A Phase 3 clinical study to Evaluate the Efficacy and Safety of WTX101 in adult patients who suffer from Wilson Disease.

Phase 1

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

• Registration Number: EUCTR2017-004135-36-HU
• Lead Sponsor: Wilson Therapeutics AB

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2018-02-07
• Last Update Posted: 26 February 2018

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 102

Inclusion Criteria

- Willing and able to give informed consent for participation in the study;
- Male or female subjects, aged 18 years or older as of signing the informed consent form (ICF);
- Able to understand and willing to comply with study procedures, restrictions, and requirements, as judged by the Investigator;
- Established diagnosis of WD by Leipzig-Score = 4 documented by testing as outlined in the 2012 European Association for the Study of Liver WD Clinical Practice Guidelines;
- Treatment for >28 days for WD with chelation therapy (ie, penicillamine, trientine hydrochloride), Zn therapy, or a combination of a chelator and Zn; OR Treatment naïve or treatment for = 28 days for WD with chelation therapy (ie, penicillamine, trientine hydrochloride), Zn therapy, or a combination of a chelator and Zn;
- Willing to undergo = 48-hour washout from current WD treatment;
- Willing to avoid use of vitamins and/or minerals containing Cu, Zn, or Mo throughout the study duration
- Willing to avoid intake of foods and drinks with high contents of Cu throughout the study duration;
please see the study protocol for details
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 102
F.1.3 Elderly (>=65 years)
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Decompensated hepatic cirrhosis;
- MELD score 13;
- Participation in a clinical study of an experimental or unapproved/unlicensed therapy at the same time or within the 4 weeks prior to this Screening Visit;
- Severe anaemia with a haemoglobin 9 g/dL;
- Pregnant (or women who are planning to become pregnant) or lactating women;
- Major systemic disease or other illness that would, in the opinion of the Investigator, compromise subject safety or interfere with the collection or interpretation of study results;
- Modified Nazer score >7
- Alanine aminotransferase >2 × ULN for subjects treated for >28 days with WD therapy (Cohort 1);
- Alanine aminotransferase Alanine aminotransferase > 5 × ULN for treatment naïve subjects or subjects who have been treated for = 28 days (Cohort 2);
please see study protocol for more details

Study & Design

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