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

Active, not recruiting

• 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-PL
• Lead Sponsor: nivar Solutions, B.V.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-06-04
• Last Update Posted: 24 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. Model for End-Stage Liver Disease (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