Multicentre, open-label study to investigate the effects Cufence has, the effects the body has on Cufence and the continued safety and efficacy on patients with Wilson Disease

Phase 1

• Conditions: Wilson Disease; MedDRA version: 22.1Level: PTClassification code 10019819Term: Hepato-lenticular degenerationSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

• Registration Number: EUCTR2020-004604-33-DE
• Lead Sponsor: nivar Solutions, B.V.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2020-12-17
• Last Update Posted: 18 June 2024

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

1. Patient (or a representative) must provide written, informed consent before the start of any study procedures.
2. Male and female patient aged = 5 years at time of consent.
3. Diagnosis of WD previously determined by the physician based on a Leipzig score = 4.
4. Patient (= 18 years) has previously been treated with D-penicillamine for WD.
5. Patient (< 18 years) has previously been treated with D-penicillamine or zinc for WD.
6. For female patients of childbearing potential, a negative pregnancy test at the Screening visit and the Baseline visit is required. In addition, a highly effective method (failure
rate <1%) of birth control must be used during the study which includes (but is not limited to) the following:
- vasectomized partner (at least 6 months prior to dosing);
- oral, patch, or injected contraceptives, or vaginal hormonal device (i.e. NuvaRing®), in use for at least 3 consecutive months prior to study dosing and throughout the study duration;
- implanted or intrauterine contraceptives in use for at least 6 consecutive months prior to study dosing and throughout the study duration;
- abstinence (must agree to use a highly effective method if they become sexually active during the study).
7. Patient is considered to be able to complete study requirements and attend the study visits, in the opinion of the investigator.
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 30
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion Criteria

1. Patient has evidence of uncontrolled liver disease, including but not limited to:
a. New Wilson index (Dhawan Index) > 10
b. Alanine aminotransferase (ALT) > 5x upper limit of normal (ULN)
c. Aspartate aminotransferase (AST) > 5x ULN
d. MELD score > 13 (only applicable for patients that are = 12 years of age)
e. Acute liver failure
f. Hepatic malignancy
2. Uncontrolled neurological disease according to the judgement of the physician.
3. Patient has severe anaemia defined as hemoglobin of < 9 g/dL.
4. Patient has a known intolerance, allergy or sensitivity to trientine dihydrochloride, including any component of the study medication.
5. Female patient is pregnant or lactating.
6. Any patients who lack the capacity to consent including the parent(s) of a paediatric patient.

Study & Design

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