Phase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease
|ClinicalTrials.gov Identifier: NCT00004314|
Recruitment Status : Completed
First Posted : October 19, 1999
Last Update Posted : June 24, 2005
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.
|Condition or disease||Intervention/treatment||Phase|
|Lesch-Nyhan Syndrome||Drug: aminomidazole carboxamide riboside||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||2 participants|
|Study Start Date :||February 1996|
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004314
|Study Chair:||Bruce Barshop||University of California, San Diego|