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

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00004314

Enrollment

Registered

1999-10-19

Start date

1996-02-29

Completion date

Unknown

Last updated

2005-06-24

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Lesch-Nyhan Syndrome

Keywords

Lesch-Nyhan syndrome, inborn errors of metabolism, neurologic and psychiatric disorders, rare disease

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 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.

Interventions

DRUGaminomidazole carboxamide riboside

Study design

Primary purpose

TREATMENT

Eligibility

Sex/Gender

MALE

Age

0 Years to No maximum

Healthy volunteers

Inclusion criteria

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

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026