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Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01472874
First Posted: November 17, 2011
Last Update Posted: May 22, 2014
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.
Collaborator:
Valeant Pharmaceuticals International, Inc.
Information provided by (Responsible Party):
Michael Schilsky, Yale University
  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: 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

Resource links provided by NLM:


Further study details as provided by Michael Schilsky, Yale University:

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

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

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

Secondary Outcome Measures:
  • 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

  • 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

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

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

  Show Detailed Description

  Eligibility

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.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
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
  Contacts and Locations
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
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

Publications:
Roberts EA, Schilsky ML; Division of Gastroenterology and Nutrition, Hospital for Sick Children, Toronto, Ontario, Canada. A practice guideline on Wilson disease. Hepatology. 2003 Jun;37(6):1475-92. Erratum in: Hepatology. 2003 Aug;38(2):536.
Schilsky ML, Scheinberg IH, Sternlieb I. Liver transplantation for Wilson's disease: indications and outcome. Hepatology. 1994 Mar;19(3):583-7.
Emre S, Atillasoy EO, Ozdemir S, Schilsky M, Rathna Varma CV, Thung SN, Sternlieb I, Guy SR, Sheiner PA, Schwartz ME, Miller CM. Orthotopic liver transplantation for Wilson's disease: a single-center experience. Transplantation. 2001 Oct 15;72(7):1232-6.
Askari FK, Greenson J, Dick RD, Johnson VD, Brewer GJ. Treatment of Wilson's disease with zinc. XVIII. Initial treatment of the hepatic decompensation presentation with trientine and zinc. J Lab Clin Med. 2003 Dec;142(6):385-90.
Brewer GJ, Dick RD, Johnson VD, Fink JK, Kluin KJ, Daniels S. Treatment of Wilson's disease with zinc XVI: treatment during the pediatric years. J Lab Clin Med. 2001 Mar;137(3):191-8.
Ferenci P. Wilson's Disease. Clin Gastroenterol Hepatol. 2005 Aug;3(8):726-33. Review.
Brewer GJ, Askari F, Lorincz MT, Carlson M, Schilsky M, Kluin KJ, Hedera P, Moretti P, Fink JK, Tankanow R, Dick RB, Sitterly J. Treatment of Wilson disease with ammonium tetrathiomolybdate: IV. Comparison of tetrathiomolybdate and trientine in a double-blind study of treatment of the neurologic presentation of Wilson disease. Arch Neurol. 2006 Apr;63(4):521-7.
Scheinberg IH, Jaffe ME, Sternlieb I. The use of trientine in preventing the effects of interrupting penicillamine therapy in Wilson's disease. N Engl J Med. 1987 Jul 23;317(4):209-13.
Walshe JM. Treatment of Wilson's disease with trientine (triethylene tetramine) dihydrochloride. Lancet. 1982 Mar 20;1(8273):643-7.
Ferenci P, Caca K, Loudianos G, Mieli-Vergani G, Tanner S, Sternlieb I, Schilsky M, Cox D, Berr F. Diagnosis and phenotypic classification of Wilson disease. Liver Int. 2003 Jun;23(3):139-42. Review.

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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
First Submitted: November 11, 2011
First Posted: November 17, 2011
Results First Submitted: April 21, 2014
Results First Posted: May 19, 2014
Last Update Posted: May 22, 2014
Last Verified: May 2014

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

Additional relevant 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
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action


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