Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02426905|
Recruitment Status : Active, not recruiting
First Posted : April 27, 2015
Last Update Posted : August 23, 2017
|Condition or disease||Intervention/treatment||Phase|
|Wilson Disease||Drug: trientine dihydrochloride||Phase 4|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||90 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine|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 Start Date :||January 2016|
|Estimated Primary Completion Date :||May 2018|
|Estimated Study Completion Date :||July 2018|
|No Intervention: Retrospective|
Drug: trientine dihydrochloride
A retrospective review of patients' medical records
- 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.
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02426905
|Heidelberg, Germany, 69120|
|"Aghia Sophia" Children's Hospital|
|Goudi, Greece, 11527|
|San Paolo Hospital UOC|
|Milan, Italy, 20142|
|Kings College Hospital|
|London, United Kingdom|
|Principal Investigator:||Karl-Heinz Weiss, MD||Universitätsklinik Heidelberg|