Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs
Condition(s) studied
Investigational drug(s) / intervention(s)
Single dose intravenous injection of BBM-H803: 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.
Study summary
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
Primary outcome measure(s)
- Phase 1/2: Incidence of dose limiting toxicity (DLT) events — 6 weeks
To access the numbers of DLT events determined by the Safety Data Review Committee (SRC) in DLT observation period after BBM-H803 injection infusion - Phase 1/2: The incidence of adverse events (AEs) and serious adverse events (SAEs) — 6 weeks
To assess the safety of BBM-H803 Injection by AEs and SAEs. - Phase 1/2: Number of participants with changes in alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels [liver function] — 6 weeks
To assess changes in liver function before and after treatment, including alanine aminotransferase (ALT) and aspartate aminotransferase (AST). - Phase 3: Annualized bleeding rate (ABR) — 52 weeks
To assess ABR, including spontaneous bleeding, traumatic bleeding and joint bleeding after administration.
Trial sites (10)
| Facility | City | Region | Status |
|---|---|---|---|
| Peking Union Medical College Hospital, Chinese Academy of Medical Sciences | Beijing | Beijing Municipality | Recruiting |
| Southern Hospital, Southern Medical University | Guangzhou | Guangdong | Recruiting |
| Affiliated Hospital of Guizhou Medical University | Guiyang | Guizhou | Recruiting |
| Wuhan Union Hospital Affiliated to Huazhong University of Science and Technology | Wuhan | Hubei | Recruiting |
| Xiangya Hospital Central South University | Changsha | Hunan | Recruiting |
| The First Affiliated Hospital of Soochow University | Suzhou | Jiangsu | Recruiting |
| The Affiliated Hospital of Northwest University Xi'an No.3 Hospital | Xi’an | Shanxi | Recruiting |
| Sichuan Provincial People's Hospital | Chengde | Sichuan | Recruiting |
| Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College | Tianjin | Tianjin Municipality | Recruiting |
| The Second Affiliated Hospital of Kunming Medical University | Kunming | Yunnan | Recruiting |
More Shanghai Xinzhi BioMed Co., Ltd. trials in China
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06111638 on ClinicalTrials.gov ↗ ← All trials in China