TR-004 UNITED Study
Research type
Research Study
Full title
Open label, Multicenter, Prospective Study to Characterize the Pharmacokinetics and Pharmacodynamics of Cufence (Trientine Dihydrochloride) and to Investigate the Efficacy and Safety in Wilson’s Disease Patients
IRAS ID
285582
Contact name
Daphne Van Scheppingen
Contact email
Sponsor organisation
Univar Solution, B.V.
Eudract number
2020-004604-33
Duration of Study in the UK
4 years, 0 months, 1 days
Research summary
Wilson disease (WD) is an inherited disorder that results in the accumulation of excessive copper in the body, particularly in the liver, brain, and eyes. WD may present at any age between 3 and 74 years, but WD is rarely symptomatic before the age of 5. If left untreated, this disease may cause liver (hepatic) disease, central nervous system dysfunction, and death. Cufence (trientine dihydrochloride) has been approved in the EU is for the treatment of WD in patients intolerant to the first line of treatment D-penicillamine in adults, adolescents and children aged 5 years or older. However, the optimal dose for each patients based on their response is not known. Furthermore, there are no clear guidelines for assessing the success of the therapy in children or adolescents. The aim of this clinical study is to obtain a better understanding about the way the body absorbs, distributes, and gets rid of Cufence in patients that are suffering from WD, also known as pharmacokinetics (PK). The study is also investigating the effects that Cufence has on the body, i.e. how long the effect of Cufence lasts, what dose works best and what side effects it might cause. This is also known as pharmacodynamics (PD). Evaluation of different patients’ responses as well as assessing the continued safety and tolerability of Cufence in different dose levels is also part of the study. This clinical study will be conducted at several hospitals across the EU and will involve approximately 50 patients in total. Participation in the study will be approximately 2 years.
REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
20/NW/0476
Date of REC Opinion
26 Feb 2021
REC opinion
Further Information Favourable Opinion