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: 2 June 2020
Main ID:  EUCTR2017-004135-36-AT
Date of registration: 17/01/2018
Prospective Registration: Yes
Primary sponsor: Alexion Pharmaceuticals, Inc.
Public title: A Phase 3 clinical study to Evaluate the Efficacy and Safety of WTX101 in adult and adolescent patients who suffer from Wilson Disease.
Scientific title: A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months
Date of first enrolment: 27/02/2018
Target sample size: 180
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
Australia Austria Belgium Brazil Canada Colombia Croatia Czech Republic
Denmark France Germany Greece Hong Kong Hungary Israel Japan
Korea, Republic of Netherlands New Zealand Poland Portugal Russian Federation Serbia Singapore
Spain Sweden Taiwan Turkey United Kingdom United States
Contacts
Name: European Clinical Trial Information   
Address:  1-15 avenue Edouard Belin 92500 Rueil-Malmaison France
Telephone: 33147100615
Email: clinicaltrials.eu@alexion.com
Affiliation:  Alexion Europe SAS
Name: European Clinical Trial Information   
Address:  1-15 avenue Edouard Belin 92500 Rueil-Malmaison France
Telephone: 33147100615
Email: clinicaltrials.eu@alexion.com
Affiliation:  Alexion Europe SAS
Key inclusion & exclusion criteria
Inclusion criteria:
- 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;
-12 years of age or older at time of informed consent/assent;
- Willing and able to give written informed consent and comply with the study visit schedule. For patients < 18 years of age patient`s legal guardian must be willing and able to give written informed consent.
- Able to understand and willing to comply with study procedures, restrictions, and requirements, as judged by the Investigator.;
- Willing to withhold treatment with SoC for > 48 hours immediately Prior to first study assessment on Day 1;
- Adequate venous access to allow collection of required blood samples;
- Willing to avoid use of vitamins and/or minerals containing Cu, Zn, or molybdenum (Mo) throughout the study duration;
- Willing to avoid intake of foods and drinks with high contents of Cu throughout the study duration;
- Female patients of childbearing potential must be willing to follow guidance for highly effective contraception;

please see the study protocol for details
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 160
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

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
- Hemoglobine < 9 g/dL
- 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);
- Patients with end-stage renal disease on dialysis (CKD 5) or creatinine clearance < 30 mL/min

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: ALXN1840
Pharmaceutical Form: Tablet
INN or Proposed INN: Not applied
CAS Number: 649749-10-0
Current Sponsor code: ALXN1840
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

Pharmaceutical Form: Capsule
INN or Proposed INN: ZINC ACETATE DIHYDRATE
CAS Number: 5970-45-6

Primary Outcome(s)
Main Objective: The primary objective is to evaluate the efficacy of ALXN1840 administered for 48 weeks, compared to standard of care (SoC), on copper (Cu) control in WD subjects aged 12 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 ALXN1840-treated subjects, the NCC level will be corrected for the amount of Cu bound to the ALXN1840 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 ALXN1840-treated subjects, the NCC level will be corrected for the amount of Cu bound to the ALXN1840 TPC.
Secondary Objective: The secondary objectives are to:
- Establish the safety and tolerability of individualised dosing of ALXN1840;
- Evaluate the effects of ALXN1840 on hepatic status assessed by the Model for End-Stage Liver Disease (MELD)
- Evaluate the effects of ALXN1840 on disability assessed by the Unified WD Rating Scale (UWDRS) Part II
- Evaluate the effects of ALXN1840 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)
2017-004135-36-GB
WTX101-301
Source(s) of Monetary Support
Alexion Pharmaceuticals, Inc
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date: 18/02/2018
Contact:
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