Kuvan® in Phenylketonuria Patients Less Than 4 Years Old
Status:
Completed
Trial end date:
2017-02-17
Target enrollment:
Participant gender:
Summary
This is a Phase 3b, multicenter, open-label, randomized, controlled study to evaluate
efficacy, safety and population pharmacokinetics of sapropterin dihydrochloride (Kuvan®) in
less than 4 year-old infants and children with phenylketonuria (PKU).