Trial of Kuvan in Lesch-Nyhan Disease

PHASE2/PHASE3WithdrawnINTERVENTIONAL
0
Timeline

Start Date

April 30, 2009

Primary Completion Date

February 28, 2010

Study Completion Date

February 28, 2010

Conditions
Behavioral Manifestations of Lesch-Nyhan Disease
Interventions
DRUG

sapropterin

oral 100mg tablets taken intact or dissolved in water or apple juice with morning meal for up to 60 days

Trial Locations (1)

92103

UCSD Mitochondrial and Metabolic Disease Center, San Diego

Sponsors

Collaborators (1)

All Listed Sponsors
collaborator

BioMarin Pharmaceutical

INDUSTRY

lead

University of California, San Diego

OTHER