart Catheterization (RHC): In this test a catheter (small wire) is inserted into a vein in your upper leg or groin area.  Using a TV screen and x-rays the physician carefully threads this wire up to the right side of your heart. A pressure sensor on the end of the wire will measure the blood flow and blood pressures in your heart and lung.
  1. Incidence of PH in scleroderma
    This study should provide additional information on how many scleroderma patients will develop PH per year.

Who can take part in this study?

You can participate in the study if you meet the following criteria:

  • Male or female
  • Age ≥ 18 years
  • Patients with definite diagnosis of Ssc (Systemic Sclerosis) by American College of Rheumatology (ACR) criteria
  • Patients with any other connective tissue diseases (CTD) who, at the same time, meet the ACR criteria for SSc
  • SSc disease duration > 3 years.
  • Diffusing capacity of the lung for carbon monoxide (DLCO) is below 60% of predicted

Study Procedures:

If you are included in the study you will have certain medical procedures and tests performed at each visit over a total of 3 years. For more information about the study and the study procedures:

http://clinicaltrials.gov/ct2/show/NCT00706082

Study Contact Information:

Study Coordinator:
Julie Konkle, RN, BSN

Mailing Address:

University of Michigan Scleroderma Program
Domino’s Farms
24 Frank Lloyd Wright Dr.
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI  48106

Telephone: (866) 628-9200

Study Status:

Recruitment is complete.

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Study Title:

An Evaluation of the Pharmacokinetics and Safety of Fixed and Escalating Doses of Oral Treprostinil Diethanolamine (UT-15C) Sustained Release Tablets in Patients with Systemic Sclerosis(DISTOL-PK)

IRB Number:  HUM00022112

Study Team:

Principal Investigator:
Schiopu, Elena, MD, University of Michigan, Internal Medicine, Scleroderma Program

Co-Investigators:
Phillips, Kristine, MD, PhD, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Purpose:

Systemic sclerosis, also called, is an autoimmune disease of the connective tissue.  Autoimmune diseases are diseases that occur when the body's immune system attacks its own tissues.  Systemic sclerosis is a widespread connective tissue disease that involves changes in the skin, blood vessels, muscles, and internal organs.  Raynaud’s phenomenon causes a reduction of blood flow to the fingers, toes, nose, or ears due to spasm in the blood vessels of these areas.  These spasms can be triggered by cold temperatures or emotional stress.

Treprostinil diethanolamine (UT-15C SR) is an investigational drug for the treatment of digital ulcers caused by systemic sclerosis or scleroderma.  An investigational drug is one that has not been approved by the Food and Drug Administration (FDA) or other agency as a prescription or over-the-counter drug.  This study drug is contained in a tablet that is taken by mouth and is released over an extended period of time (sustained release, or SR). The purpose of this research study is to assess how persons with systemic sclerosis tolerate treprostinil diethanolamine (UT-15C SR) and to determine how quickly persons with systemic sclerosis absorb treprostinil diethanolamine (UT-15C SR) into the bloodstream, and eliminate it from the body.  Blood levels of treprostinil diethanolamine (UT-15C SR) will be measured by taking multiple small blood samples. 

Who can take part in this study?

Males and females greater than 18 years of age who have been diagnosed with systemic sclerosis (SSc) who currently have a finger ulcer or who have had a finger ulcer within the past 6 months and experiencing a minimum of 6-10 Raynaud‘s phenomenon attacks per week may be eligible participate.

You should not or may not be eligible to participate in the study if you have other medical conditions such as pulmonary arterial hypertension (a type of high blood pressure in the lungs), liver or kidney disease, arthritis, smoke, or have low blood pressure.  You may not be eligible to participate if you are on or have received certain medications, or have received certain treatments for your finger ulcers. Your doctor will review this with you.

Study Procedures:

For more information about the study and the study procedures:

 http://clinicaltrials.gov/ct2/show/NCT00848939

Study Contact Information:

Study Coordinator:
Terry Thielan, LPN

Mailing Address:
University of Michigan Scleroderma Program
Domino’s Farms
24 Frank Lloyd Wright Dr.
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI  48106

Telephone: (866) 628-9200

Study Status:

This study has been completed

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Study Title:

A Randomized, Open-Label, Phase II/III Multicenter Study of High-Dose Immunosuppressive Therapy Using Total Body Irradiation, Cyclophosphamide, ATGAM, and Autologous Transplantation with Auto-CD34+HPC versus Intravenous Pulse Cyclophosphamide for the Treatment of Severe Systemic Sclerosis (SCOT)

IRB Number: 2005-0485

Study Team:

Principal Investigator:
Phillips, Kristine, MD, Ph.D, University of Michigan Department of Internal Medicine, Rheumatology

Co-Investigators:
Mineishi, Shin, MD, University of Michigan Dept. of Internal Medicine, Hematology-Oncology

Ferrara, James, MD, University of Michigan Dept. of Pediatrics and Internal Medicine

Kaplan, Mariana, MD, University of Michigan Dept. of Internal Medicine, Rheumatology

Lee, Choon-Kee, MD, University of Michigan Dept. of Internal Medicine

Levine, John, MD, University of Michigan Dept. of Pediatrics, Hematology-Oncology

Reddy, Pavan, MD, University of Michigan Dept. of Internal Medicine, Hematology-Oncology

Schiopu, Elena, MD, University of Michigan Dept. of Internal Medicine, Rheumatology

Christina, Tsien, MD, University of Michigan Dept. of Internal Medicine, Radiation Oncology

Purpose:

Severe systemic sclerosis (SSc) is a serious autoimmune disorder in which a person’s own immune cells attack organs in the body. SSc affects the skin, joints, lungs, heart, intestinal tract, and kidneys, and half of the patients with the most severe organ involvement die within 5 years. Treatment for SSc usually includes supportive care or immunosuppressive drugs (drugs to suppress (hold back)) your immune system. As the immune cells are believed to be causing the disease, researchers are looking for new therapies that either slow down or stop this process. The main purpose of this study is to determine the safety and effectiveness of high-dose immunosuppressive therapy followed by re-infusion (transplantation) of the subject’s own autologous (self) peripheral blood stem cells (PBSCs) compared to treatment with monthly (for 12 months) intravenous doses of cyclophosphamide (Cytoxan) therapy for the treatment of severe systemic sclerosis (SSc). These treatments are being given in order to determine if they will slow down or stop SSc from becoming more severe, and if they can reverse the effects of the disease. We are evaluating the effects of the two treatments on serious organ damage and survival related to SSc, while also studying the side effects of the two treatments.

Who can take part in this study?

This study is for patients with severe systemic sclerosis (SSc), also known as scleroderma.

Study Procedures:

For more information about the study and the study procedures: http://www.sclerodermatrial.org

OR

http://clinicaltrials.gov/ct/show/NCT00114530?order=4

Study Contact Information:

Study Coordinator:
Leah Kramer, BS

Mailing Address:
University of Michigan Scleroderma Program
Domino's Farms
24 Frank Lloyd Wright Drive
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106
Telephone: (866) 628-9200

Study Status:

This study has been completed

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Study Title:

A Randomized, Double-blind, Placebo-phase Multi-center, Phase 2 Trial of Intravenous Rituximab in the Treatment of Refractory Myositis in Adults and Children (RIM)

IRB Number: HUM 00003757 (Pediatric) & HUM 00001117 (Adult)

Study Team:

Coordinating Investigator:
Oddis, Chester, MD, Professor of Medicine, Division of Rheumatology and Clinical Immunology, University of Pittsburgh School of Medicine

Reed, Ann, MD, Chair, Pediatric Rheumatology, Mayo Foundation and School of Medicine at the Mayo Clinic

Co-Investigators at the University of Michigan:
Schiopu, Elena, MD, Department of Internal Medicine, University of Michigan (Adult)

Ike, Robert, MD, Department of Internal Medicine, University of Michigan (Adult)

Adams, Barbara, MD, Department of Pediatric Rheumatology, University of Michigan (Pediatric)

Haftel, Hilary, MD, Department of Pediatric Rheumatology, University of Michigan (Pediatric)

Purpose:

Myositis is the general term used to describe inflammation of the muscles.  Dermatomyositis (DM), polymyositis (PM), inclusion-body myositis (IBM) and juvenile forms of myositis (JM & PM) are considered inflammatory myopathies (diseases of the muscle with swelling and loss of muscle). Inflammatory myopathies are thought to be autoimmune meaning the body’s immune system, which normally fights infections and viruses, does not stop fighting once the infection or virus is gone.  The immune system then attacks the body's own normal, healthy tissue through inflammation or swelling.  All of these diseases can cause progressive muscle weakness and characteristic rashes.  A myositis diagnosis is often confirmed by a muscle biopsy.  The drug Rituximab has been used successfully in treating certain cancer patients in the past.  Rituximab is being used more frequently in the treatment of patients with autoimmune syndromes and is well-tolerated even in children.  Other drugs, including steroids that have been used in the past to treat autoimmune disease have led to numerous serious side effects including weight gain, glucose intolerance, cataracts, glaucoma, osteoporosis and growth arrest as well as sterility and organ damage that can be life threatening.  Rituximab may provide an alternative drug therapy to prevent disease damage in adult and pediatric patients with inflammatory myopathy based on data from several previous studies.

