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

• 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-DE
• Lead Sponsor: Alexion Pharmaceuticals, INC.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-06-05
• Last Update Posted: 28 February 2019

Recruitment & Eligibility

• Status: ot 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. Liver Transplant 2005).
GI bleed within past 6 months.
ALT > 5x 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