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Sponsored by: |
BioMarin Pharmaceutical |
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Information provided by: | BioMarin Pharmaceutical |
ClinicalTrials.gov Identifier: | NCT00445978 |
This Phase 2a, multicenter, open-label, dose-escalation study is designed to assess the safety and biologic activity of daily oral administration of 4 escalating doses of 6R-BH4 over 16 weeks in subjects with sickle cell disease. During an optional extension phase, the study will assess the safety, tolerability, and efficacy of extended treatment with 6R-BH4, for a total of up to 2 years.
Condition | Intervention | Phase |
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Sickle Cell Disease |
Drug: 6R-BH4 (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 2a, Multicenter, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of 6R-BH4 in Subjects With Sickle Cell Disease |
Estimated Enrollment: | 40 |
Study Start Date: | April 2007 |
Estimated Primary Completion Date: | June 2008 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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6R-BH4: Experimental
2.5, 5, 10, 20 mg/kg/day of 6R-BH4 during a 16-week dose escalation phase, with dose levels increasing within subjects every 4 weeks, with an optional extension phase at the highest tolerated dose for up to a total of 2 years.
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Drug: 6R-BH4 (sapropterin dihydrochloride)
Subjects will receive oral, once-daily (for 2.5, 5, 10mg/kg/day doses) or twice-daily (for the 20 mg/kg/day dose) of 6R-BH4 during a 16-week dose escalation phase, with dose levels increasing within subjects every 4 weeks as follows: 2.5, 5, 10, and 20 mg/kg/day. Subjects may continue in an optional extension phase at the highest tolerated dose for up to a total of 2 years.
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Ages Eligible for Study: | 15 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, District of Columbia | |
Washington, District of Columbia, United States | |
United States, Georgia | |
Augusta, Georgia, United States | |
Savannah, Georgia, United States | |
United States, Indiana | |
Indianapolis, Indiana, United States | |
United States, Michigan | |
Flint, Michigan, United States | |
Detroit, Michigan, United States | |
United States, New Jersey | |
Hackensack, New Jersey, United States | |
United States, North Carolina | |
Chapel Hill, North Carolina, United States | |
United States, Pennsylvania | |
Philadelphia, Pennsylvania, United States | |
United States, Texas | |
Galveston, Texas, United States | |
United States, Virginia | |
Richmond, Virginia, United States | |
Norfolk, Virginia, United States |
Study Director: | Don Nwose, MD | BioMarin Pharmaceutical |
Responsible Party: | BioMarin Pharmaceutical Inc. ( BioMarin Pharmaceutical Inc. ) |
Study ID Numbers: | SCD-001 |
Study First Received: | March 7, 2007 |
Last Updated: | July 22, 2008 |
ClinicalTrials.gov Identifier: | NCT00445978 History of Changes |
Health Authority: | United States: Food and Drug Administration |
Sickle Cell Disease SCD 6R-BH4 BH4 |
sapropterin dihydrochloride endothelial dysfunction Nitric Oxide NO |
Nitric Oxide Anemia, Hemolytic, Congenital Genetic Diseases, Inborn Hematologic Diseases Hemoglobinopathies |
Anemia Sickle Cell Anemia Anemia, Hemolytic Hemoglobinopathy Anemia, Sickle Cell |
Anemia, Hemolytic, Congenital Genetic Diseases, Inborn Hematologic Diseases Hemoglobinopathies |
Anemia Anemia, Hemolytic Anemia, Sickle Cell |