Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old

Recruiting now · Phase 1

Conditions studied: Hemophilia B

In brief

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.

Key facts

Study ID: NCT05709288
Run by: Institute of Hematology & Blood Diseases Hospital, China
People needed: 9
Starts: 2023-03-23
Expected to finish: 2035-11-01
Last updated by the study team: 2025-02-21

Who can join

Age: 12 and older, up to 18. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

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;
With ≤ 1:4 neutralizing antibodies and ≤1:200 binding antibodies against BBM-H901 capsid;
Subjects with bleeding episode and/ or FIX agents infusion events within 12 weeks prior to screening;
Have no prior history of hypersensitivity or anaphylaxis associated with any FIX or IV immunoglobulin administration;
Have no measurable FIX inhibitor as assessed by laboratory; or documented no prior history of FIX inhibitor (family history of inhibitors will not exclude the subject) and no clinical signs or symptoms of decreased response to FIX administration;
Have acceptable laboratory values:
Hemoglobin ≥11 g/dL ;
Platelets ≥100,000 cells/μL;
AST, ALT ≤1.5x upper limit of normal at the testing laboratory;
Bilirubin ≤1.5x ULN ;
glomerular filtration rate eGFR ≥ 60ml/min.
For those subjects with sexual maturity, subject and statutory guardian must know that subjects must agree to use reliable barrier contraception until 52 weeks;
with good compliance to the schedule of visit and fill in the subject diary.

You may not qualify if…

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;
Had vaccine 30 days prior to screening or have scheduled vaccination plan during the study (up to 52 weeks);
Have significant underlying liver disease, as defined by a preexisting diagnosis of portal hypertension, splenomegaly, encephalopathy, etc; other liver conditions unsuitable to gene therapy judged by investigator;
Have surgery plan within 52 weeks after gene therapy;
Have history of chronic infection or high rish of infection that the Investigator considers to constitute an unacceptable risk;
Had participated in a previous gene therapy research trial within the last 52 weeks or in a clinical study with an investigational drug within the last 12 weeks;
Had any herb that may affect the liver function within 4 weeks prior to screening;
Have history of fatal bleeding episode, eg intracranial hemorrhage, etc;
Any concurrent clinically significant major disease or any other condition that, in the opinion of the Investigator, makes the subject unsuitable for participation in the study;

Where it is running

Institute of haematology and Blood diseases hospital — Tianjin, Tianjin Municipality, China (enrolling)

Full record on ClinicalTrials.gov

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