Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old
ConditionHemophilia B
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexMale
Age12-18
SponsorInstitute of Hematology & Blood Diseases Hospital, China
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 circulating levels of endogenous FIX.
Eligibility criteria
Qualifiers
Subjects and statutory guardian must be able to understand the purpose and risks of the study and provide signed and dated informed consent;
Be male and 12≤ age <18 years of age, body wight ≥ 50kg;
Have hemophilia B with ≤2 IU/dL (≤2 %) endogenous FIX activity levels as documented by a certified clinical laboratory at the time of screening. If the screening result is >2% due to insufficient washout from FIX protein product, then the severity of hemophilia B may be confirmed by documented historical evidence from a certified clinical laboratory demonstrating ≤2% FIX coagulant activity (FIX:C) ;
Had had ≥75 prior exposure days (EDs) to any recombinant and/or plasma-derived FIX protein products based on historical data from the subject's record/history;
Disqualifiers
Hepatitis B surface antigen antibody (HBSAg-Ab) or HBV-DNA positive; hepatitis C antibody or HCV-RNA positive;
Currently on antiviral therapy for hepatitis B or C;
With coagulation disorders other than hemophilia B;
Had immunosuppressive therapy other than steroid and other suggested IST agents within 30 days prior to screening;
Trial design
Treatments tested in this trial
- BBM-H901
Treatment groups
9 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov