Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

August 22, 2017 updated by: Univar BV

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Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Anticipated)

90

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
      • Heidelberg, Germany, 69120
        • Universitätsklinik Heidelberg
  • Greece
      • Goudi, Greece, 11527
        • "Aghia Sophia" Children's Hospital
  • Italy
      • Milan, Italy, 20142
        • San Paolo Hospital UOC
  • United Kingdom
      • London, United Kingdom
        • Kings College Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 90 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
No Intervention: Retrospective
Other: Prospective
Drug: trientine dihydrochloride
A retrospective review of patients' medical records

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Clinical outcome specific to the retrospective part of the study
Time Frame: 48 months
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.
48 months
Clinical outcome specific to the prospective part of the study
Time Frame: 12 months
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.
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety Endpoint Applicable to both the Retrospective and Prospective Parts of the Study
Time Frame: Up to 60 months
All AEs related to trientine treatment, and AEs leading to discontinuation of trientine will be assessed at each available study time point.
Up to 60 months
Quality of Life Endpoints for the Prospective Part of the Study
Time Frame: 12 months
The QoL questionnaires will be completed for each time point and data will be compared to baseline (prospective part) after 6 and 12 months
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Univar BV

Investigators

  • Principal Investigator: Karl-Heinz Weiss, MD, Universitätsklinik Heidelberg

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2016

Primary Completion (Anticipated)

May 1, 2018

Study Completion (Anticipated)

July 1, 2018

Study Registration Dates

First Submitted

September 18, 2014

First Submitted That Met QC Criteria

April 24, 2015

First Posted (Estimate)

April 27, 2015

Study Record Updates

Last Update Posted (Actual)

August 23, 2017

Last Update Submitted That Met QC Criteria

August 22, 2017

Last Verified

August 1, 2017

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • UNV-TRI-002

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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