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Sponsored by: |
BioMarin Pharmaceutical |
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Information provided by: | BioMarin Pharmaceutical |
ClinicalTrials.gov Identifier: | NCT00104260 |
The primary objective is to evaluate the degree and frequency of response to Phenoptin™ (sapropterin dihydrochloride), as demonstrated by a reduction in blood phenylalanine (Phe) level among subjects with phenylketonuria (PKU) who have elevated Phe levels. A secondary objective of this study is to evaluate the safety of Phenoptin™ treatment in this subject population, and identify individuals in this subject population who respond to Phenoptin™ treatment with a reduction in blood Phe level.
Condition | Intervention | Phase |
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Phenylketonurias |
Drug: sapropterin dihydrochloride |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels |
Estimated Enrollment: | 700 |
Study Start Date: | December 2004 |
Study Completion Date: | November 2005 |
Ages Eligible for Study: | 8 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, California | |
Oakland, California, United States | |
Los Angeles, 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 |
Study ID Numbers: | PKU-001 |
Study First Received: | February 24, 2005 |
Last Updated: | April 5, 2007 |
ClinicalTrials.gov Identifier: | NCT00104260 |
Health Authority: | United States: Food and Drug Administration |
Metabolism, Inborn Errors Inborn amino acid metabolism disorder Metabolic Diseases Genetic Diseases, Inborn Amino Acid Metabolism, Inborn Errors Central Nervous System Diseases |
Brain Diseases, Metabolic, Inborn Phenylketonurias Metabolic disorder Brain Diseases Phenylketonuria Brain Diseases, Metabolic |
Nervous System Diseases |