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Randomized Double Blind Study for Newly
Diagnosed Wilson's Disease Patients Presenting with
Liver Disease Clinical Trial
The University of Michigan, which has been designated a Wilson's Disease Center of Excellence by the Wilson's Disease Association, is conducting a randomized double blind study to compare efficacy and toxicity of three anticopper drugs, penicillamine, trientine, and tetrathiomolybdate, for the initial treatment of Wilson's disease patients presenting with liver disease.
Objectives
To compare rate and degree of recovery of liver function, and to compare side effects.
Eligibility Criteria
- Patients (adults and children) must be untreated or not treated for longer than four weeks with an anticopper drug.
- Patients must have at least one of the following laboratory abnormalities: serum albumin, 2.5 g/dl or less; serum bilirubin 2.0 mg/dl or more; prolongation of prothrombin time, 4 seconds or more; an INR of 1.3 or more; or AST/ALT greater than two times the upper limit of normal.
Exclusion Criteria
- Patients with moderate degrees of hepatic decompensation will be accepted, although not those likely to be in need of immediate hepatic transplantation. Patients with concomitant neurological or psychiatric symptoms from Wilson's disease are excluded from this protocol.
The treatment period is 24 weeks, the first 6 weeks of which are spent in the General Clinical Research Center of the University of Michigan Hospital, with free medical care and hospitalization provided to the extent required for Wilson's disease. The next 18 weeks involves home treatment, with the appropriate anticopper medication provided. It will be necessary to have blood tests every 2 weeks during the 18 week period at home with the results sent to us. The blood tests involve blood counts and liver function tests, readily available anywhere. Patients will be followed with the referring physician, as desired. Patients will be responsible for travel costs to Ann Arbor and for blood tests during the last 18 weeks.
Contact
Fred Askari, M.D., Ph.D.
Associate Professor, Hepatology
University of Michigan
Telephone: (734) 647-2964
Facsimile: (734) 763-2535
E-mail: faskari@umich.edu
Randomized, Double Blind Treatment Trial for Newly Diagnosed Wilson's Disease Presenting with Neurologic Symptoms
The University of Michigan is conducting a randomized
double blind trial of two dose regimens of tetrathiomolybdate
for the initial treatment of Wilson's disease patients
presenting with the neurological symptoms of Wilson's
disease.
Objectives
- To improve initial therapy, avoid the neurological worsening that often occurs with penicillamine therapy, and minimize side effects.
Eligibility
- Patients must be untreated or not treated for longer than three weeks with an anticopper agent. If workup is incomplete or diagnosis uncertain, we will finish diagnostic workup in those cases where Wilson's disease is likely. Patients of all ages and ethnic groups, and both sexes, are most welcome in this study.
The initial treatment period is 16 weeks, the first 6 weeks of which are spent in the General Clinical Research Center of the University of Michigan Hospital, with free medical care and hospitalization provided, to the extent required for Wilson's disease. The next 10 weeks involves home treatment, with the tetrathiomolybdate medication provided. It will be necessary to have blood tests every two weeks during this 10-week period with the results sent to us. The blood tests involve blood counts and liver function tests, readily available everywhere. We will help arrange subsequent care and will wish to follow-up and help monitor the patients on a regular basis over the following 2-3 year period. After initial treatment, patients will be referred back to the referring physician, if desired.
Contact
Fred Askari, M.D., Ph.D.
Associate Professor, Hepatology
University of Michigan
Telephone: (734) 647-2964
Facsimile: (734) 763-2535
E-mail: faskari@umich.edu
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