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: 20.0Level: PTClassification code 10019819Term: Hepato-lenticular degenerationSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2013-003564-31-IT
• Lead Sponsor: IVAR LTD

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2018-04-26
• Last Update Posted: 30 April 2018

Recruitment & Eligibility

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

Inclusion Criteria

1. Patients aged 1 year to 90 years of age
2. Physician-established diagnosis of Wilson disease based on a Ferenci score = 3
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
4. Able/willing to provide written informed consent.
Are the trial subjects under 18? yes
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 23
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

1. Incomplete history of medication use for trientine from initial diagnosis to last follow-up
2. Unavailable outcome data for hepatic and neurological course of disease at all assessment time points
3. Patients with acute liver failure and fulminant hepatic disease with fatal outcome.

Study & Design

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