Who can take part in this study?

Male and female adults and children who have been diagnosed with either DM or PM will be asked to take part in this research study.  This study will be done at approximately 37 centers across North America and Europe.  202 participants will be included in the study (152 adults and 50 children).  Approximately 12 people will participate at the University of Michigan.

Study Procedures:

Baseline data collected will include demographics, disease severity, past history of medications and surgeries, current medications, laboratory data and test results from routine diagnostic testing (EKG & manual muscle testing).  Enrolled patients will be asked to complete questionnaires throughout the study at follow up visits. At baseline, enrolled subjects will be asked to if they would like to participate in two other studies related to this study that involve extra blood for DNA testing and muscle biopsies.  Patients may choose to participate in the main study without participating in the two extra studies.
Subjects will receive a total of 4 doses (infusions) of study drug.  At visit 2 they will be randomized (assigned by chance) to one of two groups.  They will receive both placebo (an inactive solution of saline) and rituximab while participating in this study.

Group A will receive the study drug, rituximab at Visit 2 and Visit 3 followed by a placebo infusion only at Visit 5 and Visit 6.

Group B will receive a placebo infusion only at Visit 2 and Visit 3 followed by an infusion of the study drug rituximab at Visit 5 and Visit 6.

This study is double-blind, which means that the subject and the study staff will not know what study drug they are receiving at each of the four infusion visits.  However, this information is available to the study doctor if needed in an emergency.
Enrolled subjects will have routine blood work done on a regular basis.  Data from these tests will be collected and entered into a database, along with data from the biannual questionnaires and patient assessments.
 
The entire length of the study with 4 infusions and all assessments will continue for 44 weeks.  There are 14 necessary visits to the University of Michigan.

Study Contact Information:

Study Coordinator:
University of Pittsburgh:
Sherrie Pryber, Clinical Study Coordinator

Mailing Address:
University of Pittsburgh
Division of Rheumatology
S 703A BST
3500 Terrace Street
Pittsburgh, PA 15261
pryber@dom.pitt.edu

Telephone:  (412) 647-3241

Study Contact Information:

Study Coordinators:
University of Michigan Scleroderma Program:
Leah Kramer, BS

Mailing Address:
University of Michigan Scleroderma Program
Domino's Farms
24 Frank Lloyd Wright Drive
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

Recruitment is complete.

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Study Title:

An Open Label Pilot Study to Assess the Safety and Efficacy of Inhaled Iloprost in Adults with Pulmonary Hypertension Secondary to Interstitial Lung Disease in Systemic Sclerosis

IRB Number: HUM00006149

Study Team:

Co-Investigators:
Schiopu, Elena, MD, Department of Internal Medicine, University of Michigan

McLaughlin, Vallerie, MD, Department of Internal Medicine, University of Michigan

Ojo, Tammy Clark, MD, Department of Internal Medicine, University of Michigan

Chan, Kevin, MD, Department of Internal Medicine, University of Michigan

Purpose:

Lung involvement in scleroderma is present in approximately 80% of the patients with varying contributions of inflammation, scarring and blood vessel injury.  Individuals with blood vessel injury develop pulmonary arterial hypertension (PAH) and have benefited from numerous advances in treatment.  This trial investigates the safety and effectiveness of one of these treatments in a group of patients who have pulmonary hypertension and moderately severe lung scarring.  The treatment, inhaled iloprost, will be administered to 10 well characterized volunteers.  The usefulness of the drug will be assessed by serial exercise testing over 12 weeks.  This is a pilot study; information from which may provide the basis for longer and larger clinical trials.

Who can take part in this study?

Patients (age 18 to 75) with diagnosed pulmonary hypertension that is due to interstitial lung disease will be screened for eligibility by a multidisciplinary team including a cardiologist, pulmonologist and a scleroderma specialist.  This study will include approximately 10 patients.

Study Procedures:

Patients who are eligible for this study will come to the General Clinical Research Center at University of Michigan for a screening visit followed by 4 monthly study visits over a total period of 16 weeks.  Each visit will take approximately 4-5 hours.  During the first study visits, patients will be instructed in using the inhalation device by trained clinical personnel.  Inhaled iloprost should be taken 6-9 times a day for the duration of the study and specialized clinical personnel will be continuously available.To assess safety of the inhaled iloprost, patients will undergo the following procedures at each study visit: medical exam, current medication review, review of adverse events (AEs), vitals signs and oxygen saturation (by forehead probe).  During the first and last visit, subjects will also have blood samples drawn (2 teaspoons). During the study visits, patients will undergo a 6 minute walk test (done at each of the 5 visits) and pulmonary function tests (done at the beginning and at the end of the study).  They will also be asked to complete questionnaires assessing shortness of breath (Borg and UCSD) and overall health (SHAQ-DI, 20 questions). As part of the study, subjects will answer one question regarding Raynaud’s phenomenon at each study visit.

Study Contact Information:

Study Coordinator:
Julie Konkle, RN, BSN

Mailing Address:
University of Michigan Scleroderma Program
Domino's Farms
24 Frank Lloyd Wright Drive
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

This study has been completed

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Study Title:

Pulmonary Hypertension - Assessment and Recognition of Outcomes in Scleroderma (PHAROS)

IRB Number: HUM 00001965

Study Team:

Coordinating Investigator:
Steen, Virginia D., MD, Professor of Medicine, Georgetown University School of Medicine

Co-Investigator at the University of Michigan:
Schiopu, Elena, MD, Department of Internal Medicine, University of Michigan

Purpose:

The purpose of this research project is to establish a database of patients with scleroderma who developed or are at increased risk of developing increased pressure in the pulmonary artery called pulmonary arterial hypertension (PAH).  There is minimal prospective data regarding the natural history of PAH in patients with systemic sclerosis and no definite risk factors have been identified to date.  Although there are outcome studies showing PAH as an independent risk for death in cohorts of scleroderma patients, little is known about the initial, pre-PAH stages and how it evolves into severe PAH.  This database will be used to determine the natural history of this complication from early stages to different clinical outcomes and to establish through various testing what the risk factors are for the development of severe pulmonary hypertension.

Who can take part in this study?

Scleroderma patients with a recent diagnosis of pulmonary hypertension or those who are at high risk for developing pulmonary hypertension will be eligible to enroll in this study.  ‘Early PAH’ will include patients that have been diagnosed within the past 6 months that meet certain eligibility requirements.  Patients who are ‘at risk for developing PAH’ must meet certain eligibility requirements from pulmonary function tests and echocardiograms.  These tests are routine for scleroderma patients.  Patients that have been diagnosed and treated for pulmonary hypertension for more than 6 months will not be eligible for this study.

Study Procedures:

Baseline data collected will include demographics, disease severity, past history of medications, current medications, laboratory data and test results from routine diagnostic testing.  Enrolled patients will be asked to complete questionnaires on a biannual basis.  At baseline, enrolled subjects will be asked to give a blood sample that will be frozen and stored for possible future analysis.  Patients may choose to participate in this study without having their blood drawn for possible future analysis.  Enrolled subjects will have routine annual pulmonary function tests, echocardiograms and cardiac catheterization (if warranted) as part of their standard care.  Data from these tests will be collected and entered into a database, along with data from the biannual questionnaires and patient assessments.
This database and patients’ assessments will continue for up to 10 years (contingent on future funding), allowing a better understanding of this complication.

