Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease
Status:
Completed
Trial end date:
2011-07-01
Target enrollment:
Participant gender:
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
Details
Lead Sponsor:
Yale University
Collaborators:
Bausch Health Americas, Inc. Valeant Pharmaceuticals International, Inc.