Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02426905
• Date of registration: 18/09/2014
• Prospective Registration: Yes
• Primary sponsor: Univar BV
• Public title: Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
• Scientific title: Multicentre, Retrospective and Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
• Date of first enrolment: January 2016
• Target sample size: 90
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT02426905
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 4

Countries of recruitment

Germany, Greece, Italy, United Kingdom

Contacts

• Name: Karl-Heinz Weiss, MD
• Affiliation: Universitätsklinik Heidelberg

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients aged 1 year to 90 years of age.

- Physician established diagnosis of Wilson disease based on a Ferenci score > 3.

- Documented treatment with d-Penicillamine, withdrawal of treatment with d-
Penicillamine, followed by treatment with trientine for at least 6 months at date of
informed consent.

- Able/willing to provide written informed consent.

- For enrolment in the prospective part, enrolment in the retrospective part of the
study is required.

Exclusion Criteria:

- Incomplete history of medication use for trientine from initial diagnosis to latest
follow up.

- Unavailable outcome data for hepatic and neurological course of disease at assessment
time points.

- Patients with acute liver failure and fulminant hepatic disease with fatal outcome.

- Hypersensitivity to trientine and severe anaemia.



Age minimum: 1 Year
Age maximum: 90 Years
Gender: All

Health Condition(s) or Problem(s) studied

Wilson Disease

Intervention(s)

Drug: trientine dihydrochloride

Primary Outcome(s)

Clinical outcome specific to the retrospective part of the study [Time Frame: 48 months]

Clinical outcome specific to the prospective part of the study [Time Frame: 12 months]

Secondary Outcome(s)

Quality of Life Endpoints for the Prospective Part of the Study [Time Frame: 12 months]

Safety Endpoint Applicable to both the Retrospective and Prospective Parts of the Study [Time Frame: Up to 60 months]

Secondary ID(s)

UNV-TRI-002

Source(s) of Monetary Support

Please refer to primary and secondary sponsors