Wilson Disease
Conditions
Sponsors
Wilson Therapeutics AB
Eligibility
Sex/Gender
All
Inclusion criteria
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) yes F.1.3.1 Number of subjects for this age range 3
Exclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary end point(s): The primary efficacy assessment will be control of free Cu, measured as the percent change from baseline (Day 1) to 48 weeks in NCC levels. For WTX101-treated subjects, the NCC level will be corrected for the amount of Cu bound to the WTX101 TPC.;Timepoint(s) of evaluation of this end point: 48 weeks;Main Objective: The primary objective is to evaluate the efficacy of WTX101 administered for 48 weeks, compared to standard of care (SoC), on copper (Cu) control in WD subjects aged 18 and older. Copper control will be assessed in terms of the percentage change from baseline (Day 1) to 48 weeks in non-ceruloplasmin-bound copper (NCC) levels. For WTX101-treated subjects, the NCC level will be corrected for the amount of Cu bound to the WTX101 tripartite complex (TPC) (NCCcorrected).; Secondary Objective: The secondary objectives are to: - Establish the safety and tolerability of individualised dosing of WTX101; - Evaluate the effects of WTX101 on hepatic status assessed by the Model for End-Stage Liver Disease (MELD) - Evaluate the effects of WTX101 on disability assessed by the Unified WD Rating Scale (UWDRS) Part II - Evaluate the effects of WTX101 on neurological status assessed by the UWDRS Part III please see study protocol for more details. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary endpoints include the following: a. Change from baseline in hepatic status at 48 weeks assessed by MELD score; b. Change from baseline to 48 weeks in UWDRS Part II score; c. Change from baseline to 48 weeks in UWDRS Part III score; d. Change from baseline to 48 weeks in the CGI-I and CGI-S; and e. NCC responder rate at 48 weeks. ;Timepoint(s) of evaluation of this end point: 48 weeks | — |
Countries
Austria, Belgium, Czech Republic, Denmark, France, Germany, Hungary, Israel, Italy, Netherlands, Poland, Portugal, Spain, Sweden, United Kingdom, United States
Contacts
Public ContactSusan Flint
Wilson Therapeutics AB
Outcome results
None listed