World Health Organization site
Skip Navigation Links

Main
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 26 February 2018
Main ID:  EUCTR2017-004135-36-HU
Date of registration: 07/02/2018
Prospective Registration: Yes
Primary sponsor: Wilson Therapeutics AB
Public title: A Phase 3 clinical study to Evaluate the Efficacy and Safety of WTX101 in adult patients who suffer from Wilson Disease.
Scientific title: A Phase 3, Randomised, Rater-Blinded, Multi-Centre Study to Evaluate the Efficacy and Safety of WTX101 Administered for 48 Weeks Versus Standard of Care in Wilson Disease Subjects Aged 18 and Older with an Extension Phase of up to 60 Months
Date of first enrolment: 12/02/2018
Target sample size: 102
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-004135-36
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: yes
Randomised: yes
Open: no
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: yes
Other trial design description: Rater Blinded
If controlled, specify comparator, Other Medicinial Product: yes
Placebo: no
Other: yes
Other specify the comparator: Penicillamine, Trientine dihydrochloride and Zinc
Number of treatment arms in the trial: 2
 
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no
Countries of recruitment
Austria Czech Republic France Germany Hungary Israel Italy Poland
Spain United Kingdom United States
Contacts
Name: Susan Flint   
Address:  1500 District Ave MA 01801 Burlington United States
Telephone: +1617 5133787
Email: susan.flint@wilsontx.eu
Affiliation:  Wilson Therapeutics AB
Name: Susan Flint   
Address:  1500 District Ave MA 01801 Burlington United States
Telephone: +1617 5133787
Email: susan.flint@wilsontx.eu
Affiliation:  Wilson Therapeutics AB
Key inclusion & exclusion 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)
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


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Wilson Disease
MedDRA version: 20.0 Level: LLT Classification code 10047988 Term: Wilson's disease System Organ Class: 100000004850
Intervention(s)

Product Name: WTX101
Product Code: WTX101
Pharmaceutical Form: Tablet
INN or Proposed INN: Not applied
CAS Number: 649749-10-0
Current Sponsor code: WTX101
Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 15-

Pharmaceutical Form: Tablet
INN or Proposed INN: PENICILLAMINE
CAS Number: 52-67-5

Pharmaceutical Form: Capsule
INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE
CAS Number: 38260-01-4
Concentration unit: g gram(s)
Concentration type: range
Concentration number: 1.2-2.4-(4-8 capsules)

Pharmaceutical Form: Capsule
INN or Proposed INN: ZINC ACETATE DIHYDRATE
CAS Number: 5970-45-6
Concentration unit: mg milligram(s)
Concentration type: up to
Concentration number: 150-3 times daily

Primary Outcome(s)
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).
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.
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.
Timepoint(s) of evaluation of this end point: 48 weeks
Secondary Outcome(s)
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
Secondary ID(s)
WTX101-301
Source(s) of Monetary Support
Wilson Therapeutics AB
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
Disclaimer: Trials posted on this search portal are not endorsed by WHO, but are provided as a service to our users. In no event shall the World Health Organization be liable for any damages arising from the use of the information linked to in this section. None of the information obtained through use of the search portal should in any way be used in clinical care without consulting a physician or licensed health professional. WHO is not responsible for the accuracy, completeness and/or use made of the content displayed for any trial record.
Copyright - World Health Organization - Version 3.6 - Version history