Study of Tetrathiomolybdate in Patients With Wilson Disease

This study has been completed.
University of Michigan
Information provided by:
National Center for Research Resources (NCRR) Identifier:
First received: October 18, 1999
Last updated: May 8, 2006
Last verified: December 2003


Evaluate the safety and efficacy of ammonium tetrathiomolybdate alone and compared with trientine therapy as initial treatment in patients with Wilson disease presenting neurologically.

Condition Intervention Phase
Wilson Disease
Drug: tetrathiomolybdate
Drug: trientine
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Masking: Double-Blind
Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by National Center for Research Resources (NCRR):

Estimated Enrollment: 90
Study Start Date: January 1994
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.


Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


--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
  Contacts and Locations
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Please refer to this study by its identifier: NCT00004339

United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48109
Sponsors and Collaborators
National Center for Research Resources (NCRR)
University of Michigan
Study Chair: George J. Brewer University of Michigan
  More Information

Publications: Identifier: NCT00004339     History of Changes
Other Study ID Numbers: NCRR-M01RR00042-1850  UMMC-801  UMICH-FDU000505 
Study First Received: October 18, 1999
Last Updated: May 8, 2006
Health Authority: United States: Federal Government

Keywords provided by National Center for Research Resources (NCRR):
Wilson disease
inborn errors of metabolism
rare disease

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
Metabolism, Inborn Errors
Metal Metabolism, Inborn Errors
Movement Disorders
Nervous System Diseases
Neurodegenerative Diseases
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Antineoplastic Agents
Chelating Agents
Enzyme Inhibitors
Growth Inhibitors
Growth Substances
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Sequestering Agents
Trace Elements processed this record on May 23, 2016