Hemophilia BPhase 2/3NCT05203679

Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug

A Phase 2/3 study testing Single dose intravenous injection of BBM-H901 in people with Hemophilia B, run by Shanghai Xinzhi BioMed Co., Ltd. at 9 sites in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

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-H901 injection in Hemophilia B subjects with ≤2 International unit per deciliter (IU/dl) residual factor IX (FIX) levels. Read more about this study

Who is it for?: Ages 18 years and older, men only
Conditions the study is for: Hemophilia B
What is being tested?: Single dose intravenous injection of BBM-H901
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 32 participants
Where: 9 sitesChina
Study dates: 30 Dec 2021to 30 Jun 2028
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

Males ≥ 18 years of age;
Have hemophilia B with ≤2 IU/dL (≤2 %) endogenous FIX activity levels;
Have had ≥100 prior exposure days (EDs) to any recombinant and/or plasma-derived FIX protein products based on historical data from the subjects' records/histories;
Have had bleeding events and/or injected with FIX protein products (including recombination and plasma source) during the last 12 weeks documented in the subjects' medical records;
Have no prior history of hypersensitivity or anaphylaxis associated with any FIX or IV immunoglobulin administration;
Agree to use a reliable barrier contraception method from the beginning of signing the informed consent to 52 weeks after administration.

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, i.e. subjects with natural clearance and anti-viral therapy clearance for hepatitis B or C are eligible;
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;
HIV positive patients;
Have participated in a previous gene therapy research trial before screening, or in a clinical study with an investigational drug within 5 half-life of the investigational product, whichever is longer;
Have alcohol or drug dependence, or cannot stop drinking throughout the study;
Any concurrent clinically significant major disease or condition that the investigator deems unsuitable for participation in the study.

Trial locations and contacts

9 sites in 1 country.

City, postcode or hospital

9 sites in 1 country

China9 sites

Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100005
Nanfang Hospital Southern Medical University
Guangzhou, Guangdong, 510515
Anhui Provincial Hospital
Hefei, Anhui, 230022
The second Affiliated Hospital of Kunming Medical University
Kunming, Yunnan, 650032
The Second People's Hospital of Shenzhen
Shenzhen, Guangdong, 518025
The Second Hospital of Shanxi Medical University
Taiyuan, Shanxi, 030001
North China University of Science and Technology Affiliated Hospital
Tangshan, Hebei, 063000
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
Tianjin, Tianjin Municipality, 300020
Henan Cancer Hospital
Zhengzhou, Henan, 450003

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 Virus Vector Containing an Expression Cassette of the Human Factor IX Transgene (BBM-H901) Injection in Patients With Hemophilia B

BBM-H901 is an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor IX (hFIX) transgene and raises circulating levels of endogenous FIX.

Conditions: Hemophilia B
Being tested: Single dose intravenous injection of BBM-H901
Keywords: Hemophilia B;gene therapy;Adeno-Associated Virus
Medical subject headings: Hemophilia B

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-H901Genetic
Single dose intravenous infusion of BBM-H901, an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor IX (hFIX) transgene in liver.

Study groups in NCT05203679
Study group	Role	What they receive
Arm of BBM-H901	Experimental	Single-dose treatment Receives: Genetic: Single dose intravenous injection of BBM-H901

What is being measured

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

Phase 1/2: The 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-H901 injection infusion.
Measured: 10 weeks post-infusion
Phase 1/2: The incidence of adverse events (AEs) and serious adverse events (SAEs)
To assess the safety of BBM-H901 Injection by AEs and SAEs.
Measured: 10 weeks post-infusion
Phase 1/2: Changes in liver function
To assess changes in liver function before and after treatment, including alanine aminotransferase (ALT) and aspartate aminotransferase (AST).
Measured: 10 weeks post-infusion
Phase 3: Annualized bleeding rate (ABR)
To assess ABR, including spontaneous bleeding and traumatic bleeding after administration.
Measured: 52 weeks post-infusion

Secondary outcomes (9)

