NCT00004314

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.

Trial Health

100
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
2

participants targeted

Target at below P25 for phase_2

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

February 1, 1996

Completed
3.7 years until next milestone

First Submitted

Initial submission to the registry

October 18, 1999

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 19, 1999

Completed
Last Updated

June 24, 2005

Status Verified

January 1, 2000

First QC Date

October 18, 1999

Last Update Submit

June 23, 2005

Conditions

Lesch-Nyhan Syndrome

Keywords

Lesch-Nyhan syndromeinborn errors of metabolismneurologic and psychiatric disordersrare disease

Interventions

aminomidazole carboxamide ribosideDRUG

Eligibility Criteria

Age0 Years+
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Men with Lesch-Nyhan syndrome Moderate erythrocyte macrocytosis No active urinary tract disease or impaired renal function

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Related Publications (2)

  • 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.

    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.

    PMID: 7660927BACKGROUND

MeSH Terms

Conditions

Lesch-Nyhan SyndromeMetabolism, Inborn ErrorsNeurologic ManifestationsMental DisordersRare Diseases

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesX-Linked Intellectual DisabilityIntellectual DisabilityNeurobehavioral ManifestationsGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHeredodegenerative Disorders, Nervous SystemPurine-Pyrimidine Metabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic DiseasesSigns and SymptomsPathological Conditions, Signs and SymptomsDisease AttributesPathologic Processes

Study Officials

  • Bruce Barshop

    University of California, San Diego

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Purpose
TREATMENT
Sponsor Type
NIH

Study Record Dates

First Submitted

October 18, 1999

First Posted

October 19, 1999

Study Start

February 1, 1996

Last Updated

June 24, 2005

Record last verified: 2000-01