Phase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease
NCT00004314 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 2
Last updated 2005-06-24
Summary
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.
Conditions
- Lesch-Nyhan Syndrome
Interventions
- DRUG
-
aminomidazole carboxamide riboside
Sponsors & Collaborators
-
University of California, San Diego
collaborator OTHER -
National Center for Research Resources (NCRR)
lead NIH
Principal Investigators
-
Bruce Barshop · University of California, San Diego
Study Design
- Purpose
- TREATMENT
Eligibility
- Min Age
- 0 Years
- Sex
- MALE
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 1996-02-29
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