Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency
Status:
Completed
Trial end date:
2009-06-01
Target enrollment:
Participant gender:
Summary
The purpose of this study is to evaluate the ability of Phenoptin to control blood
phenylalanine levels in subjects who have hyperphenylalaninemia due to a primary BH4
deficiency and to evaluate the safety of Phenoptin in this population. Some subjects were
receiving non-registered formulations of BH4 at enrollment and this treatment was suspended
after Part 1 and within one day the subjects started Phenoptin at approximately the same
dose.