N/A
Completed N=8
Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease
Source: ClinicalTrials.gov NCT01472874 ↗Enrolled (actual)
8
Serious AEs
0.0%
Results posted
May 2014
Primary outcomePrimary: ALT — 41.38 U/L
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).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY ALT |
50.89 | — |
| PRIMARY Cu Serum |
0.52 | — |
| SECONDARY INR |
1.05 | — |
| SECONDARY Albumin |
0.52 | — |
| SECONDARY Cu Urine |
313.4 | — |
| SECONDARY Zn Urine |
2214 | — |
Eligibility 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 A female who is pregnant or intends to become pregnant
Data sourced from ClinicalTrials.gov (NCT01472874). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.