Phase 1/2: Changes in liver function
To assess changes in liver function before and after treatment, including alanine aminotransferase (ALT) and aspartate aminotransferase (AST).
Measured: 52 weeks post-infusion
Phase 1/2/3: Mean FIX Padua Activity Level
Measurement of mean FIX Padua activity levels over the 52-week period following BBM-H901 injection.
Measured: 52 weeks post-infusion
Phase 1/2/3: Other FIX Protein Product Usage
Number and total volume of infusions of exogenous FIX protein products (recombinant or plasma-derived) administered within 52 weeks post-BBM-H901 injection.
Measured: 52 weeks post-infusion
Phase 1/2/3: Target Joint Count
Number of target joints recorded within 52 weeks post-BBM-H901 injection.
Measured: 52 weeks post-infusion
Phase 1/2/3: Joint Bleeding Episodes
Total number of joint bleeding events occurring within 52 weeks post-BBM-H901 injection.
Measured: 52 weeks post-infusion
Phase 1/2/3: Bleeding-Free Subjects
Proportion of subjects experiencing no bleeding events within 52 weeks post-BBM-H901 injection.
Measured: 52 weeks post-infusion
Phase 1/2/3: Adverse Event Incidence
Incidence of adverse events (AEs) and serious adverse events (SAEs) within 52 weeks post-BBM-H901 injection.
Measured: 52 weeks post-infusion
Phase 1/2/3: FIX Inhibitor Incidence
Incidence of FIX inhibitors measured by Bethesda or Nijmegen-Bethesda assays within 52 weeks post-BBM-H901 injection.
Measured: 52 weeks post-infusion
Phase 1/2/3: AAV Vector Shedding
Changes in AAV vector shedding in plasma, urine, semen, saliva, and PBMCs within 52 weeks post-BBM-H901 injection.
Measured: 52 weeks post-infusion

Timeline

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

30 Dec 202130 Jun 2028

First posted24 Jan 2022
Study start30 Dec 2021
Primary completion16 Apr 2024
Study completion30 Jun 2028 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (5)Papers the sponsor has linked to this study.
Other trials for Hemophilia BStudies 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: 32 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05203679
Sponsor's protocol ID: BBM001-CLN1001
Status last verified: Mar 2026
First submitted: 29 Dec 2021
Last updated: 1 Apr 2026
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Publications

Xue F, Li H, Wu X, Liu W, Zhang F, Tang D, Chen Y, Wang W, Chi Y, Zheng J, Du Z, Jiang W, Zhong C, Wei J, Zhu P, Fu R, Liu X, Chen L, Pei X, Sun J, Cheng T, Yang R, Xiao X, Zhang L. Safety and activity of an engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX administered with prophylactic glucocorticoids in patients with haemophilia B: a single-centre, single-arm, phase 1, pilot trial. Lancet Haematol. 2022 Jul;9(7):e504-e513. doi: 10.1016/S2352-3026(22)00113-2. Epub 2022 May 19.
PubMed 35598604
Xue F, Wang P, Yuan Z, Shi C, Fang Y, Liu W, Wang Y, Xiao X, Yang R, George LA, Zhang L. Total Knee Arthroplasty after Gene Therapy for Hemophilia B. N Engl J Med. 2022 Oct 27;387(17):1622-1624. doi: 10.1056/NEJMc2211173. No abstract available.
PubMed 36306204
Wei H, Xiao W, Dai J. China's first approved gene therapy for hemophilia B: A new era for global AAV-based treatments. Mol Ther. 2025 Jun 4;33(6):2312-2313. doi: 10.1016/j.ymthe.2025.05.014. Epub 2025 May 28. No abstract available.
PubMed 40441148
Xue F, Hu Y, Yang R, Sun J, Yang L, Wang X, Yu Z, Zhu T, Zhou H, Zhou Z, Yan Z, Du X, Zheng C, Liu W, Zhou R, Dai J, Yin J, Wang H, Tang LV, Chen S, Cheng H, Zhu M, Wang S, Song Y, Zhang P, Zhang L. Chinese guidance for the clinical application of adeno-associated virus vector-based gene therapy for hemophilia B (2025). Blood Sci. 2025 Oct 29;7(4):e00257. doi: 10.1097/BS9.0000000000000257. eCollection 2025 Dec.
PubMed 41180128
Xue F, Ju M, Zhu T, Zhou Z, Sun J, Yang L, Yan Z, Zhou H, Du X, Zheng C, Zheng J, Wu X, Du Z, Jiang W, Yang C, Xiao X, Liu W, Yang R, Zhang L. Factor IX-Padua AAV gene therapy in hemophilia B: phases 1/2 and 3 trials. Nat Med. 2026 Jan;32(1):93-102. doi: 10.1038/s41591-025-04012-y. Epub 2025 Nov 20.
PubMed 41266685

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

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 locations

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
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
Not yet recruitingPhase 1
Lentiviral FIX Gene Therapy
Hemophilia B
RecruitingPhase 3
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
Hemophilia

