Wilson Disease
Conditions
Keywords
Wilson disease, inborn errors of metabolism, rare disease
Brief summary
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.
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.
Interventions
DRUGtrientine
Sponsors
University of Michigan
National Center for Research Resources (NCRR)
Study design
Allocation
RANDOMIZED
Primary purpose
TREATMENT
Masking
DOUBLE
Eligibility
Sex/Gender
ALL
Age
0 Years to No maximum
Healthy volunteers
No
Inclusion 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
Countries
United States
Outcome results
None listed