Study Contact Information – Georgetown University:

Study Coordinator:
Georgetown University:
Suria Yesmin, Clinical Study Coordinator

Mailing Address:
Georgetown University
PHC Building
Dept. of Rheumatology
3800 Reservoir Road, 6th Floor
Washington, DC 20007

Telephone:  (202) 687-6317

Study Contact Information:

Study Coordinator:
University of Michigan Scleroderma Program:
Julie Konkle, RN, BSN

Mailing Address:
University of Michigan Scleroderma Program
Domino's Farms
24 Frank Lloyd Wright Drive
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

Recruitment is complete

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Study Title:

Validation of the Michigan Hand Outcomes Questionnaire as a Disease-Specific Outcome Measure for Scleroderma

IRB Number: HUM 00000390

Study Team:

Principal Investigator:
Chung, Kevin, MD, MS, Department of Plastic Surgery, University of Michigan

Purpose:

Hand involvement is universal in patients with scleroderma. Studies have identified upper extremity impairment as the dominant factor influencing patient functional status; however, the development of a hand-specific instrument to follow hand function as a primary or secondary end-point may be an important advancement in scleroderma research. This study attempts to validate the Michigan Hand Outcomes Questionnaire (MHQ), an outcomes instrument developed at the University of Michigan, as a disease-specific functional outcomes tool in scleroderma.

Who can take part in this study?

Consenting adults over the age of 18 with a diagnosis of systemic sclerosis (according to classification criteria of the American College of Rheumatology) and who have clear evidence for a reliable classification as diffuse or limited cutaneous scleroderma can participate in this study. Ineligibility for the study is based on the following conditions: active hand infection, ischemic digit (a decrease in the blood supply caused by constriction or obstruction of the blood vessels) or established demarcated proximal digital gangrene (distinguishable gangrene of the fingers), anticipated need for surgery in the next 6 months, and an unstable systemic disease (for instance, a new onset of scleroderma renal crisis).

Study Procedures:

Study participants will be given the Michigan Hand Outcomes Questionnaire (MHQ), the SF-36 and the HAQ-DI to complete while waiting to be seen in the clinic for routine clinical care. These surveys can be completed in approximately 30 minutes. Participants may be asked to return for a second visit within the next 14 days. The same routine clinical care and MHQ will be re-administered in the second visit. If you are unable to return to the clinic for a second visit, the MHQ may be mailed to you for you to complete at home and mail back to us.

Study Contact Information:

Study Coordinator:
Sandra Kotsis, MPH

Mailing Address:
2130 Taubman Center
1500 East Medical Center Drive
Ann Arbor, MI 48109-0340

Telephone: 734-615-6550

Study Contact Information:

Study Coordinator:
Julie Konkle, RN, BSN

Mailing Address:
University of Michigan Scleroderma Program
Domino's Farms
24 Frank Lloyd Wright Drive
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

Currently Enrolling Patients

Informed consent required.

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Study Title:

Searching for Clues to the Pathobiology of Pulmonary Hypertension

IRB Number: 2005-0082

Study Team:

Principal Investigator:
Rubenfire, Melvyn, MD, Professor of Internal Medicine, University of Michigan

Co-Investigators at the University of Michigan:
Kaplan, Mariana, MD, Assistant Professor of Internal Medicine

McLaughlin, Vallerie, MD, Associate Professor of Internal Medicine

Martinez, Fernando, MD, Professor of Internal Medicine

Pinsky, David, MD, Professor of Internal Medicine

Peters-Golden, Marc MD, Professor of Internal Medicine

Standiford, Theodore, MD, Professor of Internal Medicine

Ruwende, Cyril, MD, PhD, Assistant Professor of Internal Medicine

Schiopu, Elena, MD, Lecturer, Internal Medicine

Co-Investigators at the Institute of Respiratory Disease; University of Milan
Blasi, Francesco, MD

Bossone, E duardo, MD, PhD

Bodini, Bruno, MD, (Visiting Research Fellow, University of Michigan)
Specialty School of Occupational Medicine; University of Milan

Lippo, Giusseppina, MD, (Visiting Research Fellow, University of Michigan)

Purpose:

The purpose of this study is to obtain a better understanding of the triggers and modulators of pulmonary arterial hypertension. This may lead to improved and targeted therapies. Pulmonary hypertension (PH) or elevation of the pulmonary artery pressure results from the body’s response to various stimuli including disease states, which, when chronic, can lead to pathologic changes in the blood vessels of the lung.

Who can take part in this study?

Patients with scleroderma, pulmonary fibrosis, and other causes of pulmonary arterial hypertension are eligible. We also will need healthy normals, and patients with coronary artery disease. The study will include approximately 170 subjects.

Study Procedures:

  • Medical Records Review: the research team will review your medical records at the University of Michigan and information made available by other physicians. This information will be entered into the research record.
  • Blood Draw: up to 4 tablespoons of blood will be drawn. An additional 6ml (1 teaspoon) blood sample will be drawn if you agree to participate in the genetic component of this study. This blood will be used to test for DNA analysis relating to pulmonary hypertension and related diseases.
  • Cardiac Ultrasound: If you have not had a cardiac ultrasound at the University for clinical purposes in the past 3 months, one will be performed.
  • Follow-up: Your medical progress may be followed by the research team for up to 5 years. You will be contacted yearly by phone or by mail to obtain information regarding your health status. Your physician may be requested to provide information regarding your progress. Additionally you may be invited to have follow-up blood tests and an ultrasound of the heart at intervals of 2 and 4 years.

Study Contact Information:

Study Coordinator:
Karen Schultz, BBA

Mailing Address:
Preventive Cardiology
24 Frank Lloyd Wright Drive
Ann Arbor, MI 48106-0363

Telephone: 734-998-7411

Study Contact Information:

Study Coordinator:
Leah Kramer, BS

Mailing Address:
University of Michigan Scleroderma Program
Domino's Farms
24 Frank Lloyd Wright Drive
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

This study has been completed

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Study Title:

Scleroderma and Myositis Cohort

IRB Number: 2002-0748

Study Team:

Co-Investigators:
Schiopu, Elena, MD, Department of Internal Medicine, University of Michigan

Somers, Emily, PhD, Department of Internal Medicine, University of Michigan

Kocheril, Sosa, MD, Department of Internal Medicine, University of Michigan

Purpose:

This is a descriptive registry that has been active at the University of Michigan for many years. Key information about disease characteristics, severity and outcomes are catalogued. A main objective is to develop solid information about the natural history of disease.

Study Contact Information:

Study Coordinator:
Julie Konkle, RN, BSN

Mailing Address:
University of Michigan Scleroderma Program
Domino's Farms
24 Frank Lloyd Wright Drive
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

This study has been closed

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Study Title:

Development of Scleroderma Gastrointestinal Tract 2.0 (SSC-GIT 2.0) Quality of Life Instrument

IRB Number:  HUM00015237

Study Team:

Coordinating Investigator(s):
Furst, Daniel, MD, Khanna, Dinesh, MD, Clements, Philip, MD, from the Rheumatology Division at the University of California, Los Angeles (UCLA) initiated this study and UCLA is the coordinating center.  The University of Michigan is subcontracted by UCLA and is one of the three co-investigating sites. 

Co-Investigators at the University of Michigan:

Schiopu, Elena, MD, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Phillips, Kristine, MD, PhD, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Purpose:

Scleroderma is an autoimmune disease of the connective tissue.  Autoimmune diseases are illnesses that occur when the body's tissues are attacked by its own immune system. Scleroderma is characterized by the formation of scar tissue (fibrosis) in the skin and organs of the body. This leads to thickening of involved areas.
Gastrointestinal tract (GIT) is the most common organ system involved in scleroderma.  Organs of the gastrointestinal tract include the mouth, throat, stomach, intestines, and anus.  GIT involvement in scleroderma can be severely debilitating and even life threatening.  Some problems associated with GIT may include heartburn, loss of voice or hoarseness, ulcers (open sores), difficulty swallowing, constipation, diarrhea, malabsorption (impaired absorption of nutrients from the GI tract), diminished peristalsis (decreased in the wavelike motion in the muscles of the intestines), and the inability to control your bowel movements.

