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Tests treatment safety and results for Hemophilia A
Official title Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs
ClinicalTrials.gov ID: NCT06111638
What this study is testing
What is Single dose intravenous injection of BBM-H803?
Single dose intravenous injection of BBM-H803 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for hemophilia a.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older, men only
You may be able to join if
- of Phase 1/2/3:
- people voluntarily sign informed consent form;
- Males ≥ 18 years;
- people are clinically diagnosed with severe hemophilia A;
- Have \> = 150 documented exposure days to a Factor VIII protein product
You likely can't join if
- of Phase 1/2/3:
- 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...
- 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;
- Have vaccination history within 2 months before administration, or vaccination schedule during immunomodulatory therapy;
See the full eligibility criteria
- of Phase 1/2/3:
- people voluntarily sign informed consent form;
- Males ≥ 18 years;
- people are clinically diagnosed with severe hemophilia A;
- Have \> = 150 documented exposure days to a Factor VIII protein product
- No prior history of hypersensitivity or anaphylaxis associated with any FVIII immunoglobulin;
- Use a reliable contraception method during the study;
- Capsid antibody negative;
- people have good compliance.
- of Phase 1/2/3:
- 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). people with medical history of...
- 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;
- Have vaccination history within 2 months before administration, or vaccination schedule during immunomodulatory therapy;
- Have potential liver diseases, such as previous diagnosis of portal hypertension, splenomegaly, hepatic encephalopathy or liver fibrosis (fibrosis stage ≥ 3); nodules or cysts were found by B ultrasound, or elevated...
- Scheduling of elective major surgery known or planned during the insertion period or the 52-week study period following BBM-H803 infusion;
- Have participated in a previous gene therapy research trial before screening or have used other test drugs within 4 weeks before screening, or within 5 half-life of the test drug, whichever is longer; Have received...
- Any herbal preparations (herbal supplements or traditional Chinese medicines of plant, mineral or animal origin) used during the 4 weeks prior to or during the study that may affect liver function, except topical use...
- Have alcohol or drug addiction, or cannot stop drinking as advised by the researcher throughout the study;
- Have acute/chronic infections or other chronic diseases that may pose a risk for the study, or have a major illness, who have a current or previous history of malignant tumors, or who have other unstable medical...
- Any other conditions that the investigator deems unsuitable for participation in the study.
The study team makes the final eligibility decision.
Where it's taking place
- Beijing, Beijing Municipality, China
- Guangzhou, Guangdong, China
- Guiyang, Guizhou, China
- Wuhan, Hubei, China
- Changsha, Hunan, China
- Suzhou, Jiangsu, China
- Xi’an, Shanxi, China
- Chengde, Sichuan, China
- Tianjin, Tianjin Municipality, China
- Kunming, Yunnan, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Beijing, Beijing Municipality, China; Guangzhou, Guangdong, China; Guiyang, Guizhou, China; Wuhan, Hubei, China; Changsha, Hunan, China; Suzhou, Jiangsu, China and 4 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.