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

Status

Completed

Phases

Phase 4

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-516431-27-00

Acronym

TR-004

Enrollment

Registered

2024-09-11

Start date

2021-02-23

Completion date

2025-09-19

Last updated

2024-10-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Wilson’s Disease

Brief summary

Concentration of trientine in plasma, Trientine clearance (CL/F) and volume(s) of distribution (V/F), 24-hr UCE (copper marker), NCC (copper marker), Serum copper (copper marker), Serum ceruloplasmin (copper marker), Patient characteristics for covariates

Detailed description

Concentration of MAT in plasma, Concentration of DAT in plasma, AUC0-t, Cmax and Ctrough for trientine and metabolites. (As secondary endpoint, pre-dose concentrations are reported and summarized using NCA. While the pre-dose PK samples are collected shortly prior to the next dose, the pre-dose concentration is assumed to approximate the Ctrough.), Number of AEs including number of AEs leading to discontinuation of treatment with Cufence, Changes in neurological disease status (Unified Wilson’s Disease Rating Scale (UWDRS)), Changes in psychiatric symptoms (SCID-5, MMSE, EQ-5D-3L, PHQ-9, PHQ-9-A, CBCL), Changes in hepatic disease (full liver panel to determine the APRI, the Child-Pugh score, the FIB4 index and the New Wilson Index (Dhawan Index), and Fibroscan)

Interventions

DRUGCufence 200 mg hard capsules

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Concentration of trientine in plasma, Trientine clearance (CL/F) and volume(s) of distribution (V/F), 24-hr UCE (copper marker), NCC (copper marker), Serum copper (copper marker), Serum ceruloplasmin (copper marker), Patient characteristics for covariates	—

Secondary

Measure	Time frame
Concentration of MAT in plasma, Concentration of DAT in plasma, AUC0-t, Cmax and Ctrough for trientine and metabolites. (As secondary endpoint, pre-dose concentrations are reported and summarized using NCA. While the pre-dose PK samples are collected shortly prior to the next dose, the pre-dose concentration is assumed to approximate the Ctrough.), Number of AEs including number of AEs leading to discontinuation of treatment with Cufence, Changes in neurological disease status (Unified Wilson’s Disease Rating Scale (UWDRS)), Changes in psychiatric symptoms (SCID-5, MMSE, EQ-5D-3L, PHQ-9, PHQ-9-A, CBCL), Changes in hepatic disease (full liver panel to determine the APRI, the Child-Pugh score, the FIB4 index and the New Wilson Index (Dhawan Index), and Fibroscan)	—

Measure

Time frame

—

Countries

Denmark, France, Germany, Poland

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026