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Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease

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ClinicalTrials.gov Identifier: NCT01472874
Recruitment Status : Completed
First Posted : November 17, 2011
Results First Posted : May 19, 2014
Last Update Posted : April 15, 2020
Sponsor:
Collaborator:
Bausch Health Americas, Inc.
Information provided by (Responsible Party):
Yale University

Brief Summary:

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 or disease Intervention/treatment Phase
Wilson Disease Drug: Once a day Trientine Not Applicable

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease
Study Start Date : January 2010
Actual Primary Completion Date : July 2011
Actual Study Completion Date : July 2011


Arm Intervention/treatment
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




Primary Outcome Measures :
  1. ALT [ Time Frame: Pre Treatment (mean) ]
    Alanine transaminase

  2. ALT [ Time Frame: Months 1,2,3,6,9,12 (mean) ]
    Alanine transaminase

  3. Cu Serum [ Time Frame: Pre Treatment (mean) ]
  4. Cu Serum [ Time Frame: Months 1,2,3,6,9,12 (mean) ]

Secondary Outcome Measures :
  1. INR [ Time Frame: Pre Treatment (mean) ]
    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

  2. INR [ Time Frame: Months 1,2,3,6,9,12 (mean) ]
    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

  3. Albumin [ Time Frame: Pre Treatment (mean) ]
  4. Albumin [ Time Frame: Months 1,2,3,6,9,12 (mean) ]
  5. Cu Urine [ Time Frame: Pre Treatment (mean) ]
  6. Cu Urine [ Time Frame: Months 1,2,3,6,9,12 (mean) ]
  7. Zn Urine [ Time Frame: Pre Treatment (mean) ]
  8. Zn Urine [ Time Frame: Months 1,2,3,6,9,12 (mean) ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
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

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): NCT01472874


Locations
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United States, Connecticut
Yale University
New Haven, Connecticut, United States, 06520
Sponsors and Collaborators
Yale University
Bausch Health Americas, Inc.
Investigators
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Principal Investigator: Michael Schilsky, MD Yale University
Publications:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Yale University
ClinicalTrials.gov Identifier: NCT01472874    
Other Study ID Numbers: 0902004694
ORPH-SYP-001 ( Other Identifier: Valeant reference # )
First Posted: November 17, 2011    Key Record Dates
Results First Posted: May 19, 2014
Last Update Posted: April 15, 2020
Last Verified: April 2020
Keywords provided by Yale University:
Wilson Disease
Trientine
One Daily Dosage
Additional relevant MeSH terms:
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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
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action