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

Lesch-Nyhan Syndrome Clinical Trial

Status Completed

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Primary Purpose: Treatment

Related Conditions & MeSH terms

• Lesch-Nyhan Syndrome

• NCT number: NCT00004314
• Study type: Interventional
• Source: Office of Rare Diseases (ORD)
• Status: Completed
• Phase: Phase 2
• Start date: February 1996