Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old
Recruiting
Phase 1
Interventional Study
Hemophilia B
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 12 – 18
- Sex
- Male
- Trial phase
- Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 9 (estimated)
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China · Other
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
About This Trial
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 circ…
Trial Locations
Loading…
Loading trial locations…
Eligibility Criteria
Inclusion Criteria:
1. Subjects and statutory guardian must be able to understand the purpose and risks of the study and provide signed and dated informed consent;
2. Be male and 12≤ age \<18 years of age, body wight ≥ 50kg;
3. Have hemophilia B with ≤2 IU/dL (≤2 %) endogenous FIX activity levels a…
Contacts