Wilson Disease MedDRA version: 22.1 Level: PT Classification code 10019819 Term: Hepato-lenticular degeneration System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Univar Solutions, B.V.
Eligibility
Sex/Gender
All
Inclusion criteria
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 (=65 years) yes F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The principle objective for this study is to provide more data on possible pharmacokinetic (PK) and pharmacodynamic (PD), further evaluate the efficacy and safety and find out the optimal treatment dose in adult and paediatric patients.;Secondary Objective: The secondary objectives are as follows: 1. to investigate the contribution of Cufence to the exposure and copper markers in patients with Wilson Disease. 2. To investigate the relationship between Cufence exposure and clinical efficacy measures in patients with Wilson Disease. 3. To investigate the relationship between copper markers and clinical efficacy measures in patients with Wilson Disease.;Primary end point(s): • Concentration of trientine in plasma • Trientine clearance (CL/F) and volume(s) of distribution (V/F) • 24-h UCE (copper marker) • NCC (copper marker) • Serum copper (copper marker) • Serum ceruloplasmin (copper marker) • Patient characteristics for covariates ;Timepoint(s) of evaluation of this end point: • Concentration of trientine in plasma at Visit 2- Visit 10 • Trientine clearance (CL/F) and volume(s) of distribution (V/F) at Visit 2- Visit 10 except Visit 5 • 24-h UCE (copper marker)at Visit 2- Visit 10 except Visit 5 • NCC* (copper marker) at Visit 2- Visit 10 except Visit 5 Serum copper (copper marker)- Visit 2- Visit 10 except Visit 5 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Concentration of MAT in plasma • Concentration of DAT in plasma • AUC0-tau, Cmax and Cmin for trientine and metabolites (by NCA) • 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) ;Timepoint(s) of evaluation of this end point: • Concentration of MAT in plasma at Visit 2- Visit 10 • Concentration of DAT in plasma at Visit 2- Visit 10 • AUC0-tau, Cmax and Cmin for trientine and metabolites (by NCA) at Visit 2- Visit 10 except Visit 5 • Number of AEs including number of AEs leading to discontinuation of treatment with Cufence at all visits • Changes in neurological disease status (Unified Wilson’s Disease Rating Scale (UWDRS)) at Visit 2,4 and Visit 6-10 • Changes in psychiatric symptoms (SCID-5, MMSE, EQ-5D-3L, PHQ-9, PHQ-9-A, CBCL)-various timepoint for each assessment • 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) | — |
Countries
Denmark, France, Germany, Poland
Contacts
Public ContactKarin Flicker
QPS Austria
Outcome results
None listed