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Phase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease

N

National Center for Research Resources (NCRR)

Status and phase

Completed
Phase 2

Conditions

Lesch-Nyhan Syndrome

Treatments

Drug: aminomidazole carboxamide riboside

Study type

Interventional

Funder types

Other
NIH

Identifiers

NCT00004314
199/11823
UCSD-733

Details and patient eligibility

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

Male

Volunteers

No Healthy 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

Trial contacts and locations

0

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Data sourced from clinicaltrials.gov

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