Retrospective Study of Trientine in Wilson's Disease Subjects
Research type
Research Study
Full title
Multicentre, Retrospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn from Therapy With d-Penicillamine
IRAS ID
148984
Contact name
Anil Dhawan
Contact email
Sponsor organisation
Univar B.V.
Clinicaltrials.gov Identifier
EUDRACT number:, 2013-003564-31
Research summary
This is a retrospective, multicentre study of the efficacy, safety and tolerance of Trientine in Wilson’s disease patients who are withdrawn from therapy with d-Penicillamine.
This study will investigate the safety, efficacy and tolerance of trientine retrospectively in patients who have received treatment with trientine for at least 6 months after withdrawal of treatment with d-Penicillamine. Existing data from patient's files will be collected and no clinical procedures and/or examinations are specifically planned as part of this study. We will examine the influence of treatment with Trientine on the copper deposits and progress of neurological and liver disease. In addition, all illnesses that occurred during the treatment with Trientine will be evaluated. We have planned to include 90 patients aged 1 year to 90 years in this study in all participanting countries.
In the UK, Trientine is approved for the treatment of Wilson's disease in patients who do not tolerate d-Penicillamine. However, Trientine is made available to patients in the rest of the European Union only on a named prescription basis. To enable easy access of Trientine to patients in the European Union it needs to obtain a marketing authorisation from the European Medicines Agency (EMA) in those countries. This current study aims to collect data about the efficacy and safety of Trientine to support its approval across the European Union.REC name
West of Scotland REC 5
REC reference
14/WS/1008
Date of REC Opinion
17 Jun 2014
REC opinion
Further Information Favourable Opinion