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Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

Primary Purpose

Wilson Disease

Status
Unknown status
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
trientine dihydrochloride
Sponsored by
Univar BV
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Wilson Disease

Eligibility Criteria

1 Year - 90 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Patients aged 1 year to 90 years of age.
  • Physician established diagnosis of Wilson disease based on a Ferenci score > 3.
  • 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 is required.

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 assessment time points.
  • Patients with acute liver failure and fulminant hepatic disease with fatal outcome.
  • Hypersensitivity to trientine and severe anaemia.

Sites / Locations

  • Universitätsklinik Heidelberg
  • "Aghia Sophia" Children's Hospital
  • San Paolo Hospital UOC
  • Kings College Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

No Intervention

Other

Arm Label

Retrospective

Prospective

Arm Description

Outcomes

Primary Outcome Measures

Clinical outcome specific to the retrospective part of the study
The clinical course of neurological and hepatic disease for each available time point after initiation of treatment (6, 12, 24, 36, and 48 months, and at the last available time point while taking second line trientine) will be scored (Investigator's score) based on neurological and hepatic status at the time of initiating trientine as: 1 = Unchanged 2 = Improved but not normal 3 = Improved to normal 4 = Asymptomatic over duration of therapy 5 = Worsened.
Clinical outcome specific to the prospective part of the study
The clinical course of neurological and hepatic disease will be scored (Investigator's score) based on the status at 6 and 12 months after Baseline as: 1 = Unchanged 2 = Improved but not normal 3 = Improved to normal 4 = Asymptomatic over duration of therapy 5 = Worsened A patient will be counted as a responder if they have a rating of ≤4 at the 12 month visit for both the neurological and hepatic Investigator's score. They will be counted as a non-responder if they have a rating = 5 for one or both scores at the 12 month visit or if they were discontinued from the study for any reason prior to the 12 month visit.

Secondary Outcome Measures

Safety Endpoint Applicable to both the Retrospective and Prospective Parts of the Study
All AEs related to trientine treatment, and AEs leading to discontinuation of trientine will be assessed at each available study time point.
Quality of Life Endpoints for the Prospective Part of the Study
The QoL questionnaires will be completed for each time point and data will be compared to baseline (prospective part) after 6 and 12 months

Full Information

First Posted
September 18, 2014
Last Updated
August 22, 2017
Sponsor
Univar BV
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1. Study Identification

Unique Protocol Identification Number
NCT02426905
Brief Title
Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
Official Title
Multicentre, Retrospective and Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
Study Type
Interventional

2. Study Status

Record Verification Date
August 2017
Overall Recruitment Status
Unknown status
Study Start Date
January 2016 (undefined)
Primary Completion Date
May 2018 (Anticipated)
Study Completion Date
July 2018 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Univar BV

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
A study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them for a further 12 months.
Detailed Description
A retrospective study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them prospectively for a further 12 months.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Wilson Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
90 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Retrospective
Arm Type
No Intervention
Arm Title
Prospective
Arm Type
Other
Intervention Type
Drug
Intervention Name(s)
trientine dihydrochloride
Intervention Description
A retrospective review of patients' medical records
Primary Outcome Measure Information:
Title
Clinical outcome specific to the retrospective part of the study
Description
The clinical course of neurological and hepatic disease for each available time point after initiation of treatment (6, 12, 24, 36, and 48 months, and at the last available time point while taking second line trientine) will be scored (Investigator's score) based on neurological and hepatic status at the time of initiating trientine as: 1 = Unchanged 2 = Improved but not normal 3 = Improved to normal 4 = Asymptomatic over duration of therapy 5 = Worsened.
Time Frame
48 months
Title
Clinical outcome specific to the prospective part of the study
Description
The clinical course of neurological and hepatic disease will be scored (Investigator's score) based on the status at 6 and 12 months after Baseline as: 1 = Unchanged 2 = Improved but not normal 3 = Improved to normal 4 = Asymptomatic over duration of therapy 5 = Worsened A patient will be counted as a responder if they have a rating of ≤4 at the 12 month visit for both the neurological and hepatic Investigator's score. They will be counted as a non-responder if they have a rating = 5 for one or both scores at the 12 month visit or if they were discontinued from the study for any reason prior to the 12 month visit.
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Safety Endpoint Applicable to both the Retrospective and Prospective Parts of the Study
Description
All AEs related to trientine treatment, and AEs leading to discontinuation of trientine will be assessed at each available study time point.
Time Frame
Up to 60 months
Title
Quality of Life Endpoints for the Prospective Part of the Study
Description
The QoL questionnaires will be completed for each time point and data will be compared to baseline (prospective part) after 6 and 12 months
Time Frame
12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
90 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients aged 1 year to 90 years of age. Physician established diagnosis of Wilson disease based on a Ferenci score > 3. 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 is required. 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 assessment time points. Patients with acute liver failure and fulminant hepatic disease with fatal outcome. Hypersensitivity to trientine and severe anaemia.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Karl-Heinz Weiss, MD
Organizational Affiliation
Universitätsklinik Heidelberg
Official's Role
Principal Investigator
Facility Information:
Facility Name
Universitätsklinik Heidelberg
City
Heidelberg
ZIP/Postal Code
69120
Country
Germany
Facility Name
"Aghia Sophia" Children's Hospital
City
Goudi
ZIP/Postal Code
11527
Country
Greece
Facility Name
San Paolo Hospital UOC
City
Milan
ZIP/Postal Code
20142
Country
Italy
Facility Name
Kings College Hospital
City
London
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
35482910
Citation
Weiss KH, Kruse C, Manolaki N, Zuin M, Ferenci P, van Scheppingen D, Wijnberg L, de Koning CE, Dhawan A. Multicentre, retrospective study to assess long-term outcomes of chelator based treatment with trientine in Wilson disease patients withdrawn from therapy with d -penicillamine. Eur J Gastroenterol Hepatol. 2022 Sep 1;34(9):940-947. doi: 10.1097/MEG.0000000000002387. Epub 2022 Apr 29.
Results Reference
derived

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Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

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