Researchers at UCLA have developed a new quality of life instrument to assess the severity of your GIT involvement due to scleroderma and its affect on physical and mental well-being. The purpose of the study is to validate this instrument for scleroderma care and future clinical trials focusing on improving GIT symptoms.

Who can take part in this study?

This study is for patients with scleroderma who are over the age of 18 and had bothersome GIT signs and symptoms in the last 3 months.

Study Procedures:

If you agree to participate in this study, you will complete several questionnaires during your routine visits at the clinic.  This study requires 2 times at which you will complete the questionnaires.  They will be separated by 3 to 6 months.  At each visit the following will be done:

  • Complete SF-36 Questionnaire.  This questionnaire will ask you questions about how you feel and how well you are able to do your usual activities,
  • Complete SSC-GIT 1.0 Questionnaire.  This questionnaire will ask you questions about your GIT disease and how it is affecting your life,
  • Complete the Scleroderma – Health Assessment Questionnaire (S-HAQ) Questionnaire.  This questionnaire will ask you questions about any vascular (blood vessels) problems you may have such as Raynaud’s phenomenon and digital ulcers, any GIT symptoms, any lung symptoms, and your overall severity of your disease,
  • Complete the Center for Epidemiological Study of Depression Scale (CESD) Questionnaire.  This questionnaire will ask you questions about your mood and it’s affects on your daily activities,
  • Complete Categorical Self Rating of Severity and Activity of Overall, Upper, and Lower GI Involvement questions.  For example, “During the past 1 week, how severe were your overall gastrointestinal (gut) symptoms due to your scleroderma?”

We will also collect information about you by reviewing your medical chart at each visit.  During these visits tests may be ordered, however, these tests are part of your standard of care treatment and not part of this study.  You would have these tests even if you were not in this study.  The information collected may be from the following tests and procedures:

  • Demographic data.  This includes year of birth, gender, ethnic origin, annual income, year your disease was diagnosed,
  • GIT symptoms and severity,
  • Medications you are taking for your GIT problem.
  • Skin score (skin thickness at 17 different areas)

The questionnaires that you completed will be stored at UCLA for 10 years after the completion of all data analysis or 20 years, whichever is longer and then your data will be destroyed.
Once you have completed the 2 visits, your participation in this study is completed.  You will continue to return to the clinic for your routine visits and have tests and procedures done as part of your standard of care treatment.

Study Contact Information:

Study Coordinator:
University of Michigan Scleroderma Program:
Julie Konkle, RN, BSN

Mailing Address:
University of Michigan Scleroderma Program
Domino's Farms
24 Frank Lloyd Wright Drive
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

Recruitment is complete

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Study Title:

Endostatin and Other Angiogenic/Angiostatic Mediators in Patients with Systemic Sclerosis

IRB Number: HUM 11196

Study Team:

Coordinating Investigator(s):
Jaffe, Michele L, MD, MPH, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Seibold, James R, MD, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Koch, Alisa E, MD, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Purpose:

Changes in the tubes that carry blood through the body (blood vessels) and increased fibrous material in the body (collagen) production are common to all patients with scleroderma. These abnormalities lead to both decreased ability to function and death.  Proteins in the body that act on the tubes that carry blood through the body are important in creating abnormalities in these tubes.  One protein that is produced as a breakdown product of the abnormal fibrous tissue seen in patients with scleroderma and acts on these tubes is called endostatin.  Skin biopsy samples and blood samples will be evaluated for differences in the level of endostatin between subjects with scleroderma and healthy subjects.  Three other proteins that act on the tubes have been found in preliminary studies to be abnormal in patients with scleroderma as compared to healthy controls. These proteins are called CXCL16, JAM-A and JAM-C.  Skin biopsy samples and blood samples will be evaluated for differences in the levels of these proteins between subjects with scleroderma and controls  If any of these proteins is changed in subjects with scleroderma as compared to controls, they may become a target for drug therapies.

Who can take part in this study?

Persons who are 18 years of age and older, can read and write English, have systemic sclerosis (scleroderma) with skin involvement above the elbow and/or knee or have no chronic health conditions which require daily use of medications can participate in the study.  Persons are not eligible for the study if their systemic sclerosis is a result of being exposed to chemicals or drugs that can cause a scleroderma-like illness, they have an overlap of systemic sclerosis with another autoimmune disease (for example, lupus or rheumatoid arthritis) or have been treated with a medication called cyclophosphamide in the past 8 weeks.  Also, subjects are not eligible if they report a history of active infection (including hepatitis C, hepatitis B, or HIV) or are prone to bleeding because they are treated with medications that thin the blood or have a low platelet count.  All participants must provide written informed consent and be willing to comply with laboratory tests.

Study Procedures:

On the first day, all subjects and healthy controls will sign the informed consent, and have two tubes of blood drawn from their arm to confirm that they do not have an excessive tendency to bleed.  Later that day, or on any of the next 30 days following signing of the informed consent, all subjects and healthy controls will have their blood drawn and skin biopsied and subjects with scleroderma will have a brief medical history and an examination of their joints and skin will be performed.  All subjects with scleroderema and healthy controls will have 10 cc (two tubes) of blood drawn from their arm.  For subjects with scleroderma, the skin will be anaesthetized (numbed with a shot of numbing medication under the skin) in the lower and upper arm and two samples of their skin will be biopsied.  For healthy subjects, the skin will be anaesthetized (numbed with a shot of numbing medication under the skin) in the lower arm and one sample of their skin will be biopsied.  In patients with scleroderma and in healthy controls, the skin will be anaesthetized with a small amount of lidocaine placed under the skin with a needle.

Study Contact Information:

Study Coordinator:
University of Michigan Scleroderma Program:
Leah Kramer

Mailing Address:
University of Michigan Scleroderma Program
Domino's Farms
24 Frank Lloyd Wright Drive
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

Recruitment is complete

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Study Title:

Provisional Combined Response Index for Patients with Diffuse Systemic Sclerosis Pulmonary Hypertension (CRISS)

IRB Number: HUM 00015154

Study Team:

Coordinating Investigators:
Furst, Daniel E, MD, Khanna, Dinesh, MD, and Clements, Philip, MD from the Rheumatology Division at the University of California, Los Angeles (UCLA) initiated this study and UCLA is the coordinating center.

Co-Investigators at the University of Michigan:
Phillips, Kristine, MD, Ph.D, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Schiopu, Elena, MD, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Purpose:

Scleroderma is an autoimmune disease of the connective tissue.  Autoimmune diseases are illnesses that occur when the body's tissues are attacked by its own immune system. Scleroderma is characterized by the formation of scar tissue (fibrosis) in the skin and organs of the body. This leads to thickening of involved areas. Diffuse Scleroderma is diagnosed whenever there is tight skin above the elbows or knees (the face doesn’t count in this definition).  The cause of scleroderma is not known, however, inheritance (or genes) seems to play a role in this disease as do some environmental factors.  In scleroderma, internal organs including the lungs, heart, digestive tract, kidney, muscles, and nerves can be involved.

There is no approved treatment by the Food and Drug Administration (FDA) for scleroderma.  The purpose of the study is to develop a response criteria (measure) for clinical trials in diffuse scleroderma that can assist in developing new therapies and hopefully, can lead to effective treatments for scleroderma.

Who can take part in this study?

This study is for patients with early diffuse scleroderma (less than 5 years of disease) who are over the age of 18.

