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.

Phase 1

• Conditions: Wilson disease, also known as hepatolenticular degeneration, a rare automsomal, recessively inherited disorder which results in chronic copper intoxication.; MedDRA version: 19.1Level: LLTClassification code 10047988Term: Wilson's diseaseSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2013-003564-31-DE
• Lead Sponsor: nivar B.V.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-10-22
• Last Update Posted: 27 August 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 90

Inclusion Criteria

- Patients aged 1 year to 90 years of age at initiation of treatment with trientine.
- Physician established diagnosis of Wilson disease based on a Ferenci score > 3 at the date of diagnosis.
- 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.
- On-going treatment with trientine.
- Negative pregnancy test.
- Able/willing to provide written informed consent.
Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 45
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 40

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 initiation of treatment with trientine and at least 1 time point post-initiation of treatment with trientine.

For enrolment in the prospective part:
- Hypersensitivity to trientine
- Severe anaemia.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified