• 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)
  
  
• 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)
  
  
• Consortium of Rheumatology Researchers of North America, Inc. (CORRONA) Data Collection Program
  The CORRONA – New York University (NYU) Pharmacogenomics Substudy
  
  
• 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)
  
  
• 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)
  
  
• Pulmonary Hypertension - Assessment and Recognition of Outcomes in Scleroderma (PHAROS)
  
  
• Validation of the Michigan Hand Outcomes Questionnaire as a Disease-Specific Outcome Measure for Scleroderma
  
  
• Development of Scleroderma Gastrointestinal Tract 2.0 (SSC-GIT 2.0) Quality of Life Instrument
  
  
• Endostatin and Other Angiogenic/Angiostatic Mediators in Patients with Systemic Sclerosis
  
  
• Provisional Combined Response Index for Patients with Diffuse Systemic Sclerosis Pulmonary Hypertension (CRISS)
  
  
• Pain, Pain Self-efficacy, and Depression in Scleroderma
  
  
• 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
  
  
• 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
  
  
• Fibrocyte Phenotypes as Biomarkers in IPFnet Patients
  
  
• Novel Biomarkers in Clinical Pulmonary Syndromes
  
  
• Scleroderma Pulmonary Hypertension Quality Enhancement Research Initiative (S-QuERI)
  
  
• Evaluation of the expression profile of inflammation and fibrosis mediators in skin, blood and urine from subjects with diffuse systemic sclerosis (SSc) (CENTOCOR)
  
  
• Genome-Wide Association Study In Systemic Sclerosis (GWAS)
  
  
• Digital Ischemic Lesions in Scleroderma Treated with Oral Treprostinil Diethanolamine: A randomized, double-blind, placebo-controlled, multicenter study (DISTOL-1)
  
  
• Mycophenolate vs. Oral Cyclophosphamide in Scleroderma Interstitial Lung Disease (SLS-II)
  
  
• An Open-Label Study to Evaluate the Safety of Dasatinib in the Treatment of Scleroderma Pulmonary Fibrosis
  
  
• 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
  

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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:
McLauglin, 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.

2. 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:

Currently Enrolling Patients
Informed consent required

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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:
Seibold, James R., MD, University of Michigan, Internal Medicine, Scleroderma Program
Co-Investigators:
Phillips, Kristine, MD,PhD; Department of Internal Medicine, Division of Rheumatology, University of Michigan
Tishkowski, Elena Schiopu, MD; 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.

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.

Study Procedures:

For more information about the study and the study procedures:

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:

Currently Enrolling Patients
Informed consent required

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Study Title:

Consortium of Rheumatology Researchers of North America, Inc. (CORRONA) Data Collection Program
The CORRONA – New York University (NYU) Pharmacogenomics Substudy

IRB Number: HUM00019600

Study Team:

Purpose:

Who can take part in this study?

Study Procedures:

Your physician will be asked to complete forms providing information about you and your condition for inclusion in the CORRONA Database.

You will be asked to provide a one-time sample of blood of about 2 tablespoons.

Study Contact Information:

Study Status:

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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:
Seibold, James, MD; University of Michigan Department of Internal Medicine

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:
Bethany Baker, MSA

Mailing Address:

Telephone: (866) 628-9200

Study Status:

Currently Enrolling Patients

Informed consent required

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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:
Seibold, James, MD; Department of Internal Medicine, University of Michigan (Adult)
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:

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:
Bethany Baker and 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:

Currently Enrolling Patients
Informed consent required

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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

Study Team:

Coordinating Investigator:
Seibold, James R., MD, Department of Internal Medicine, University of Michigan

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:

Who can take part in this study?

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:
Ann J. Impens, PhD

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:

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

Study Team:

Coordinating Investigator:
Steen, Virginia D., MD, Professor of Medicine, Georgetown University School of Medicine
Co-Investigators at the University of Michigan:
Seibold, James R., MD; Department of Internal Medicine, 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:
Ann J. Impens, PhD

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:

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

Study Team:

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

Co-Investigators:
Seibold, James, MD; Department of Internal Medicine, 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:
Ann J. Impens, PhD

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

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
Seibold, James, 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:

Currently Enrolling Patients

Informed consent required

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Study Title:

Scleroderma and Myositis Cohort

Study Team:

Principal Investigator:
Seibold, James, MD; Department of Internal Medicine, University of Michigan

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:
Ann J. Impens, PhD

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

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Study Title:

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

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:
Seibold, James, MD; Department of Internal Medicine, Division of Rheumatology, 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:
Ann J. Impens, Ph.D.

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:

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

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:

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:
Bethany Baker or 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:

This study has been completed

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Study Title:

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

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:
Seibold, James, MD; Department of Internal Medicine, Division of Rheumatology, 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. 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 Rheumatology
1000 Veteran Ave, #32-59 Rehab
Los Angeles, CA 90024

Telephone:  (310) 825-2448

Study Contact Information:

Study Coordinator – University of Michigan Scleroderma Program:
Ann J. Impens, PhD

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:

Pain, Pain Self-efficacy, and Depression in Scleroderma

Study Team:

Principal Investigator:
Seibold, James, MD; 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
Ann J. Impens, PhD; 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:
Ann J. Impens, PhD
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:

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:

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 Status:

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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:

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 Status:

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Study Title:

Fibrocyte Phenotypes as Biomarkers in IPFnet Patients

Study Team:

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?

Study Procedures:

Study Contact Information:

Study Status:

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Study Title:

Novel Biomarkers in Clinical Pulmonary Syndromes

IRB Number: HUM00028972

Study Team:

Purpose:

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:

Study Contact Information:

Study Status:

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Study Title:

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

IRB Number: HUM00013792

Study Team:

Purpose:

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?

Study Procedures:

Study Contact Information:

Study Status:

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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:

Purpose:

Who can take part in this study?

Study Procedures:

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:

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Study Title:

Genome-Wide Association Study in Systemic Sclerosis (GWAS)

IRB Number: HUM00030376

Study Team:

Seibold, James R., MD, University of Michigan, Internal Medicine, Scleroderma Program
Schiopu, Elena MD, University of Michigan, Internal Medicine, Scleroderma Program
Phillips, Kristine MD, PhD, University of Michigan, Internal Medicine, Scleroderma Program Mahmood, Fazle MD, University of Michigan, Internal Medicine, Scleroderma Program

Purpose:

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?

Study Procedures:

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 Status:

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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:
Seibold, James R., MD, University of Michigan, Internal Medicine, Scleroderma Program

Purpose:

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?

Study Procedures:

Study Contact Information:

Study Status:

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Study Title:

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

Study Team:

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

Purpose:

Who can take part in this study?

Study Procedures:

Study Contact Information:

Study Status:

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Study Title:

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

Study Team:

Principal Investigator:
Seibold, James R,, MD, University of Michigan, Internal Medicine, Scleroderma Program

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

Purpose:

Who can take part in this study?

Study Procedures:

Study Contact Information:

Study Status:

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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:

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

Purpose:

Who can take part in this study/Study procedures?

Study Contact Information:

Study Status:

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