WithdrawnPHASE2, PHASE3NCT00935753

Trial of Kuvan in Lesch-Nyhan Disease

Studying Disorder of purine metabolism

Last synced from ClinicalTrials.gov May 8, 2026

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Key facts

Sponsor: University of California, San Diego
Principal Investigator: William L Nyhan, MD, PhD
University of California, San Diego
Intervention: sapropterin(drug)
Eligibility: 4 years · All sexes
Timeline: 2009 – 2010

Study locations (1)

UCSD Mitochondrial and Metabolic Disease Center, San Diego, California, United States

Collaborators

BioMarin Pharmaceutical

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Open NCT00935753 on ClinicalTrials.gov

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