Phase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease
Phase 2
Completed
- Conditions
- Lesch-Nyhan Syndrome
- Registration Number
- NCT00004314
- Lead Sponsor
- National Center for Research Resources (NCRR)
- Brief 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.
- Detailed 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 2
Inclusion Criteria
Not provided
Exclusion Criteria
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method