Hemophilia APhase 2/3NCT06111638

Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs

A Phase 2/3 study testing Single dose intravenous injection of BBM-H803 in people with Hemophilia A, run by Shanghai Xinzhi BioMed Co., Ltd. at 10 sites in China.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts

Official record

Search all trials

Trial 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. Read more about this study

Who is it for?: Ages 18 years and older, men only
Conditions the study is for: Hemophilia A
What is being tested?: Single dose intravenous injection of BBM-H803
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 55 participants
Where: 10 sitesChina
Study dates: 3 Jan 2024to 30 May 2031
Phase: Phase 2/3
Are there results?: Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
Men only
No healthy volunteers
Adult, Older adult

Who may be able to join

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
No prior history of hypersensitivity or anaphylaxis associated with any FVIII immunoglobulin;
Use a reliable contraception method during the study;
Capsid antibody negative;
Subjects have good compliance.

Who cannot join

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).
Subjects with medical history of hepatitis B or C can be regarded as negative only when 2 required samplings are conducted at least 3 months apart and both test results of indicators aforementioned are negative, HIV positive patients or Syphilis seropositive patients;
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;

Show all 13Show fewer

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 alpha-fetoprotein was detected by laboratory tests. Subjects who are not eligible for the study if the abnormalities are clinically significant regarding to the medical judgement of the investigator;
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 emesezumab within 6 months before screening; Or drugs evaluated by the researcher to affect the study;
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; Or any Chinese herbal medicine that the researcher evaluates to affect the study;
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 conditions, including poor mental state and risk of suicide, that the investigator deems unsuitable for participation in the study;
Any other conditions that the investigator deems unsuitable for participation in the study.

Trial locations and contacts

10 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Wenqi Shao, Master contact

13636317441

ra@beliefbiomed.com

City, postcode or hospital

Sites recruiting now only

10 sites in 1 country

China10 sites, 10 recruiting

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Beijing, Beijing Municipality, 100005
Tienan Zhu, MD, 18611743292, Zhutn@pumch.cn
Recruiting
Xiangya Hospital Central South University
Changsha, Hunan, 410008
Xielan Zhao, MD, 13707489198, zhaoxl9198@163.com
Recruiting
Sichuan Provincial People's Hospital
Chengde, Sichuan, 610031
Xiaobing Huang, MD, 18981838236, hxb_trial@163.com
Recruiting
Southern Hospital, Southern Medical University
Guangzhou, Guangdong, 510515
Chongyuan Xu, MD, 13926186470, nfyygcp@126.com
Jing Sun, MD, 13316202696, jsun_cn@hotmail.com
Recruiting
Affiliated Hospital of Guizhou Medical University
Guiyang, Guizhou, 550001
Jiao Jin, Bachelor, 15180802411, jinjiao999@163.com
Recruiting
The Second Affiliated Hospital of Kunming Medical University
Kunming, Yunnan, 650032
Zeping Zhou, MD, 18788571605, zhouzeping@kmmu.edu.cn
Recruiting
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215006
Ziqiang Yu, MD, 13913518032, yuziqiang@suda.edu.cn
Recruiting
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
Tianjin, Tianjin Municipality, 300020
Lei Zhang, MD, 0086-22-23909095, zhanglei1@ihcams.ac.cn
Recruiting
Wuhan Union Hospital Affiliated to Huazhong University of Science and Technology
Wuhan, Hubei, 430000
Yu Hu, MD, 13986183871, dr_huyu387@163.com
Recruiting
The Affiliated Hospital of Northwest University Xi'an No.3 Hospital
Xi’an, Shanxi, 710000
Xiequn Chen, MD, 13991832567, cxq@nwu.edu.cn
Recruiting

Other trials in China

About this study

Official title: A Phase 1/2/3 Open-label Study to Evaluate the Safety, Tolerability and Efficacy of an Adeno-associated Viral Vector Containing an Expression Cassette of the Human Factor VIII Transgene (BBM-H803) Injection in Participants With Hemophilia A

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.

