drugset / Trial / NCT00484991
Sapropterin Expanded Access Program
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.
Timeline
- Start
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- Primary completion
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- Completion
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Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | sapropterin | Unknown | 20 mg/kg | Oral |