Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine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
Sponsor:
Information provided by (Responsible Party):
Univar BV

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.

Condition or disease Intervention/treatment Phase
Wilson Disease Drug: trientine dihydrochloride Phase 4

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 90 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine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


Arm Intervention/treatment
No Intervention: Retrospective
Prospective Drug: trientine dihydrochloride
A retrospective review of patients' medical records




Primary Outcome Measures :
  1. 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.

  2. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   1 Year to 90 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

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


Locations
Layout table for location information
Germany
Universitätsklinik Heidelberg
Heidelberg, Germany, 69120
Greece
"Aghia Sophia" Children's Hospital
Goudi, Greece, 11527
Italy
San Paolo Hospital UOC
Milan, Italy, 20142
United Kingdom
Kings College Hospital
London, United Kingdom
Sponsors and Collaborators
Univar BV
Investigators
Layout table for investigator information
Principal Investigator: Karl-Heinz Weiss, MD Universitätsklinik Heidelberg

Layout table for additonal information
Responsible Party: Univar BV
ClinicalTrials.gov Identifier: NCT02426905     History of Changes
Other Study ID Numbers: UNV-TRI-002
First Posted: April 27, 2015    Key Record Dates
Last Update Posted: August 23, 2017
Last Verified: August 2017

Additional relevant MeSH terms:
Layout table for MeSH terms
Hepatolenticular Degeneration
Liver Diseases
Digestive System Diseases
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metal Metabolism, Inborn Errors
Metabolic Diseases
Trientine
Penicillamine
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action
Antidotes
Protective Agents
Physiological Effects of Drugs
Antirheumatic Agents