Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs
ConditionHemophilia A
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexMale
Age18+
SponsorShanghai Xinzhi BioMed Co., Ltd.
About this trial
This is a multi-center, Phase 1/2/3, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H803 injection in severe Hemophilia A subjects.
BBM-H803 is an adeno-associated viral (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII transgene and raises circulating levels of endogenous FVIII.
Eligibility criteria
Qualifiers
Subjects voluntarily sign informed consent form;
Males ≥ 18 years;
Subjects are clinically diagnosed with severe hemophilia A;
Have > = 150 documented exposure days to a Factor VIII protein product
Disqualifiers
Being positive for hepatitis B surface antigen (HBsAg) or hepatitis B virus-DNA (HBV-DNA). Being positive for hepatitis C virus antibody (HCV-Ab) or hepatitis C virus RNA (HCV-RNA).
Currently on antiviral therapy for hepatitis B or C;
Suffer from coagulation disorders other than hemophilia A;
In addition to glucocorticoids, any other immunosuppressants are being used before selection;
Trial design
Treatments tested in this trial
- Single dose intravenous injection of BBM-H803
Treatment groups
55 Participants
are divided into 1 treatment groupSponsors and collaborators
Shanghai Xinzhi BioMed Co., Ltd.
Lead sponsor
Shanghai Mianyi Biopharmaceutical Co., Ltd.
Collaborator
Source ClinicalTrials.gov