Clinical Trials Directory

Trials / Completed

CompletedNCT00004314

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

Status
Completed
Phase
Phase 2
Study type
Interventional
Enrollment
2 (planned)
Sponsor
National Center for Research Resources (NCRR) · NIH
Sex
Male
Age
0 Years
Healthy volunteers
Not accepted

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.

Conditions

Interventions

TypeNameDescription
DRUGaminomidazole carboxamide riboside

Timeline

Start date
1996-02-01
First posted
1999-10-19
Last updated
2005-06-24

Source: ClinicalTrials.gov record NCT00004314. Inclusion in this directory is not an endorsement.