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.
- Conditions
- Wilson DiseaseMedDRA version: 20.0Level: LLTClassification code 10047988Term: Wilson's diseaseSystem Organ Class: 100000004850Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
- Registration Number
- EUCTR2014-001703-41-PL
- Lead Sponsor
- Wilson Therapeutics AB
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 30
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 within or above 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
Treatment for greater than 24 months days 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
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
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