Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs
Recruiting
Phase 2
Phase 3
Interventional Study
Hemophilia A
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 18 and older
- Sex
- Male
- Trial phase
- Phase 2/3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 55 (estimated)
- Sponsor
- Shanghai Xinzhi BioMed Co., Ltd. · Industry
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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 end…
Trial Locations
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Eligibility Criteria
Inclusion Criteria of Phase 1/2/3:
1. Subjects voluntarily sign informed consent form;
2. Males ≥ 18 years;
3. Subjects are clinically diagnosed with severe hemophilia A;
4. Have \> = 150 documented exposure days to a Factor VIII protein product
5. No prior history of hypersensitivity or anaphylaxi…
Contacts