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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

2 participants

Study Classification

INTERVENTIONAL

Study Start Date

1996-02-29

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Lesch-Nyhan Syndrome

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Primary Study Purpose

TREATMENT

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

aminomidazole carboxamide riboside

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

PROTOCOL ENTRY CRITERIA:

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

Minimum Eligible Age

0 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Bruce Barshop

Role: STUDY_CHAIR

University of California, San Diego

References

Explore related publications, articles, or registry entries linked to this study.

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.

Reference Type BACKGROUND

Page T, Barshop B, Yu AL, Nyhan WL. Treatment of Lesch-Nyhan syndrome with AICAR. Adv Exp Med Biol. 1994;370:353-6. doi: 10.1007/978-1-4615-2584-4_76. No abstract available.

Reference Type BACKGROUND

PMID: 7660927 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

UCSD-733

Identifier Type: -

Identifier Source: secondary_id

199/11823