Overview

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

Status:
Completed

Trial end date:
1969-12-31

Target enrollment:

Participant gender:

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.

Phase:

Phase 2

Details

Lead Sponsor:

National Center for Research Resources (NCRR)

Collaborator:

University of California, San Diego