A Clinical Study of AAV Vector Expressing Human Coagulation Factor FVIII Gene Therapy for Hemophilia A
This is a single-arm, open-label, clinical study to evaluate the safety, tolerability of BBM 002 injection in Hemophilia A subjects with residual factor VIII (FVIII) levels ≤2 International unit per deciliter (IU/dl) . BBM 002 injection is an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII (hFVIII) transgene and raises circulating levels of endogenous FVIII.
• Subjects are fully aware of the purpose, nature, methods and possible adverse reactions of the trial and voluntarily sign informed consent.
• Males ≥ 18 years of age.
• Have hemophilia A with ≤2 IU/dL (≤2 %) endogenous FVIII activity levels.
• Have had ≥150 prior exposure days (EDs) to any recombinant and/or plasma-derived FVIII protein products.
• Have had bleeding events and/or infusions with FVIII protein products (including recombination and plasma source) during the last 12 weeks documented in the subjects' medical records.
• Have no prior history of hypersensitivity or anaphylaxis associated with any FVIII or IV immunoglobulin administration.
• Have no FVIII inhibitor. (eg \<0.6BU/ml Bethesda Units; or the patient's FVIII inhibitor titer was detected \<0.6BU/ml in 2 consecutive times within 1-4 weeks using Bethesda method or Nijmegen method), or no prior medical history of FVIII inhibitor after 150 EDs of FVIII products; no clinical signs or symptoms of decreased response to FVIII products infusion.
• Agree to use a reliable barrier contraception method from the beginning of signing the informed consent to 52 weeks after BBM002 infusion.
• Compliance is good, patients and their families have the will of 'gene therapy' clinical trials.