Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease
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).
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease|
- ALT [ Time Frame: -3,-2,-1, baseline, 1, 2, 3, 6, 9, 12 months ] [ Designated as safety issue: Yes ]Alanine transaminase
- Ceruloplasmin [ Time Frame: -3,-2,-1, baseline, 1, 2, 3, 6, 9, 12 months ] [ Designated as safety issue: No ]Ceruloplasmin is the major copper-carrying protein in the blood.
- Clinical status [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]The primary objective of the statistical analysis will be to compare parameters pertaining to adverse events (AEs) and clinical status for subjects receiving "current standard therapy" vs. Trientine therapy.
- Other LFTs [ Time Frame: -3,-2,-1, baseline, 1, 2, 3, 6, 9, 12 months ] [ Designated as safety issue: Yes ]Liver function tests
|Study Start Date:||January 2010|
|Study Completion Date:||July 2011|
|Primary Completion Date:||July 2011 (Final data collection date for primary outcome measure)|
Experimental: Once a day Trientine
Patients receive once a day trientine
Drug: Once a day Trientine
Trientine at a dosage of ~15 mg/kg rounded upwards to the nearest 250 or 300 mg in a single daily dosage. The entire daily dosage will be taken at once in the AM an hour before any meal. Duration of the study is 1 year.
Other Name: Syprine
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|United States, Connecticut|
|New Haven, Connecticut, United States, 06520|
|Principal Investigator:||Michael Schilsky, MD||Yale University|