Study Procedures:

Participation in this study will take place during 3 routine clinical visits: baseline (start of study), and again at about months 6 and 12.  Data collected includes demographics, disease severity, past history of medications, current medications, laboratory data and test results from routine diagnostic testing.  A skin examination (part of routine clinic visit) will be conducted to determine how hard and thick the skin is by using a device called a durometer, which applies light pressure to parts of your arms and legs for a few seconds at a time.
Patients will complete several questionnaires.  One questionnaire is called the Health Assessment Questionnaire Disability Index (HAQ DI) and asks questions about the disease and how it affects activities of daily living.  Another questionnaire is called SF-36 and it asks questions about quality of life.  Other questionnaires asks about shortness of breath (BORG Dyspnea Scale and St. George’s Respiratory Questionnaire).  Questions are also asked regarding how Raynauds’s (a condition that causes some areas of your body — such as your fingers, toes, tip of your nose and your ears — to feel numb and cool in response to cold temperatures or stress) affects your function and about how your digestive system affects you.  These questionnaires should take about 30 to 40 minutes at each visit to complete. 

Study Contact Information:
University of California Los Angeles (UCLA):

Principal Investigator:
Dinesh Khanna, MD

Mailing Address:
University of California Los Angeles
Dept. of Rheumato</strong><br>logy
1000 Veteran Ave, #32-59 Rehab
Los Angeles, CA 90024

Telephone:  (310) 825-2448

Study Contact Information:

Study Coordinator:
University of Michigan Scleroderma Program:
Julie Konkle, RN, BSN

Mailing Address:
University of Michigan Scleroderma Program
Domino's Farms
24 Frank Lloyd Wright Drive
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

Recruitment is complete

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Study Title:

Pain, Pain Self-efficacy, and Depression in Scleroderma

IRB Number: HUM 00014737

Study Team:

Principal Investigator:
Impens, Ann, Ph.D, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Co-Investigators:
Jaffe, Michele, MD, MPH, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Phillips, Kristine, MD, PhD, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Schiopu, Elena, MD, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Purpose:

The purpose of this research project is to obtain a better understanding of the level of pain and depression experienced by scleroderma patients and its impact on quality of life.  Pain reported by scleroderma patients has been linked to physical functioning but surprisingly little research has been done on the importance and impact of pain in this patient population.

Who can take part in this study?

English-speaking patients of the University of Michigan Scleroderma Clinic aged 18 and older are eligible to participate in this study.

Study Procedures:

Study participants are asked to complete several questionnaires.  These questionnaires will ask questions about the level of pain they experience, how they deal with this pain, about depression, and quality of life.  Participants will also be asked some questions about how their scleroderma affects their day-to-day activities and about the severity of their disease. 
As part of this study, information about their disease will also be taken from medical records.  This information includes: demographic information (e.g., age, gender, race), length of disease, type of disease, medications, digital ulcers, and other organ involvement. in this study.  .

Study Contact Information:

Study Coordinator:
Julie Konkle, RN, BSN

Mailing Address:
University of Michigan Scleroderma Program
Domino's Farms
24 Frank Lloyd Wright Drive
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

This study is on hold.

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Study Title:

A Phase 1B, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate Safety of Multiple-Dose, Intravenously Administered MEDI-545, a Fully Human Anti-Interferon-Alpha Monoclonal Antibody, in Adult Patients with Dermatomyositis or Polymyositis

IRB Number: HUM00016798

Study Team:

Principle Investigator:
Schiopu, Elena MD, University of Michigan, Internal Medicine, Scleroderma Program

Co-Investigators:
Phillips, Kristine MD, PhD, University of Michigan, Internal Medicine, Scleroderma Program

Purpose:

Myositis is an inflammation of your skeletal muscles, which are also called the voluntary muscles. These are the muscles you consciously control that help you move your body. In myositis, your immune system functions abnormally and attacks cells and organs in your own body. The diseases dermatomyositis and polymyositis both involve myositis. Polymyositis causes muscle weakness, usually in the muscles closest to the trunk of your body. Dermatomyositis causes muscle weakness, plus a skin rash. Both diseases are usually treated with prednisone, a steroid medicine, and sometimes other medicines that inhibit the immune system. Based on previous studies, MEDI-545 may reduce the muscle and skin symptoms in people with myositis.

This study will help find out whether MEDI-545 is safe in people with myositis, how well it is tolerated when it is injected directly into the vein (called an intravenous or IV infusion), and the amount of MEDI-545 in the blood when the drug is given at different doses. In addition, we will examine whether MEDI-545 reduces the symptoms of myositis and prevents muscle breakdown. We will also test whether your body generates antibodies against MEDI-545 and, if it does, how much antibodies are made.

The information learned in this study may be helpful in the further development of MEDI-545 for the treatment of myositis.

Who can take part in this study?

Both men and women can be in this study. Volunteers in this study must be at least 18 years old and have dermatomyositis or polymyositis as determined by their doctor.

Women must not be pregnant and agree not to become pregnant during the study. This includes the period from screening until Study Day 350. Women who are sexually active must use an effective method of avoiding pregnancy such as oral, injectable, transdermal, or implanted contraceptives, intrauterine device, female condom with spermicide, diaphragm with spermicide, cervical cap, abstinence, use of a condom with spermicide by the sexual partner (or sterile sexual partner) from screening through end of study, unless they are surgically sterile (eg, have had “tubes tied” or had a hysterectomy) or at least 2 years postmenopausal. Male volunteers must use two effective methods of avoiding pregnancy (condom with spermicide or abstinence) from the day of the first dose of study drug until the end of the study.

Study Procedures:

The study will take place over about one year from screening to the end of the study. You will come to the study site for a total of 23 visits, plus one or more screening visits. You will be asked not to take certain medications during the entire study from screening to the final visit, unless your doctor considers it medically necessary. If you decide, at any time during the study, that you do not want to receive any more infusions of study drug, you will still be asked to return to the clinic for the scheduled tests to be performed.

Mandatory Interferon-alpha Gene Expression analysis

As a part of this study, you are asked to give blood samples for interferon-alpha gene expression signature analysis. Genes are messages coded in a type of molecule called DNA. DNA contains many genes, which are instructions for making living organisms. RNA is a type of molecule that makes proteins from various genes, a process known as “gene expression.” Proteins are the building blocks and the tools that form cells and tissues and organs and carry out all the functions of the body. Myositis may change the amount and patterns of RNA for certain proteins, especially interferon alpha, that are involved in the immune system. By analyzing the levels of these RNA molecules in blood, we may better understand how MEDI-545 affects interferon-alpha gene expression.

It is important to know that RNA analysis is different from DNA analysis or genetic testing. Rather, the RNA molecules studied are related to DNA but only contain instructions for the production of specific proteins, such as interferon alpha. Your blood samples for RNA analysis will not be used to identify genetic information that could tell people about your risk of getting certain diseases. You must agree to have blood drawn for RNA analysis to take part in this study.

For more information about the study and the study procedures:
http://clinicaltrials.gov/ct2/show/NCT00533091

Study Contact Information:

Study Coordinator:
Leah Kramer

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Dr.
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

Recruitment is complete.

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Study Title:

A Phase 1 Multi-center, Open-label Study to Evaluate the Safety and Tolerability of Single and Multiple Intravenous Doses of MEDI-546, a Fully Human Monoclonal Antibody Directed Against Subunit 1 of the Type I Interferon Alpha Receptor, in Adult Subjects with Scleroderma

IRB Number: HUM00029740

Study Team:

Principle Investigator:
Schiopu, Elena MD, University of Michigan, Internal Medicine, Scleroderma Program

Co-Investigators:
Phillips, Kristine MD, PhD, University of Michigan, Internal Medicine, Scleroderma Program

Purpose:

The main goal of this study is to see whether MEDI-546 is safe for people with scleroderma. This study will also see how well MEDI-546 is tolerated when it is given directly into the vein (called an intravenous or IV infusion). This study will also measure the amount of MEDI-546 in the blood when the drug is given at different doses and whether your body makes antibodies against MEDI-546.

Who can take part in this study?

Both men and women can be in this study. You must be at least 18 years of age and have active scleroderma, including thickening of the skin in at least one place on the body such as the arms, legs, or trunk. Females must be unable to become pregnant or must agree to avoid pregnancy from the time the informed consent is signed through the end of the study by agreeing to use effective birth control. There will also be additional questions asked and testing done to make sure that you continue to qualify for the study.

