Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

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Brief Title

Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

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Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

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 Phase

Phase 4

Study Type

Interventional

Primary Outcome

Clinical outcome specific to the retrospective part of the study

Secondary Outcome

 Safety Endpoint Applicable to both the Retrospective and Prospective Parts of the Study

Condition

Wilson Disease

Intervention

trientine dihydrochloride

Study Arms / Comparison Groups

 Retrospective
Description:

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Drug

Estimated Enrollment

90

Start Date

January 2016

Completion Date

July 2018

Primary Completion Date

May 2018

Eligibility 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.

Gender

All

Ages

1 Year - 90 Years

Accepts Healthy Volunteers

No

Contacts

Karl-Heinz Weiss, MD, ,

Location Countries

Germany

Location Countries

Germany

Administrative Informations

NCT ID

NCT02426905

Organization ID

UNV-TRI-002

Responsible Party

Sponsor

Study Sponsor

Univar BV

Study Sponsor

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

Verification Date

August 2017