Clinical trialNCT00004314CompletedPhase 2Interventional

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

SponsorNational Center for Research Resources (NCRR)Enrollment2Lesch-Nyhan Syndrome

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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.

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Investigators

The overall officials registered on this study. Names that resolve to a FindMyKOL profile link to it; the rest are shown as registered.

Bruce Barshop
University of California, San Diego
Study Chair
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A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

No facility list is in our current data snapshot for this study. The registry record on ClinicalTrials.gov ↗ carries any published locations. Why? →

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

Aminomidazole carboxamide riboside

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 29 Feb 1996
Primary completion: —
Completion: —
Last update posted: 24 Jun 2005
Registered enrollment: 2
Registry id: NCT00004314

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Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; FindMyKOL does not interpret them.