Clinical Trial: Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn From Therapy

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 Summary: 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.
Sponsor: Univar BV

Current Primary Outcome:

• 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.
• 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.

Original Primary Outcome: Same as current

Current Secondary Outcome:

• 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.
• 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

Original Secondary Outcome: Same as current

Information By: Univar BV

Dates:
Date Received: September 18, 2014
Date Started: January 2016
Date Completion: May 2018
Last Updated: March 13, 2017
Last Verified: March 2017