Study Procedures:

The study will take place over 7 to 9 months, from the time you sign the informed consent until the last study day for the last subject entered. If you qualify for the study you will come to the study site for a total of 8 to 10 visits, depending on whether you are in the group that receives 1 dose of MEDI-546 or the group that receives 4 doses of MEDI-546, plus one or more screening visits. MEDI-546 will be given to you through the vein by IV infusion over at least 30 minutes. You will also be asked not to take other experimental drugs or vaccines during the entire study from the time the informed consent is signed to the final visit, unless your doctor considers it medically necessary. If you decide at any time during the study that you do not want to receive any more infusions of MEDI-546, you will be asked to return to the clinic for the scheduled tests to be performed. If you are female and able to become pregnant, you will have blood and urine pregnancy tests done before your first dose of MEDI-546. You will not be able to enter the study if the pregnancy test is positive.

Mandatory RNA analysis
As a part of this study, blood will be taken for RNA (gene) testing. Genes are messages coded in a type of molecule called DNA. DNA contains many genes, which are the instructions for making living organisms. RNA is a type of molecule that makes proteins from various genes, a process known as “gene expression.” Proteins are the building blocks and the tools that form cells and tissues and organs and carry out all the functions of the body. Scleroderma may change the amount and patterns of RNA for certain proteins, such as interferon alpha, that are involved in the immune system. By analyzing the levels of these RNA molecules in blood, we may better understand how MEDI-546 affects the way certain genes are “expressed” in the disease and whether MEDI-546 has any effects on the gene expression. It is important to know that RNA analysis is different from DNA analysis or genetic testing. The RNA molecules studied are related to DNA but only contain instructions for the production of specific proteins, such as interferon alpha. Your blood samples for RNA analysis will not be used to identify genetic information that could tell you about your risk of getting certain diseases. You must agree to have blood drawn for RNA analysis to take part in this study.

Mandatory skin biopsy
If you agree to be in this study, you will be asked to provide two 4 mm (about the size of a pencil eraser) punch biopsies of skin at two different time points during the study. A punch biopsy is the removal of a piece of skin using a “cookie cutter” like punch tool. The skin area where you will have the biopsy will be cleaned and injected with a local anesthetic which may sting for the first few seconds. The doctor will check that the area is numb before doing the biopsy. A stitch is sometimes used to close each biopsy site. You may end up having a very small scar. This will occur on Study Day 0 (before you receive MEDI-546) and again on Study Day 7 (if you are in a group that gets 1 dose of MEDI-546) or Study Day 28 (if you are in a group that gets 4 doses of MEDI-546).

For more information about the study and the study procedures:

http://clinicaltrials.gov/ct2/show/NCT00930683

Study Contact Information:

Study Coordinator:
Monika Benedict-Blue, BSW

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Dr.
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

Recruitment is complete.

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Study Title:

Fibrocyte Phenotypes as Biomarkers in IPFnet Patients

IRB Number: HUM00023436

Study Team:

Principle Investigator:
Moore, Bethany B, Ph.D, University of Michigan, Internal Medicine and Microbiology and Immunology

Co-Investigators:
Phillips, Kristine MD, PhD, University of Michigan, Internal Medicine, Scleroderma Program

Schiopu, Elena MD, University of Michigan, Internal Medicine, Scleroderma Program

Purpose:

Fibrotic lung disease causes an accumulation of scar tissue in the lung and the eventual loss of lung function. Prior research suggests that circulating fibrocytes (cells in the blood that can contribute to scar tissue formation) may be elevated in patients with some forms of lung fibrosis. We are trying to determine whether the number of fibrocytes in the circulation of patients with lung fibrosis correlated with the severity of their lung disease. We also plan to study fibrocytes obtained from the circulation to try to understand how they contribute to the development of disease. For purposes of comparison, we will study these cells in the circulation of patients with idiopathic pulmonary fibrosis (a fibrotic lung disease of unknown cause), scleroderma and COPD as well as in normal volunteers.

Who can take part in this study?

This study is looking for three populations of patients.

1. Subjects with a confirmed diagnosis of Scleroderma over the age of 18 who undergo routine pulmonary function testing.
2. Subjects with a confirmed diagnosis of COPD over the age of 18 who undergo routine pulmonary function testing.
3. Subjects, male or female, over the age of 18 without evidence of active pulmonary (lung) or systemic (throughout the body) infection, and without significant heart disease.

Study Procedures:

Blood will be drawn using a needle and tubing from a ven in your arm. Aprroximately 20cc (4 tablespoons) of blood will be collected. We would like to collect blood 3 times at approximately 9 month intervals. We will also access the medical records of subjects with Scleroderma or COPD to obtain information about their pulmonary function testing which is performed as part of their routine clinical care.

Study Contact Information:

Study Coordinator:
Terry Thielan, LPN

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Dr.
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

Recruitment is complete.

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Study Title:

Novel Biomarkers in Clinical Pulmonary Syndromes

IRB Number: HUM00028972

Study Team:

Principle Investigator:
Moore, Bethany B, Ph.D, University of Michigan, Internal Medicine and Microbiology and Immunology

Co-Investigators:
Phillips, Kristine MD, PhD, University of Michigan, Internal Medicine, Scleroderma Program

Schiopu, Elena MD, University of Michigan, Internal Medicine, Scleroderma Program

Purpose:

Fibrotic lung disease causes an accumulation of scar tissue in the lung and the eventual loss of lung function. It is believed that this is related to lung injury. Following injury, the body produces fibrinogen in a healing response. We will test for the presence of fibrinogen fragments in the plasma from fibrosis and control patients to see if fibrinogen fragments are elevated in patients with fibrosis. These studies may identify a biomarker to help us diagnose lung fibrosis. For purposes of comparison, we will study patients with IPF or scleroderma who have lung fibrosis and will compare them to patients with COPD or normal volunteers who do not have fibrosis.

Who can take part in this study?

This study is looking for four populations of patients:
1. Subjects with a confirmed diagnosis of Scleroderma with lung involvement over the age of 18.
2. Subjects with a confirmed diagnosis of COPD over the age of 18.
3. Subjects with a confirmed diagnosis of IPF over the age of 18.
4. Subjects, male or female, over the age of 18 without evidence of active pulmonary (lung) or systemic (throughout the body) infection, and without significant heart disease.

Study Procedures:

Blood will be drawn using a needle and tubing from a vein in your arm. Approximately 5 cc (1 tablespoon) of blood will be collected. Once collected, Dr. Moore’s laboratory will collect the plasma from your blood. Plasma is the non-cellular portion of your blood. Once the plasma is obtained, Dr. Moore will ship the plasma sample to Pfizer in St. Louis, MO where they will analyze the plasma sample for the presence of fibrinogen A and fibrinogen B fragments using techniques called liquid chromatography and mass spectrometry. The only identifying information that will be on the plasma sample sent to Pfizer will be the disease diagnosis. No personal information will be shipped. Once the measurements have been made, the data will be sent back to Dr. Moore for evaluation. Results will be kept until published in scientific journals.

Study Contact Information:

Study Coordinator:
Terry Thielan, LPN

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Dr.
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

Recruitment is complete.

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Study Title:

Scleroderma Pulmonary Hypertension Quality Enhancement Research Initiative (S-QuERI)

IRB Number: HUM00013792

Study Team:

Principal Investigator:
Phillips, Kristine, MD, PhD, Internal Medicine, Division of Rheumatology, University of Michigan

Co-Investigators:
Schiopu, Elena MD, University of Michigan, Internal Medicine, Scleroderma Program

Purpose:

The purpose of this study is to develop a Registry (database) of patients with scleroderma who therefore may be at risk for developing Pulmonary Artery Hypertension (PAH). Pulmonary hypertension is increased blood pressure in the lungs from scleroderma, caused by the thickening of the blood vessels. It is one of the more serious complications in scleroderma. This registry will be used to follow the procedures and treatments used by your doctor in the diagnoses and treatment of pulmonary hypertension. You are being asked to allow data from your clinical procedures to be collected about your diagnosis and management.

A number of therapies have been approved for this condition and your doctor will select one or more of them when needed as part of your treatment and according to current guidelines for the treatment of your disease. Therefore, there is no new or investigational medication used as part of this Registry.

