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

Lesch-Nyhan Syndrome Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00004314
• Other study ID #: 199/11823
• Secondary ID: UCSD-733
• Status: Completed
• Phase: Phase 2
• First received: October 18, 1999
• Last updated: June 23, 2005
• Start date: February 1996

Study information

• Verified date: January 2000
• Source: Office of Rare Diseases (ORD)
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 2
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics-- Men with Lesch-Nyhan syndrome Moderate erythrocyte macrocytosis No active urinary tract disease or impaired renal function

Study Design

Primary Purpose: Treatment

Related Conditions & MeSH terms

• Lesch-Nyhan Syndrome

Intervention

Drug:
• aminomidazole carboxamide riboside

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
National Center for Research Resources (NCRR)	University of California, San Diego

References & Publications (2)

Page T, Barshop B, Yu AL, Nyhan WL. Treatment of Lesch-Nyhan syndrome with AICAR. Adv Exp Med Biol. 1994;370:353-6. — View Citation

Page T, Barshop BA, Yu A, et al.: Treatment of Lesch-Nyhan syndrome with AICAR. In: Sahota A, Tayor M, eds.: Purine and Pyrimidine Metabolism in Man VIII. Plenum Press, New York, NY: 1995, pp 353-356.