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Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease

This study has been completed.
Sponsor:
Collaborator:
Valeant Pharmaceuticals International, Inc.
Information provided by (Responsible Party):
Michael Schilsky, Yale University
ClinicalTrials.gov Identifier:
NCT01472874
First received: November 11, 2011
Last updated: May 19, 2014
Last verified: May 2014
  Purpose

Hypothesis: The investigators postulate that patients with Wilson disease who are asymptomatic or who have been effectively treated for their symptoms and are in a maintenance phase therapy can be safely and effectively treated with a single daily dosage of the chelating agent trientine.

Specific Aims: To demonstrate that a single daily treatment with trientine is as effective or better than a patient's current maintenance therapy. This will be accomplished by performance of a case control prospective study of patients on their prior therapy, and during a period of treatment with a single weight based dose regimen of trientine.

The primary endpoint for this study is the demonstration of equivalence to a patient's prior therapy. Secondary endpoints include: 1) demonstration of stability or improvement in parameters of copper metabolism; 2) improvement in adherence to therapy; 3) no progression of liver disease (defined by changes in synthetic function, albumin and INR, and fibrosis by Fibrotest).


Condition Intervention
Wilson Disease
Drug: Once a day Trientine

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease

Resource links provided by NLM:


Further study details as provided by Yale University:

Primary Outcome Measures:
  • ALT [ Time Frame: Pre Treatment (mean) ] [ Designated as safety issue: Yes ]
    Alanine transaminase

  • ALT [ Time Frame: Months 1,2,3,6,9,12 (mean) ] [ Designated as safety issue: Yes ]
    Alanine transaminase

  • Cu Serum [ Time Frame: Pre Treatment (mean) ] [ Designated as safety issue: Yes ]
  • Cu Serum [ Time Frame: Months 1,2,3,6,9,12 (mean) ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • INR [ Time Frame: Pre Treatment (mean) ] [ Designated as safety issue: Yes ]
    The International Normalized Ratio (INR) is a standard way to describe the time it takes for blood to clot; an INR range of 0.8 to 1.2 is considered normal for a healthy person who is not using oral anticoagulant therapy

  • INR [ Time Frame: Months 1,2,3,6,9,12 (mean) ] [ Designated as safety issue: Yes ]
    The International Normalized Ratio (INR) is a standard way to describe the time it takes for blood to clot; an INR range of 0.8 to 1.2 is considered normal for a healthy person who is not using oral anticoagulant therapy

  • Albumin [ Time Frame: Pre Treatment (mean) ] [ Designated as safety issue: Yes ]
  • Albumin [ Time Frame: Months 1,2,3,6,9,12 (mean) ] [ Designated as safety issue: Yes ]
  • Cu Urine [ Time Frame: Pre Treatment (mean) ] [ Designated as safety issue: Yes ]
  • Cu Urine [ Time Frame: Months 1,2,3,6,9,12 (mean) ] [ Designated as safety issue: Yes ]
  • Zn Urine [ Time Frame: Pre Treatment (mean) ] [ Designated as safety issue: Yes ]
  • Zn Urine [ Time Frame: Months 1,2,3,6,9,12 (mean) ] [ Designated as safety issue: Yes ]

Enrollment: 8
Study Start Date: January 2010
Study Completion Date: July 2011
Primary Completion Date: July 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Once a day Trientine
Patients receive once a day trientine
Drug: Once a day Trientine
Trientine at a dosage of ~15 mg/kg rounded upwards to the nearest 250 or 300 mg in a single daily dosage. The entire daily dosage will be taken at once in the AM an hour before any meal. Duration of the study is 1 year.
Other Name: Syprine

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  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Established diagnosis of Wilson Disease:

  • That have been treated for at least 1 year
  • Compensated liver disease and/or stable neurological or psychiatric disease.
  • Normal or minimal elevation of serum ALT (<2 times upper limit of normal)
  • Non-ceruloplasmin copper <25 mcg/dl

Exclusion Criteria:

  • Wilson disease diagnosis not well established Wilson disease treated for less than one year Decompensated liver disease (ascites, jaundice, encephalopathy, bleeding due to portal hypertension) Liver disease with elevations of ALT > 2 times upper limit of normal A female who is pregnant or intends to become pregnant
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01472874

Locations
United States, Connecticut
Yale University
New Haven, Connecticut, United States, 06520
Sponsors and Collaborators
Yale University
Valeant Pharmaceuticals International, Inc.
Investigators
Principal Investigator: Michael Schilsky, MD Yale University
  More Information

Additional Information:
Publications:

Responsible Party: Michael Schilsky, Assoc Prof Int Med Digestive Disease and Surg Transplant, Yale University
ClinicalTrials.gov Identifier: NCT01472874     History of Changes
Other Study ID Numbers: ORPH-SYP-001
Study First Received: November 11, 2011
Results First Received: April 21, 2014
Last Updated: May 19, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by Yale University:
Wilson Disease
Trientine
One Daily Dosage

Additional relevant MeSH terms:
Hepatolenticular Degeneration
Basal Ganglia Diseases
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Central Nervous System Diseases
Digestive System Diseases
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Liver Diseases
Metabolic Diseases
Movement Disorders
Nervous System Diseases
Neurodegenerative Diseases
Metabolism, Inborn Errors
Metal Metabolism, Inborn Errors
Trientine
Chelating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Sequestering Agents

ClinicalTrials.gov processed this record on November 23, 2014