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Sponsored by: |
BioMarin Pharmaceutical |
Information provided by: | BioMarin Pharmaceutical |
ClinicalTrials.gov Identifier: | NCT00104247 |
The primary objective of this study is to evaluate the efficacy of Phenoptin™ (sapropterin dihydrochloride) in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria.
Condition | Intervention | Phase |
Phenylketonurias |
Drug: sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin |
Phase III |
Genetics Home Reference related topics: | argininosuccinic aciduria citrullinemia N-acetylglutamate synthase deficiency ornithine translocase deficiency phenylketonuria tetrahydrobiopterin deficiency |
MedlinePlus related topics: | Phenylketonuria |
ChemIDplus related topics: | 5,6,7,8-Tetrahydrobiopterin Phenylalanine Sapropterin Sapropterin dihydrochloride |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study |
Official Title: | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels |
Estimated Enrollment: | 100 |
Study Start Date: | March 2005 |
Study Completion Date: | February 2006 |
Ages Eligible for Study: | 8 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, California | |||||
Los Angeles, California, United States | |||||
Oakland, California, United States | |||||
United States, Connecticut | |||||
New Haven, Connecticut, United States | |||||
United States, Illinois | |||||
Chicago, Illinois, United States | |||||
United States, Massachusetts | |||||
Boston, Massachusetts, United States | |||||
United States, Minnesota | |||||
Minneapolis, Minnesota, United States | |||||
United States, Missouri | |||||
St. Louis, Missouri, United States | |||||
United States, New York | |||||
New York, New York, United States | |||||
United States, Oregon | |||||
Portland, Oregon, United States | |||||
United States, Pennsylvania | |||||
Pittsburgh, Pennsylvania, United States | |||||
United States, Texas | |||||
Dallas, Texas, United States | |||||
United States, Utah | |||||
Salt Lake City, Utah, United States | |||||
United States, Wisconsin | |||||
Madison, Wisconsin, United States |
BioMarin Pharmaceutical |
BioMarin Pharmaceutical Inc. home page 
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NDA Review Package 
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Product Label 
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Study ID Numbers: | PKU-003 |
First Received: | February 24, 2005 |
Last Updated: | April 29, 2008 |
ClinicalTrials.gov Identifier: | NCT00104247 |
Health Authority: | United States: Food and Drug Administration |
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