Conditions: Hemophilia A
Being tested: Single dose intravenous injection of BBM-H803
Keywords: Hemophilia A, gene therapy, Adeno-Associated Virus
Medical subject headings: Hemophilia A

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Single dose intravenous injection of BBM-H803Genetic
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 groups in NCT06111638
Study group	Role	What they receive
Arm of BBM-H803	Experimental	1×10^13 vg/kg, Single-dose treatment Receives: Genetic: Single dose intravenous injection of BBM-H803

What is being measured

The primary outcome is the main question the study is designed to answer.

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
Measured: 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.
Measured: 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).
Measured: 6 weeks
Phase 3: Annualized bleeding rate (ABR)
To assess ABR, including spontaneous bleeding, traumatic bleeding and joint bleeding after administration.
Measured: 52 weeks

Secondary outcomes (3)

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.
Measured: 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.
Measured: 52 weeks
Phase 1/2/3: 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).
Measured: 52 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

3 Jan 202430 May 2031

First posted1 Nov 2023
Study start3 Jan 2024
Primary completion30 May 2027 (estimated)
Study completion30 May 2031 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Hemophilia AStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2/3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 55 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT06111638
Sponsor's protocol ID: BBM002-CLN1001
Status last verified: Mar 2026
First submitted: 27 Oct 2023
Last updated: 17 Mar 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Interested in taking part?

Wenqi Shao, Master

13636317441

ra@beliefbiomed.com

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See sites and contacts

RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B

All Hemophilia A trials

Study at a glance

From the registry record, last updated by the sponsor on 17 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 1/2/3 Open-label Study to Evaluate the Safety, Tolerability and Efficacy of an Adeno-associated Viral Vector Containing an Expression Cassette of the Human Factor VIII Transgene (BBM-H803) Injection in Participants With Hemophilia A
ClinicalTrials.gov ID: NCT06111638
Acronym / sponsor ID: BBM002-CLN1001
Conditions: Hemophilia A
Keywords: Hemophilia A, gene therapy, Adeno-Associated Virus
Phase: Phase 2/3
Study type: Interventional
Recruitment status: Recruiting
Enrolment: 55 participants (estimated)
Lead sponsor: Shanghai Xinzhi BioMed Co., Ltd.Industry (pharma, biotech, devices)
Collaborators: Shanghai Mianyi Biopharmaceutical Co., Ltd.
Study start: 3 Jan 2024 (actual)
Primary completion: 30 May 2027 (estimated)
Study completion: 30 May 2031 (estimated)
First posted: 1 Nov 2023 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Arm of BBM-H803	Experimental	Genetic: Single dose intravenous injection of BBM-H8031×10^13 vg/kg, Single-dose treatment

Who may qualify

Ages: 18 years and older
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (8 listed)

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
No prior history of hypersensitivity or anaphylaxis associated with any FVIII immunoglobulin;

Key exclusion criteria (13 listed)

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).
Subjects with medical history of hepatitis B or C can be regarded as negative only when 2 required samplings are conducted at least 3 months apart and both test results of indicators aforementioned are negative, HIV positive patients or Syphilis seropositive patients;
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;

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Single dose intravenous injection of BBM-H803Single 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.	Genetic	Arm of BBM-H803

Primary outcome

Phase 1/2: Incidence of dose limiting toxicity (DLT) events
Time frame: 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)
Time frame: 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]
Time frame: 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)
Time frame: 52 weeks
To assess ABR, including spontaneous bleeding, traumatic bleeding and joint bleeding after administration.

3 secondary outcomes

Phase 1/2/3: Plasma FVIII activity level
Time frame: 52 weeks
Phase 1/2/3: The incidence of adverse events (AEs) and serious adverse events (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]
Time frame: 52 weeks

Results and safety

This study is still under way. Results are not available in the source record yet.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 2026.
Individual participant dataSharing planned: No

Related trials for Hemophilia A

RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.

Wenqi Shao, Master contact

13636317441

ra@beliefbiomed.com

Lei Zhang, MD study chair

Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College

10 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Single dose intravenous injection of BBM-H803 in people with Hemophilia A, run by Shanghai Xinzhi BioMed Co., Ltd. at 10 sites in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 17 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 10 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Wenqi Shao, Master (13636317441, ra@beliefbiomed.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Phase 1/2: Incidence of dose limiting toxicity (DLT) events (time frame: 6 weeks). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06111638.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Mar 2026.