Study of Tetrathiomolybdate in Patients With Wilson Disease
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