Who can take part in this study?

This study is for patients with scleroderma over the age of 18 whom have not been diagnosed with pulmonary hypertension.

Study Procedures:

All procedures in this study are standard test that are performed routinely in the care of scleroderma patients. There will be no procedures done for this research. You will be treated with medication(s) chosen by your doctor. As part of this registry, data will be collected about your management including the tests performed and the treatment that you receive. This information will be entered in a database so that the researchers can analyze the information obtained from all individuals participating in this study. You will be assigned a study number which has no identifying information. This means that information shared with the coordinating center will not have your name or other identifying information. You are being asked to allow your physician to share your information for up to 3 years to assess how effective the tests are in detecting pulmonary hypertension and to follow the treatment prescribed by your doctor.

Study Contact Information:

Study Coordinator:
Leah Kramer, BS

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Dr.
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

Recruitment is complete

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Study Title:

Evaluation of the expression profile of inflammation and fibrosis mediators in skin, blood and urine from subjects with diffuse systemic sclerosis (SSc) (CENTOCOR)

IRB Number: HUM00023617

Study Team:

Principal Investigator:
Phillips, Kristine, MD, PhD, Department of Internal Medicine, Division of Rheumatology, University of Michigan

Co-Investigators:
Schiopu, Elena MD, University of Michigan, Internal Medicine, Scleroderma Program

Purpose:

The purpose of the study is to determine if “interleukin 13” (IL-13) and related molecules are present in the skin, blood, or urine of subjects with diffuse SSc . Interleukins are proteins, basic components of living cells that are part of the immune system.

Who can take part in this study?

If you choose to volunteer for this research study, the study doctor will let you know if you can or cannot participate.

Study Procedures:

If you qualify and agree to participate in this study, the following will occur:

Subjects with diffuse SSC:

  • If you have diffuse SSc you will have 4 small pieces of skin removed; two from an area of involved skin (usually from your lower arm), and two from an area of uninvolved skin (usually your upper arm). If you do not have an area of suitable uninvolved skin, only the 2 biopsies from
  • The area will be cleaned and injected with a local anesthetic which may sting for the first few seconds. The doctor will check that the area is numb before doing the biopsy.
  • On your lower arm, the biopsy will remove two 4 mm pieces of skin (about the size of a pencil eraser).
  • On your upper arm, the biopsy will remove another two 4 mm pieces of your skin.
  • A stitch is sometimes used to close each biopsy site. This helps with healing and could leave a very small scar.

If you have diffuse scleroderma, you may have had some of the assessments named below done previously as a part of your routine care, or as a part of another study. If information from these tests is available, we would like to use it for this study, but you will not have to undergo these assessments for the specific purpose of this research.

• Tendon friction rubs
• MRSS (If not obtained within the last 3 months)
• Durometer skin score
• Tender joint count
• Active digital tip ulcer count
• Physician global assessment
• Physician skin disease activity assessment
• Scleroderma-related health transition by physician
• History of Scleroderma renal crisis
• Pulmonary function tests
• Right heart catheterization (only if clinically indicated)
• 6-minute walk test (only if clinically indicated)
• Borg dyspnea index
• Doppler echocardiogram
• Labs/bloodwork including autoantibody profile
• High resolution CT of the chest (only if clinically indicated)
• Scleroderma Health Assessment Questionnaire
• Short form (SF)-36
• Patient Skin Disease Activity Assessment
• Patient Global Assessment

Healthy Control Volunteers:
If you are a volunteer you will have 2 small pieces of skin removed from your lower arm.
The area will be cleaned and injected with a local anesthetic which may sting for the first few seconds. The doctor will check that the area is numb before doing the biopsy.
On your lower arm, the biopsy will remove two 4 mm pieces (about the size of a pencil eraser) of skin.
A stitch is sometimes used to close each biopsy site. This helps with healing and could leave a very small scar.

All subjects:
We will collect blood and urine from you to check for factors that might play a role in diffuse SSc. The blood will be drawn through a needle inserted into a vein in your lower arm. The nurse will draw about 3 tablespoons of blood from you. We will ask you to collect urine in a cup. Five to eight hair pieces of hair will also be collected by plucking hair from your head. In addition, we would like you to answer some simple questions about how you are feeling in the form of a questionnaire, which will take about 10 minutes.

Your skin biopsies, hair sample, urine sample, and blood sample will be sent to Centocor and other labs where they will be analyzed factors believed to play role in diffuse SSc. Information about your age, sex, race, and medical history will also be sent to Centocor.

You will be called by telephone 7 to 10 days after your visit to see how you are feeling and if you have had any problems because of your participation in the research study.

Optional DNA Testing
There is a second part to this study that involves testing your DNA using about 1 ½ teaspoons of your blood. DNA makes up your “genes” and helps to determine traits like eye color, skin color, blood type, and many other things that make you unique. DNA is passed down from your parents. Differences in our genes help explain why we all look different. Differences in our genes may also help explain why some drugs work and are safe in some people, but not in others. Differences in our genes also help explain why some people get certain diseases, but others do not. In this study we want to see if there are differences in the genes of people with diffuse SSc by comparing them to the genes of healthy controls. If you want to take part in this study you will need to read and sign the separate DNA consent form that is attached at the end of this document. This part of the study is totally voluntary.

Study Contact Information:

Study Coordinator:
Monika Benedict-Blue, BSW

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Dr.
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

This study has been completed

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Study Title:

Genome-Wide Association Study in Systemic Sclerosis (GWAS)

IRB Number: HUM00030376

Study Team:

Coordinating Investigator:
Mayes, Maureen D, MD, MPH, University of Texas - Houston Health Science Center

Participating Investigators:
Schiopu, Elena MD, University of Michigan, Internal Medicine, Scleroderma Program

Phillips, Kristine MD, PhD, University of Michigan, Internal Medicine, Scleroderma Program

Purpose:

The purpose of the study is to develop a bank of DNA, RNA, serum and plasma samples from scleroderma patients. The second purpose of the project is to test the samples (Genome Wide Association Scan, ANA, auto antibodies, and other markers) to answer the question, whether there is a genetic link to scleroderma and if so, which genes are involved.

Specifically, the study, Genome Wide Association Studies (GWAS), will compare your genetic information (information passed down to you from your parents that determines all of your traits such as your eye color and body size) with genetic information from other individuals to look for similarities and differences that may help doctors find new or better treatments for many diseases (such as asthma, cancer, diabetes, heart disease, mental illnesses, etc).

If you agree to take part in this study your DNA (genetic information) will undergo genome-wide analysis (all of the instructions that make up your body) and your genotypic (the genetic code) and phenotypic (the expression of the genetic code) data will be shared for research purposes through the NIH GWAS data repository.

Who can take part in this study?

This study is for patients who have been diagnosed with scleroderma. This research will address questions about the study of genes that may cause scleroderma or make it more severe or less severe.

Study Procedures:

If you agree to take part in the project, we will gather information about you, to be entered into the projects computer database. The type of information will include birth date, social security number (optional), gender, racial/ethnic background, date of diagnosis, type of scleroderma, degree of skin and internal organ involvement, and results of blood tests, x-rays and heart and lung tests. This information will be obtained from your doctors, from hospitals that you may have been admitted to, and from a written questionnaire that you will complete. In addition, medical records will be requested to verify diagnosis. The social security number is optional and is recorded in order to ensure that individuals are not entered more than once. You realize that the project will require no new or additional tests.

You also agree to have blood drawn. This will occur one time and be about 55cc (4.0 tablespoons) of blood. Blood samples will be banked indefinitely for future research in scleroderma and other autoimmune diseases. Blood samples will be banked for an indefinite time for future research. DNA samples become property of the University of Texas Health Science Center-Houston. This research will address questions regarding the study of genes that may cause scleroderma or make it more severe or less severe. Some samples may also be used to try to find better tests for this disease. These samples will be shared with other investigators without identifying information.
Deidentified samples (all identifying information removed) will be provided to other researchers upon approval by the Scleroderma Registry Scientific Advisory Committee. You should be aware that your samples may ultimately be used in studies of other diseases unrelated to scleroderma. If you object to this, you should not participate.

