Wilson disease, also known as hepatolenticular degeneration, a rare automsomal, recessively inherited disorder which results in chronic copper intoxication. MedDRA version: 19.1 Level: LLT Classification code 10047988 Term: Wilson's disease System Organ Class: 100000004850
Conditions
Interventions
Trade Name: Trientine dihydrochloride 300mg capsules
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE
CAS Number: 38260-01-4
Concentration unit: mg milligram(s)
Concen
Sponsors
Univar B.V.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Patients aged 1 year to 90 years of age at initiation of treatment with trientine. - Physician established diagnosis of Wilson disease based on a Ferenci score > 3 at the date of diagnosis. - Documented treatment with d-Penicillamine, withdrawal of treatment with d- Penicillamine, followed by treatment with trientine for at least 6 months at date of informed consent. - Able/willing to provide written informed consent. For enrolment in the prospective part: - Enrolment in the retrospective part of the study. - On-going treatment with trientine. - Negative pregnancy test. - Able/willing to provide written informed consent. Are the trial subjects under 18? yes Number of subjects for this age range: 5 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 45 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 40
Exclusion criteria
Exclusion criteria: - Incomplete history of medication use for trientine from initial diagnosis to latest follow up. - Unavailable outcome data for hepatic and neurological course of disease at initiation of treatment with trientine and at least 1 time point post-initiation of treatment with trientine. For enrolment in the prospective part: - Hypersensitivity to trientine - Severe anaemia.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: - Assess the clinical course of Cu stores, neurological disease, and hepatic disease following initiation of treatment with trientine following withdrawal of d-Penicillamine. Retrospective data will be collected from visits at 6, 12, 24, 36 and 48 months after initiation of treatment, and at the last available follow up visit. Assessments will be based on the Investigator’s score (unchanged, improved but not normal, improved to normal, asymptomatic over duration, and worsened). Prospective assessments will be performed at Baseline (enrolment) and at 6 months and 12 months following the Baseline visit. - Assess the safety of trientine treatment based on reported AEs, including AEs leading to discontinuation of treatment with trientine. - Determine duration of treatment with second line therapy trientine, defined as time to discontinuation of treatment due to AEs and/or inadequate response. ;Secondary Objective: For the prospective part of the study: - Determine the proportion of patients with stable disease according to the Investigator's score after 12 months of treatment with trientine. - Assess the course of the hepatic disease. - Assess the course of disease according to the neurologic subscore of the Unified Wilson Disease Rating Scale (UWDRS). - Assess serum and urinary parameters of copper metabolism. - Assess quality of life (QoL).;Primary end point(s): - The clinical course of hepatic disease at 6, 12, 24, 36, and 48 months and at the last available time point while taking second line trientine, based on the Investigator’s score and based on hepatic status at the time of initiating trientine. - The clinical course of neurological disease at 6, 12, 24, 36, and 48 months and at the last available time point while taking second line trientine, based on the Investigator’s score and based on neurological status at the time of initiating trientine. - Cu storage and metabolism, measured at 6, 12, 24, 36, and 48 months and at the last available tim | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): For the prospective part:- - The clinical course of hepatic disease at 6 and 12 months while taking second line trientine, based on the Investigator’s score and based on hepatic status at the time of enrolment into prospective part in terms of: *AST to Platelet Ratio Index (APRI) based on AST, gender, and platelet count *Child-Pugh Score based on INR, total bilirubin, albumin, hepatic encephalopathy, and esophageal varices *FIB4 based on age, ALT, AST, and platelets *Model for End-Stage Liver Disease (MELD) based on INR, total bilirubin, creatinine, and sodium *Modified Nazer Score based on albumin, AST, total bilirubin, INR, and leukocytes *Fibroscan. - The clinical course of neurological disease at 6 and 12 months while taking second line trientine, based on the Investigator’s score and based on neurological status at the time of enrolment into prospective part. - The neurologic subscore of the UWDRS at Baseline, 6 and 12 months. - Cu storage and metabolism, measured at 6 and 12 months after initiating second line trientine therapy, by available tests, e.g., 24-hour urinary Cu excretion, serum CPN, and Serum Cu, compared to Cu storage values at the time of enrolment into prospective part. - Quality of life questionnaires will be completed for each time point and data will be compared to baseline (prospective part) after 6 and 12 months.;Timepoint(s) of evaluation of this end point: The 6 month and 12 month visits. | — |
Countries
Germany, Italy
Contacts
Public ContactTrientine Enquiries
Univar B.V.
Outcome results
None listed