A clinical trial in adult Wilson Disease Patients to evaluate efficacy and safety of WTX101 following administration for 24 weeks and an extension phase of 36 months.

Phase 1

Active, not recruiting

• Conditions: Wilson Disease; MedDRA version: 20.0 Level: LLT Classification code 10047988 Term: Wilson's disease System Organ Class: 100000004850; Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

• Registration Number: EUCTR2014-001703-41-AT
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-06-24
• Last Update Posted: 30 April 2019

Recruitment & Eligibility

• Status: Not Recruiting
• Sex: Not specified
• Target Recruitment: 30

Inclusion Criteria

- Willing and able to give informed consent for participation in the study.
- Male or female patients, aged 18 years or older as of signing the ICF.
- Able to understand and willing to comply with study procedures, restrictions and requirements, as judged by the Investigator.
- Newly established diagnosis of Wilson Disease by Leipzig-Score = 4 (Ferenci et al 2003) documented by testing as outlined in 2012 EASL WD Clinical Practice Guidelines.
- NCC levels above or within the normal reference range (0,8- 2.3 µM).
- Willing to undergo 48 hour washout from current Wilson Disease treatment.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 29
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

- Treatment for greater than 24 months for Wilson Disease with chelation therapy (i.e. penicillamine, trientine hydrochloride) or zinc therapy.
- Decompensated hepatic cirrhosis.
- Model for End-Stage Liver Disease (MELD) score > 11.
- Modified Nazer score > 6 (Dhawan et al., 2005).
- GI bleed within past 6 months.
- ALT > 5x upper limit of normal (ULN).
- Hepatic fibrosis on either liver biopsy or fibroscan in subjects with ALT or AST > 2x upper limit of normal (ULN).
- Marked neurological disease requiring either nasogastric (NG) feeding or intensive in-patient medical care.
- Severe anaemia with a haemoglobin < 9 g/dL.

Study & Design

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