Study Contact Information:

Study Coordinator:
Terry Thielan, LPN

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Dr.
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

Currently Enrolling Patients
Informed consent required

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Study Title:

Digital Ischemic Lesions in Scleroderma Treated with Oral Treprostinil Diethanolamine: A randomized, double-blind, placebo-controlled, multicenter study (DISTOL-1)

IRB Number: HUM00025768

Study Team:

Principal Investigator:
Schiopu, Elena, MD, University of Michigan, Internal Medicine, Scleroderma Program

Co-Investigators:
Phillips, Kristine MD, PhD, University of Michigan, Internal Medicine, Scleroderma Program

Purpose:

Systemic sclerosis, also called scleroderma, is an autoimmune disease of the connective tissue. Autoimmune diseases occur when the body's immune system attacks its own tissues. Systemic sclerosis is a widespread connective tissue disease that involves changes in the skin, blood vessels, muscles, and internal organs.

Treprostinil diethanolamine (UT-15C SR) is an investigational drug for the treatment of digital ulcers caused by systemic sclerosis or scleroderma. An investigational drug is one that has not been approved by the Food and Drug Administration (FDA) or other agency as a prescription or over-the-counter drug. This study drug is contained in a tablet that is taken by mouth and is released over an extended period of time (sustained release, or SR).

The purpose of this research study is to determine if treprostinil diethanolamine (UT-15C SR) is effective at healing your digital ulcers, monitor formation of any new digital ulcers, and to determine the most effective dose.

Who can take part in this study?

Males and females greater than 18 years of age who have been diagnosed with systemic sclerosis (SSc) who currently have a finger ulcer. You should not or may not be eligible to participate in the study if you have other medical conditions such as pulmonary arterial hypertension (a type of high blood pressure in the lungs), be pregnant or fail to practice acceptable birth control, have severe diarrhea, anemia (low levels of iron in your blood), liver or kidney disease, arthritis, any form of tobacco or nicotine use, or have low blood pressure. You may not be eligible to participate if you are on or have received certain medications, or have received certain treatments for your finger ulcers. Your doctor will review this with you.

Study Procedures:

For more information about the study and the study procedures:
http://clinicaltrials.gov/ct2/show/NCT00775463

Study Contact Information:

Study Coordinator:
Linda Briggs, PhD, MPT

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Dr.
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

Recruitment is complete.

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Study Title:

Mycophenolate vs. Oral Cyclophosphamide in Scleroderma Interstitial Lung Disease (SLS-II)

IRB Number: HUM00019590

Study Team:

Principal Investigator:
Phillips, Kristine, MD, PhD, University of Michigan, Internal Medicine, Scleroderma Program

Co-Investigators:
Schiopu, Elena MD, University of Michigan, Internal Medicine, Scleroderma Program

Purpose:

The purpose of this study is to compare mycophenolate and cyclophosphamide for effectiveness at preventing ongoing lung damage from scleroderma, and for safety.

Who can take part in this study?

You can participate in this study if you are between 18 and 75 years of age, and have had scleroderma for at least 5 years with evidence of lung involvement (such as shortness of breath).

Study Procedures:

For more information about the study and the study procedures:
http://clinicaltrials.gov/ct2/show/NCT00883129

Study Contact Information:

Study Coordinator:
Monika Benedict-Blue, BSW

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Dr.
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

Currently Enrolling Patients
Informed consent required

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Study Title:

An Open-Label Study to Evaluate the Safety of Dasatinib in the Treatment of Scleroderma Pulmonary Fibrosis

IRB Number: HUM00024672

Study Team:

Principal Investigator:
Schiopu, Elena, MD, University of Michigan, Internal Medicine, Scleroderma Program

Co-Investigators:
Phillips, Kristine, MD, PhD, University of Michigan, Internal Medicine, Scleroderma Program

Purpose:

The purpose of this open-label study is to evaluate the safety of dasatinib in patients with moderate to severe scleroderma-associated pulmonary fibrosis.
Dasatinib is currently approved by the United States Food & Drug Administration (FDA) for patients who have a certain type of leukemia (CML). It is currently being studied in subjects who have cancerous tumors including prostate and breast cancer. Use in both of these cancers is considered an “experimental therapy.”
In animal and other laboratory experiments dasatinib has shown to decrease fibrosis (or scarring) of skin and lungs. Thus, there is reason to believe that dasatinib might be useful in scleroderma-associated pulmonary fibrosis.

Who can take part in this study?

People who are at least 18 years old and have established diffuse scleroderma and who meet certain criteria for skin and lung disease involvement can participate in this study.

Study Procedures:

For more information about the study and the study procedures:
http://clinicaltrials.gov/ct2/show/NCT00764309

Study Contact Information:

Study Coordinator:
Linda Briggs, PhD, MPT

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Dr.
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

Recruitment is complete.

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Study Title:

A Study of a Topical Formulation of Nitroglycerin, Vascana® (MQX-503), and Matching Vehicle in the Treatment and Prevention of Symptoms Associated With Raynaud's Phenomenon

IRB Number: HUM00029561

Study Team:

Principal Investigator:
Schiopu, Elena, MD, University of Michigan, Internal Medicine, Scleroderma Program

Co-Investigator:
Phillips, Kristine, MD, PhD, University of Michigan, Internal Medicine, Scleroderma Program

Purpose:

Raynaud's Phenomenon is a sometimes debilitating condition in which blood flow to the fingers or toes is compromised when a patient is exposed to cold or stress. Current therapies for Raynaud's are suboptimal because they are associated with significant side effects. In this study, patient responses to cold temperature exposures in a climate-controlled room after application of the study medication are measured.

Who can take part in this study/Study procedures?

For more information about who can take part in this study and information on the study procedures: http://clinicaltrials.gov/ct2/show/NCT00934427

Study Contact Information:

Study Coordinator:
Julie Konkle, RN, BSN

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Dr.
P.O. Box 481, Lobby M, Suite 2500
Ann Arbor, MI 48106

Telephone: (866) 628-9200

Study Status:

Recruitment complete


Study Title:

A Phase 2 Open-label Study to Evaluate the Long-term Safety of Sifalimumab in Adult Subjects with Systemic Lupus Erythematosus or Myositis (CP212)

IRB Number: HUM00040791

Study Team:

Coordinating Investigator:
Schiopu, Elena MD, University of Michigan, Internal Medicine, Scleroderma Program

Study Purpose:

The main goal of the study is to see if sifalimumab is safe in subjects with lupus or myositis when it is given for a long period of time. We will evaluate how well it is tolerated when it is given directly in the vein (called an intravenous or IV infusion) and need to determine the amount of sifalimumab in the blood. We will also test whether your body generates antibodies against sifalimumab and, if it does, how many antibodies are made. In this study, we will also examine changes in your disease activity and symptoms. The information learned in this study may be helpful in the further development of sifalimumab for the treatment of lupus and myositis.

Who can take part in this study?

Subjects over the age of 18 who complete the treatment period and follow up through Day 266 for either MI-CP151 and MI-CP152 or Day 168 for MI-CP179 subjects may participate in this study.  This includes both sifalimumab and placebo subjects. This does not include subjects who stopped study drug (sifalimumab or placebo) in MI-CP151, MI-CP152, or MI-CP179 for safety reasons. Female subjects must not be pregnant at time of entry into the study.

Study Procedures:

The study will take place over about 3 years and 4 months, which includes up to 28 days for screening, 36 months for administration of study drug, and a 12 week follow-up period after the last dose of study drug. You will come to the study site for screening procedures and then for up to 44 visits for treatment and follow-up.   

In this study, subjects will receive sifalimumab in the vein every 14 days during the first 28 days and then every 4 weeks for 3 years at a fixed dose of 500 mg. You will be asked not to take certain medications during the entire study from screening to the final visit, unless your doctor considers it medically necessary.

Study Contact Information:

Study Coordinator:
Leah Kramer

Mailing Address:
University of Michigan Scleroderma Program
24 Frank Lloyd Wright Drive
PO Box 481, Lobby M, Suite 2500
Ann Arbor, MI  48106

Telephone:
(866) 628-9200

Study Status:
Recruitment is complete


 

 

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