Overview

Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease

Status:
Completed
Trial end date:
2011-07-01
Target enrollment:
0
Participant gender:
All
Summary
Hypothesis: The investigators postulate that patients with Wilson disease who are asymptomatic or who have been effectively treated for their symptoms and are in a maintenance phase therapy can be safely and effectively treated with a single daily dosage of the chelating agent trientine. Specific Aims: To demonstrate that a single daily treatment with trientine is as effective or better than a patient's current maintenance therapy. This will be accomplished by performance of a case control prospective study of patients on their prior therapy, and during a period of treatment with a single weight based dose regimen of trientine. The primary endpoint for this study is the demonstration of equivalence to a patient's prior therapy. Secondary endpoints include: 1) demonstration of stability or improvement in parameters of copper metabolism; 2) improvement in adherence to therapy; 3) no progression of liver disease (defined by changes in synthetic function, albumin and INR, and fibrosis by Fibrotest).
Phase:
N/A
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Yale University
Collaborators:
Bausch Health Americas, Inc.
Valeant Pharmaceuticals International, Inc.
Treatments:
Trientine
Criteria
Inclusion Criteria:

Established diagnosis of Wilson Disease:

- That have been treated for at least 1 year

- Compensated liver disease and/or stable neurological or psychiatric disease.

- Normal or minimal elevation of serum ALT (<2 times upper limit of normal)

- Non-ceruloplasmin copper <25 mcg/dl

Exclusion Criteria:

- Wilson disease diagnosis not well established Wilson disease treated for less than one
year Decompensated liver disease (ascites, jaundice, encephalopathy, bleeding due to
portal hypertension) Liver disease with elevations of ALT > 2 times upper limit of
normal A female who is pregnant or intends to become pregnant