Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.
ConditionWilson's Disease
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age8+
SponsorOrphalan
About this trial
The goal of this clinical trial is to learn if a new trientine tetrahydrochloride (TETA 4HCl) formulation administered once a day compared to d-Penicillamine (DPA) as a first line treatment for people living with Wilson's disease (WD) is effective and safe. The study is enrolling children aged 8 years and older weighing at least 55 lb (25 kg) and adults with a recent diagnosis of WD. People recently diagnosed with WD, may be eligible for the study if they have either not started copper chelating treatment (such as DPA or trientine) or have been taking zinc salts for less than 28 days. Participants will be randomly allocated (like tossing a coin) to receive either DPA or TETA 4HCL for 48 weeks. During this time period participants will have up to 12 visits for health checks and assessments including blood and urine testing. In addition, at some visits participants may be asked to complete questionnaires on treatment satisfaction, and overall well-being.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Participant is aged 8 years or older and is willing and able to give informed consent for participation in the study, or by a parent/legally authorized representative (LAR) and assent obtained (in accordance with local regulations) for any participant less than the age of majority (e.g. less than 18 years of age, depending on local requirements).
Participant has a body weight of at least 25 kg at screening.
Participant has a diagnosis of WD, as defined by a Leipzig score of greater than or equal to 4. Note that historical test results can be used for the diagnosis.
Received no prior prescribed therapy [a] for the treatment of WD (treatment-naïve), or
Disqualifiers
Any known contraindications for treatment with DPA.
Any known contraindications for treatment with TETA 4HCl.
Unable to swallow tablets/capsules independently or considered high risk for aspiration, in the opinion of the Investigator
Acute liver failure (ALF) or at high risk of ALF, in the opinion of the Investigator.
Trial design
Design model
Parallel
Treatments tested in this trial
TETA 4HCl formulation
DrugThe new formulation of TETA 4HCl will be administered once a day. Each film-coated tablet is scored to enable halving, if required. Randomized participants are planned to receive TETA 4HCl for the 48-week post-randomization period.
D-Penicillamine
DrugStandard of care DPA is to be used, per the sites and treating physician's usual practice. To be administered in accordance with the product labelling and/or the institutions treatment practice guidelines. Randomised participants are planned to receive DPA for the 48-week post-randomization period.
Treatment groups
38 Participants
are divided into 2 treatment groupsGroup A: TETA 4HCl formulationExperimental treatment 1 intervention
Group B: Standard of care d-Penicillamine (DPA)Active comparator 1 intervention
Trial outcomes
Primary outcomes
1
Absolute value of serum NCC at Week 48 assessed using the NCC-speciation assay (serum NCC-Sp)
Time frame
Week 48Secondary outcomes
1
Serum NCC-Sp
Change from Baseline, within specified thresholds \[\>200, ≤ 200, ≤ 150, ≤ 100, ≤ 80, ≤ 50 μg/L and 50 to 80 μg/L\]
Time frame
Week 482
24-hour UCE
Absolute value, change from Baseline, within specified thresholds \[\< 100, \<200, \> 500, 150 to 500, and 200 to 500 μg/24h\]
Time frame
Week 483
Investigator's assessment of signs and symptoms
Assessment of of the signs and symptoms of WD using GAS for WD, comprising a 4-item Global Disability (Tier 1) and 10-item Neurological Assessment (Tier 2). Total score, Tier 1 score, Tier 2 score, each item score.
Time frame
Week 484
Clinical Global Impression of Change
Investigator rating of change in the patients WD relating to the prior clinic visit on a 7-point scale by responding to the following statement "Please rate the change in the overall severity of the patients Wilson's disease compared to the previous clinic visit".
Time frame
Week 48 Source ClinicalTrials.gov