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Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine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 : Unknown
Verified August 2017 by Univar BV.
Recruitment status was:  Active, not recruiting
First Posted : April 27, 2015
Last Update Posted : August 23, 2017
Sponsor:
Information provided by (Responsible Party):
Univar BV

Tracking Information
First Submitted Date  ICMJE September 18, 2014
First Posted Date  ICMJE April 27, 2015
Last Update Posted Date August 23, 2017
Study Start Date  ICMJE January 2016
Estimated Primary Completion Date May 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 24, 2015)
  • 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 Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02426905 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 24, 2015)
  • 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 Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
Official Title  ICMJE 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
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Wilson Disease
Intervention  ICMJE Drug: trientine dihydrochloride
A retrospective review of patients' medical records
Study Arms  ICMJE
  • No Intervention: Retrospective
  • Prospective
    Intervention: Drug: trientine dihydrochloride
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: April 24, 2015)
90
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE July 2018
Estimated Primary Completion Date May 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Year to 90 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Germany,   Greece,   Italy,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02426905
Other Study ID Numbers  ICMJE UNV-TRI-002
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Univar BV
Study Sponsor  ICMJE Univar BV
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Karl-Heinz Weiss, MD Universitätsklinik Heidelberg
PRS Account Univar BV
Verification Date August 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP