Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
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Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
A study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them for a further 12 months.
A retrospective study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them prospectively for a further 12 months.
Clinical outcome specific to the retrospective part of the study
Safety Endpoint Applicable to both the Retrospective and Prospective Parts of the Study
Study Arms / Comparison Groups
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Primary Completion Date
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.
1 Year - 90 Years
Accepts Healthy Volunteers
Karl-Heinz Weiss, MD, ,
Karl-Heinz Weiss, MD, Principal Investigator, Universitätsklinik Heidelberg