Evaluate the safety and efficacy of ammonium tetrathiomolybdate alone and compared with
trientine therapy as initial treatment in patients with Wilson disease presenting
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
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:
- Wilson disease presenting with neurologic or psychiatric symptoms
- No concurrent seizure activity
- No white matter lesions on brain magnetic resonance imaging
- No more than 2 weeks of prior therapy
- No penicillamine or trientine for longer than 2 weeks
- Hepatic: No severe hepatic failure
- Other: No psychiatric or medical contraindication to protocol therapy
- Not pregnant