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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: 12 February 2018
Main ID:  EUCTR2016-003876-29-IT
Date of registration: 06/02/2018
Prospective Registration: No
Primary sponsor: GMP ORPHAN SA
Public title: Chelate Study: Trientine for the treatment of Wilson's disease
Scientific title: CHELATE STUDY: Trientine tetrahydrochloride (TETA 4HCl) for the treatment of Wilson’s disease - Chelate Study: Trientine for the treatment of Wilson's disease
Date of first enrolment: 13/12/2017
Target sample size: 55
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-003876-29
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: yes
Randomised: yes
Open: yes
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: yes
Placebo: no
Other: no
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 Brazil Denmark France Germany Italy Micronesia, Federated States of Poland
United Kingdom United States
Contacts
Name: Clinical Trials Information   
Address:  Pépinière Paris Santé Cochin, 27-29 rue Faubourg 75014 Saint-Jacques - Paris France
Telephone: 0033142498264
Email: naseem@gmp-o.com
Affiliation:  gmp-orphan SA
Name: Clinical Trials Information   
Address:  Pépinière Paris Santé Cochin, 27-29 rue Faubourg 75014 Saint-Jacques - Paris France
Telephone: 0033142498264
Email: naseem@gmp-o.com
Affiliation:  gmp-orphan SA
Key inclusion & exclusion criteria
Inclusion criteria:
1. Patient is able to provide and has provided written informed consent has been obtained
2. Written documentation has been obtained in accordance with the relevant country and local privacy requirements, where applicable, including for EU sites, Data Protection Consent
3. Male or female, aged = 18 and = 75 years old at time of consent
4. Patient has a diagnosis of Wilson’s disease, as defined by a prior or current Leipzig score of = 4
5. Patient’s Wilson’s disease is clinically stable, in the opinion of the investigator, and being treated with penicillamine for at least 1 year (52 weeks) prior to the screening visit
6. Patient is on a stable dose and regimen of penicillamine for at least 4 months (16 weeks) prior to the screening visit (other prescribed treatments for Wilson’s disease not permitted during this period)
7. No anticipated need that patient will require additional pharmacological therapies other than study medication, including prescribed zinc therapy, for the management of copper levels during the study
8. Patient must be willing to maintain stable diet throughout the study, and avoid foods with high copper content, including the screening period
9. Patient considered suitable to receive therapy with both TETA 4HCl and penicillamine administered twice a day
10. Negative central laboratory tests for HIV and viral hepatitis (results will be available after start of run-in period)
11. For female patients of childbearing potential, negative urine pregnancy test (at screening visit and prior to randomization)
12. For females of childbearing potential, use of a reliable form of contraceptive
13. Patient is considered as able to complete study requirements and attend the study visits, in he opinion of the investigator
Are the trial subjects under 18? no
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 50
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion criteria:
1. Patient is in ‘de-coppering’ phase of treatment for Wilson’s disease, in the opinion of the investigator
2. Patient evidence of uncontrolled liver disease, including but not limited to:
a. Modified Nazer score of = 4 (result may not be available until after start of run-in period on lab results*)
b. decompensated cirrhosis
c. acute hemolytic anemia
d. acute hepatitis
e. hepatic malignancy
f. evidence of acute liver failure
3. Cause of patient’s liver disease is due to another condition, in the investigator’s opinion
4. Patient has severe anemia defined as hemoglobin of = 9 g/dL (result will be available after start of run-in period*)
5. Patient has experienced a gastrointestinal bleed within 6 months (24 weeks) prior to screening visit
6. Patient has moderate or severe renal impairment defined as creatinine clearance of = 30 mL/min (result will may not be available until after start of run-in period*), or patient has nephritis or nephrotic syndrome, in the opinion of the investigator
7. Patient has neurological disease that prevents swallowing of study medication (e.g. requires a nasogastric feeding tube) or requires intensive in-patient medical care
8. Patient is currently taking medication containing trientine for management of Wilson’s disease or has taken it within 4 months (16 weeks) of screening visit
9. Patient is currently receiving prescribed zinc therapy for management of Wilson’s disease or has taken it within 4 months (16 weeks) of screening visit
10. Patient is taking any of the following concomitant therapies: gold therapy, antimalarial therapy, cytotoxic drugs, oxyphenbutazone, phenyl butazone
11. Patient has a known intolerance, allergy or sensitivity to penicillamine (that is uncontrolled) or to TETA 4HCl, including any component of the study medication
12. For female patients of childbearing potential, planning a pregnancy during study period or currently nursing
13. For female patients of childbearing potential, unable or unwilling to use a reliable form of contraceptive throughout the study
14. Patient is currently participating in another therapeutic study, or has previously participated in a therapeutic study within 30 days of screening visit (or longer, if local requirements specify this)
15. Patient has any condition or in any situation which, in the investigator’s opinion, puts the patient at significant risk, could confound study results, or may interfere significantly with the patient’s participation in the study

