Institute of Hematology & Blood Diseases Hospital, China
Zhang lei, MD
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.
Hemophilia B
BBM-H901
Phase 1
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. Nine subjects will be enrolled and administered with single infusion of BBM-H901, an AAV at one dose level of 5x10'12 vg/Kg. Subjects and statutory guardian must provide informed consent and then undergo screening assessments up to 4-8weeks prior administration of BBM-H901. All subjects will undergo 52(+- 2) weeks safety observation and will be continuously followed up to evaluate long- term safety and efficacy of BBM-H901 up to ten years. The first subject will be dosed at 5x10'12 vg/Kg and undergo 8 weeks safety observation of which the data will undergo review by an independent safety committee.}}
Study Type : | Interventional |
Estimated Enrollment : | 9 participants |
Masking : | None (Open Label) |
Primary Purpose : | Treatment |
Official Title : | A Pilot Study Evaluating the Safety, Tolerability and Efficacy of Gene Therapy With BBM-H901 in Hemophilia B Patients Aged 12-18 Years Old |
Actual Study Start Date : | March 23, 2023 |
Estimated Primary Completion Date : | June 2035 |
Estimated Study Completion Date : | November 2035 |
Arm | Intervention/treatment |
---|---|
Experimental: BBM-H901 administration group Subjects will be administered with single dose intravenous infusion of BBM-H901. |
Drug: BBM-H901 |
Ages Eligible for Study: | 12 Years to 18 Years |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.
Recruiting
Institute of haematology and Blood diseases hospital
Tianjin, Tianjin, China, 300020