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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title:

Brief 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 Summary:

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.

Sponsor: National Center for Research Resources (NCRR)

Current Primary Outcome:

Original Primary Outcome:

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Office of Rare Diseases (ORD)

Dates:
Date Received: October 18, 1999
Date Started: February 1996
Date Completion:
Last Updated: June 23, 2005
Last Verified: January 2000