Study of Tetrathiomolybdate in Patients With Wilson Disease
Primary Purpose
Wilson Disease
Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
tetrathiomolybdate
trientine
Sponsored by
About this trial
This is an interventional treatment trial for Wilson Disease focused on measuring Wilson disease, inborn errors of metabolism, rare disease
Eligibility Criteria
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Wilson disease presenting with neurologic or psychiatric symptoms No concurrent seizure activity No white matter lesions on brain magnetic resonance imaging --Prior/Concurrent Therapy-- No more than 2 weeks of prior therapy No penicillamine or trientine for longer than 2 weeks --Patient Characteristics-- Hepatic: No severe hepatic failure Other: No psychiatric or medical contraindication to protocol therapy Not pregnant
Sites / Locations
- University of Michigan
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT00004339
First Posted
October 18, 1999
Last Updated
May 8, 2006
Sponsor
National Center for Research Resources (NCRR)
Collaborators
University of Michigan
1. Study Identification
Unique Protocol Identification Number
NCT00004339
Brief Title
Study of Tetrathiomolybdate in Patients With Wilson Disease
Study Type
Interventional
2. Study Status
Record Verification Date
December 2003
Overall Recruitment Status
Completed
Study Start Date
January 1994 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Name of the Sponsor
National Center for Research Resources (NCRR)
Collaborators
University of Michigan
4. Oversight
5. Study Description
Brief Summary
OBJECTIVES:
Evaluate the safety and efficacy of ammonium tetrathiomolybdate alone and compared with trientine therapy as initial treatment in patients with Wilson disease presenting neurologically.
Detailed Description
PROTOCOL OUTLINE: This a double blind, randomized study. Patients are randomized into one of two treatment arms.
Arm I: Patients receive tetrathiomolybdate (TM) 3 times a day with meals and 3 times a day between meals for 8 weeks in the absence of neurologic deterioration or unacceptable toxicity.
Arm II: Patients receive trientine therapy for 8 weeks in the absence of neurologic deterioration and unacceptable toxicity.
Additional therapy (off study): Patients in the TM group may receive maintenance zinc, while those in the trientine group may continue on trientine or switch to zinc.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Wilson Disease
Keywords
Wilson disease, inborn errors of metabolism, rare disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Masking
Double
Allocation
Randomized
Enrollment
90 (false)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
tetrathiomolybdate
Intervention Type
Drug
Intervention Name(s)
trientine
10. Eligibility
Sex
All
Minimum Age & Unit of Time
0 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Wilson disease presenting with neurologic or psychiatric symptoms
No concurrent seizure activity
No white matter lesions on brain magnetic resonance imaging
--Prior/Concurrent Therapy--
No more than 2 weeks of prior therapy
No penicillamine or trientine for longer than 2 weeks
--Patient Characteristics--
Hepatic: No severe hepatic failure
Other: No psychiatric or medical contraindication to protocol therapy
Not pregnant
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
George J. Brewer
Organizational Affiliation
University of Michigan
Official's Role
Study Chair
Facility Information:
Facility Name
University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
8198464
Citation
Brewer GJ, Dick RD, Johnson V, Wang Y, Yuzbasiyan-Gurkan V, Kluin K, Fink JK, Aisen A. Treatment of Wilson's disease with ammonium tetrathiomolybdate. I. Initial therapy in 17 neurologically affected patients. Arch Neurol. 1994 Jun;51(6):545-54. doi: 10.1001/archneur.1994.00540180023009.
Results Reference
background
PubMed Identifier
8859064
Citation
Brewer GJ, Johnson V, Dick RD, Kluin KJ, Fink JK, Brunberg JA. Treatment of Wilson disease with ammonium tetrathiomolybdate. II. Initial therapy in 33 neurologically affected patients and follow-up with zinc therapy. Arch Neurol. 1996 Oct;53(10):1017-25. doi: 10.1001/archneur.1996.00550100103019.
Results Reference
background
PubMed Identifier
16606763
Citation
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. doi: 10.1001/archneur.63.4.521.
Results Reference
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Study of Tetrathiomolybdate in Patients With Wilson Disease
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