Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency

PHASE2CompletedINTERVENTIONAL
Enrollment

12

Participants

Timeline

Start Date

August 31, 2006

Primary Completion Date

June 30, 2009

Study Completion Date

June 30, 2009

Conditions
Tetrahydrobiopterin DeficienciesHyperphenylalaninemia, Non-Phenylketonuric
Interventions
DRUG

Phenoptin

5mg/kg/day orally, dose may be adjusted to between 5-20 mg/kg/day by investigator at week 6 to control blood Phe levels

Trial Locations (10)

10029

New York

27599

Chapel Hill

40225

Düsseldorf

53705

Madison

55455

Minneapolis

60614

Chicago

84132

Salt Lake City

90095

Los Angeles

97239

Portland

98105

Seattle

Sponsors
All Listed Sponsors
lead

BioMarin Pharmaceutical

INDUSTRY