A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease
Status:
Completed
Trial end date:
2009-06-01
Target enrollment:
Participant gender:
Summary
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
sapropterin dihydrochloride 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 sapropterin dihydrochloride, for a total of up to 2 years; The
extension phase of this study was terminated.