Last updated on November 2019

Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease

Brief description of study

This is a multicenter, randomized, open-label study with an active standard-of-care comparator (penicillamine)

Detailed Study Description

This is a multicenter, randomized, open label study with an active standard-of-care comparator.

Stable patients who are already considered to be stable on their standard-of-care penicillamine chelation therapy for at least 1 year will enroll in the study and enter a 12-week Penicillamine Baseline Period comprising of 1 month (4 weeks) run-in period followed by a 2 month (8 weeks) evaluation period. During this time all patients will continue to take their current penicillamine under study conditions. At the end of the Penicillamine Baseline Period, patients who fulfill the protocol definition of being adequately controlled and tolerating penicillamine will be randomized in a 1:1 ratio to receive either TETA 4HCl or to continue to receive penicillamine. There is then a 24-week Post-randomization Phase comprising of a 1 month (4 weeks) run-in period for both treatment arms and a 5 month (20 weeks) evaluation period.

Patients who successfully complete the 24-week Post-randomization Phase of the study will have the opportunity to enter an 18 month (72 weeks) Extension Phase. Initially they continue to receive their allocated TETA 4HCl or penicillamine for a further 24 weeks (i.e., up to Week 60 of the study). Thereafter all patients will receive TETA 4HCl for 48 weeks (i.e., between Week 60 and Week 108). Study clinic visits occur every 6 months (24 weeks) for all patients (i.e., at Weeks 60, 84 and 108).

Clinical Study Identifier: NCT03539952

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