Study of Tetrathiomolybdate in Patients With Wilson Disease

National Center for Research Resources (NCRR)90 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Wilson Disease

Interventions

tetrathiomolybdateDRUG

trientineDRUG

Eligibility

Sex: ALLMin age: 0 Years

Medical Language ↔ Plain English

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

Locations (1)

University of Michigan

Ann Arbor, Michigan, United States

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.