*Samples for screening/enrolment labs can be taken within ± 7 days of visit. In the event that lab value is above the specified threshold, it can be repeated. If repeat value within specified range, patient can continue in the study, otherwise the patient will be a screen failure. Note: if it is not possible to obtain a repeat value prior to the Week 4 clinic visit, then the Week 4 value will serve as the repeat lab value(s).


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Wilson's disease (WD) is a life-threatening inborn error of copper metabolism leading to an excessive copper accumulation, mainly in the liver or brain, causing hepatic and neurologic severe symptoms.
MedDRA version: 20.0 Level: LLT Classification code 10047988 Term: Wilson's disease System Organ Class: 100000004850
Intervention(s)

Product Name: TETA.4HCI
Product Code: TETA.4HCI
Pharmaceutical Form: Tablet
INN or Proposed INN: Trientine tetrahydrochloride
CAS Number: 4961-40-4
Current Sponsor code: TETA4HCI
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 300-

Trade Name: PEMINE - 150 MG CAPSULE RIGIDE 50 CAPSULE
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: D-penicillamine
CAS Number: 52-67-5
Current Sponsor code: D-penicillamine
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-

Primary Outcome(s)
Main Objective: To evaluate the efficacy of TETA 4HCl compared to penicillamine.
Primary end point(s): To evaluate the efficacy of TETA 4HCl compared to penicillamine:
The primary hypothesis is that the efficacy of TETA 4HCl, as assessed by serum NCC levels, is not inferior to the efficacy of penicillamine, at 36 weeks of treatment.
The secondary efficacy measures are:
- 24-hour urinary copper excretion
- CGIC score
Other efficacy measures are:
- Unified Wilson’s Disease Rating Scale (UWDRS), neurological scale
- Serum total copper and serum ceruloplasmin (used to derive non-ceruloplasmin bound copper).
Significance tests will be performed at the a = 0.05 significance level for two-tailed tests at a= 0.025 for one tailed test
Secondary Objective: The safety of TETA 4HCl compared to penicillamine will also be evaluated.
Timepoint(s) of evaluation of this end point: Measured at Screening/Enrolment, Weeks 4, 8, 12, 16, 20, 24, 28, 32 and 36
Secondary Outcome(s)
Secondary end point(s): The safety of TETA 4HCl compared to penicillamine will also be evaluated
Safety will be evaluated by assessing treatment emergent AEs (TEAEs), and other safety measures (vital signs, hematology, biochemistry, coagulation analysis, urinalysis, modified Nazer score, and urine pregnancy test).
Timepoint(s) of evaluation of this end point: Measured at Screening, Weeks 4, 8, 12, 16, 20, 24, 28, 32 and 36
Secondary ID(s)
2016-003876-29-DK
GMPO-131-002
ISRCTN12345678
NCT12345678
Source(s) of Monetary Support
gmp-orphan SA
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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