Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old
Status:
Not yet recruiting
Trial end date:
2035-11-01
Target enrollment:
Participant gender:
Summary
This is a Phase 1, open- label, non- randomized, uncontrolled, single dose pilot study to
evaluate the safety, tolerability and efficacy of a single intravenous infusion of BBM-H901
in hemophilia B subjects with ≤2IU/dl residual FIX levels and aged 12-18 years old. BBM-H901
is an adeno-associated viral (AAV) vector designed to drive expression of the human factor IX
(hFIX) transgene and raise circulating levels of endogenous FIX.