Status and phase
Conditions
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About
OBJECTIVES: I. Determine the effects of aminoimidazole carboxamide riboside (AICAR) on hematologic manifestations of Lesch-Nyhan disease.
II. Assess the behavioral and neurological benefits of AICAR in patients with Lesch-Nyhan disease.
III. Examine the effect of AICAR on purine production.
Full description
PROTOCOL OUTLINE:
After a 3-day baseline, patients are treated with aminoimidazole carboxamide riboside (AICAR) by continuous infusion for 12 days. Concurrent allopurinol and folate are allowed.
Sex
Volunteers
Inclusion and exclusion criteria
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics-- Men with Lesch-Nyhan syndrome Moderate erythrocyte macrocytosis No active urinary tract disease or impaired renal function
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Data sourced from clinicaltrials.gov
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