Study of Tetrathiomolybdate in Patients With Wilson Disease

NCT00004339 | Completed Phase 3

• 3 other identifiers: NCRR-M01RR00042-1850UMMC-801UMICH-FDU000505
• Study Type: interventional
• Target: 90
• Locations: 1 country
• Sites: 1

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.

Conditions

Wilson Disease

Keywords

Wilson disease; inborn errors of metabolism; rare disease

Interventions

tetrathiomolybdate DRUG

trientine DRUG

Eligibility Criteria

• Age: 0 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• National Center for Research Resources (NCRR): lead
• University of Michigan: collaborator

Study Sites (1)

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Related Publications (3)

• 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.

  PMID: 8198464 BACKGROUND

• 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.

  PMID: 8859064 BACKGROUND

• 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.

  PMID: 16606763 BACKGROUND

MeSH Terms

Conditions

Hepatolenticular Degeneration; Metabolism, Inborn Errors; Rare Diseases

Interventions

tetrathiomolybdate; Trientine

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metal Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Ethylenediamines; Diamines; Polyamines; Amines; Organic Chemicals

Study Officials

• George J. Brewer

  University of Michigan

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Purpose: TREATMENT
• Sponsor Type: NIH

Study Record Dates

• First Submitted: October 18, 1999
• First Posted: October 19, 1999
• Study Start: January 1, 1994
• Last Updated: May 9, 2006

Record last verified: 2003-12

Locations

US (1)