Study of Tetrathiomolybdate in Patients With Wilson Disease - Full Text View

Purpose

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.

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

Genetics Home Reference related topics:

familial encephalopathy with neuroserpin inclusion bodies familial paroxysmal nonkinesigenic dyskinesia Wilson disease

MedlinePlus related topics:

Wilson Disease

Drug Information available for:

Tetrathiomolybdate

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double-Blind

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.

Eligibility

Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004339

Locations


United States, Michigan
	University of Michigan
	Ann Arbor, Michigan, United States, 48109

Sponsors and Collaborators

National Center for Research Resources (NCRR)

University of Michigan

Investigators


Study Chair:	George J. Brewer	University of Michigan

More Information

Publications:

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.

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.

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.

Study ID Numbers:	NCRR-M01RR00042-1850, UMMC-801, UMICH-FDU000505
First Received:	October 18, 1999
Last Updated:	May 8, 2006
ClinicalTrials.gov Identifier:	NCT00004339
Health Authority:	United States: Federal Government

Keywords provided by National Center for Research Resources (NCRR):

Wilson disease

inborn errors of metabolism

rare disease

Study placed in the following topic categories:

Wilson disease

Liver Diseases

Metabolic Diseases

Ganglion Cysts

Hepatolenticular Degeneration

Molybdenum

Basal Ganglia Diseases

Rare Diseases

Central Nervous System Diseases

Neurodegenerative Diseases

Brain Diseases

Tetrathiomolybdate

Metabolism, Inborn Errors

Digestive System Diseases

Heredodegenerative Disorders, Nervous System

Genetic Diseases, Inborn

Movement Disorders

Brain Diseases, Metabolic, Inborn

Triethylenetetramine

Metabolic disorder

Brain Diseases, Metabolic

Additional relevant MeSH terms:

Molecular Mechanisms of Pharmacological Action

Antineoplastic Agents

Growth Substances

Nervous System Diseases

Physiological Effects of Drugs

Trace Elements

Enzyme Inhibitors

Metal Metabolism, Inborn Errors

Angiogenesis Inhibitors

Pharmacologic Actions

Therapeutic Uses

Micronutrients

Chelating Agents

Angiogenesis Modulating Agents

Growth Inhibitors

ClinicalTrials.gov processed this record on November 30, 2008

Sponsors and Collaborators:	National Center for Research Resources (NCRR) University of Michigan
Information provided by:	National Center for Research Resources (NCRR)
ClinicalTrials.gov Identifier:	NCT00004339