Study of Tetrathiomolybdate in Patients With Wilson Disease

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.

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