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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
55
Single dose intravenous infusion of BBM-H803, an adeno-associated viral (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII transgene in liver. The dose of BBM-H803 will be calculated according to participant's weight.
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Beijing, Beijing Municipality, China
RECRUITINGSouthern Hospital, Southern Medical University
Guangzhou, Guangdong, China
Phase 1/2: Incidence of dose limiting toxicity (DLT) events
To access the numbers of DLT events determined by the Safety Data Review Committee (SRC) in DLT observation period after BBM-H803 injection infusion
Time frame: 6 weeks
Phase 1/2: The incidence of adverse events (AEs) and serious adverse events (SAEs)
To assess the safety of BBM-H803 Injection by AEs and SAEs.
Time frame: 6 weeks
Phase 1/2: Number of participants with changes in alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels [liver function]
To assess changes in liver function before and after treatment, including alanine aminotransferase (ALT) and aspartate aminotransferase (AST).
Time frame: 6 weeks
Phase 3: Annualized bleeding rate (ABR)
To assess ABR, including spontaneous bleeding, traumatic bleeding and joint bleeding after administration.
Time frame: 52 weeks
Phase 1/2/3: Plasma FVIII activity level
All samples collected from participants for plasma FVIII activity levels were analyzed and used to determine peak and steady-state vector-derived circulating FVIII activity levels.
Time frame: 52 weeks
Phase 1/2/3: The incidence of adverse events (AEs) and serious adverse events (SAEs)
To assess the safety of BBM-H803 Injection by AEs and SAEs.
Time frame: 52 weeks
Phase 1/2/3: Number of participants with changes in alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels [liver function]
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Affiliated Hospital of Guizhou Medical University
Guiyang, Guizhou, China
RECRUITINGWuhan Union Hospital Affiliated to Huazhong University of Science and Technology
Wuhan, Hubei, China
RECRUITINGXiangya Hospital Central South University
Changsha, Hunan, China
RECRUITINGThe First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, China
RECRUITINGThe Affiliated Hospital of Northwest University Xi'an No.3 Hospital
Xi’an, Shanxi, China
RECRUITINGSichuan Provincial People's Hospital
Chengde, Sichuan, China
RECRUITINGInstitute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
Tianjin, Tianjin Municipality, China
RECRUITINGThe Second Affiliated Hospital of Kunming Medical University
Kunming, Yunnan, China
RECRUITINGTo assess changes in liver function before and after treatment, including alanine aminotransferase (ALT)and aspartate aminotransferase (AST).
Time frame: 52 weeks