Phase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|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|
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004314
|Study Chair:||Bruce Barshop||University of California, San Diego|