Sapropterin Expanded Access Program
NCT00484991 · Status: APPROVED_FOR_MARKETING · Type: EXPANDED_ACCESS
Last updated 2008-04-15
Summary
The Purpose of this study is to provide patients with hyperphenylalaninemia (HPA) due to Phenylketonuria (PKU) access to sapropterin dihydrochloride and to collect more information about the safety of the drug in an expanded access program (EAP) until commercial product is available.
Conditions
Interventions
- DRUG
-
Sapropterin dihydrochloride
20mg/kg/day orally, physicians may adjust the dose within a range of 5-20mg/kg/day as warranted by their clinical judgment.
Sponsors & Collaborators
- lead INDUSTRY
Eligibility
- Min Age
- 9 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
Countries
- United States
Study Locations
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