All Hemophilia B trials

Study at a glance

From the registry record, last updated by the sponsor on 1 Apr 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 Virus Vector Containing an Expression Cassette of the Human Factor IX Transgene (BBM-H901) Injection in Patients With Hemophilia B
ClinicalTrials.gov ID: NCT05203679
Acronym / sponsor ID: BBM001-CLN1001
Conditions: Hemophilia B
Keywords: Hemophilia B;gene therapy;Adeno-Associated Virus
Phase: Phase 2/3
Study type: Interventional
Recruitment status: Active, not recruiting
Enrolment: 32 participants (actual)
Lead sponsor: Shanghai Xinzhi BioMed Co., Ltd.Industry (pharma, biotech, devices)
Study start: 30 Dec 2021 (actual)
Primary completion: 16 Apr 2024 (actual)
Study completion: 30 Jun 2028 (estimated)
First posted: 24 Jan 2022 (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-H901	Experimental	Genetic: Single dose intravenous injection of BBM-H901Single-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 (6 listed)

Males ≥ 18 years of age;
Have hemophilia B with ≤2 IU/dL (≤2 %) endogenous FIX activity levels;
Have had ≥100 prior exposure days (EDs) to any recombinant and/or plasma-derived FIX protein products based on historical data from the subjects' records/histories;
Have had bleeding events and/or injected with FIX protein products (including recombination and plasma source) during the last 12 weeks documented in the subjects' medical records;
Have no prior history of hypersensitivity or anaphylaxis associated with any FIX or IV immunoglobulin administration;

Key exclusion criteria (6 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, i.e. subjects with natural clearance and anti-viral therapy clearance for hepatitis B or C are eligible;
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;
HIV positive patients;
Have participated in a previous gene therapy research trial before screening, or in a clinical study with an investigational drug within 5 half-life of the investigational product, whichever is longer;
Have alcohol or drug dependence, or cannot stop drinking throughout the study;

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-H901Single dose intravenous infusion of BBM-H901, an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor IX (hFIX) transgene in liver.	Genetic	Arm of BBM-H901

Primary outcome

Phase 1/2: The incidence of dose limiting toxicity (DLT) events
Time frame: 10 weeks post-infusion
To access the numbers of DLT events determined by the Safety Data Review Committee (SRC) in DLT observation period after BBM-H901 injection infusion.
Phase 1/2: The incidence of adverse events (AEs) and serious adverse events (SAEs)
Time frame: 10 weeks post-infusion
To assess the safety of BBM-H901 Injection by AEs and SAEs.
Phase 1/2: Changes in liver function
Time frame: 10 weeks post-infusion
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 post-infusion
To assess ABR, including spontaneous bleeding and traumatic bleeding after administration.

9 secondary outcomes

Phase 1/2: Changes in liver function
Time frame: 52 weeks post-infusion
Phase 1/2/3: Mean FIX Padua Activity Level
Time frame: 52 weeks post-infusion
Phase 1/2/3: Other FIX Protein Product Usage
Time frame: 52 weeks post-infusion
Phase 1/2/3: Target Joint Count
Time frame: 52 weeks post-infusion
Phase 1/2/3: Joint Bleeding Episodes
Time frame: 52 weeks post-infusion
Phase 1/2/3: Bleeding-Free Subjects
Time frame: 52 weeks post-infusion
Phase 1/2/3: Adverse Event Incidence
Time frame: 52 weeks post-infusion
Phase 1/2/3: FIX Inhibitor Incidence
Time frame: 52 weeks post-infusion
Phase 1/2/3: AAV Vector Shedding
Time frame: 52 weeks post-infusion

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 1 Apr 2026.
Background: PubMed 35598604Xue F, Li H, Wu X, Liu W, Zhang F, Tang D, Chen Y, Wang W, Chi Y, Zheng J, Du Z, Jiang W, Zhong C, Wei J, Zhu P, Fu R, Liu X, Chen L, Pei X, Sun J, Cheng T, Yang R, Xiao X, Zhang L. Safety and activity of an engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX administered with prophylactic glucocorticoids in patients with haemophilia B: a single-centre, single-arm, phase 1, pilot trial. Lancet Haematol. 2022 Jul;9(7):e504-e513. doi: 10.1016/S2352-3026(22)00113-2. Epub 2022 May 19.
Background: PubMed 36306204Xue F, Wang P, Yuan Z, Shi C, Fang Y, Liu W, Wang Y, Xiao X, Yang R, George LA, Zhang L. Total Knee Arthroplasty after Gene Therapy for Hemophilia B. N Engl J Med. 2022 Oct 27;387(17):1622-1624. doi: 10.1056/NEJMc2211173. No abstract available.
Results publication: PubMed 40441148Wei H, Xiao W, Dai J. China's first approved gene therapy for hemophilia B: A new era for global AAV-based treatments. Mol Ther. 2025 Jun 4;33(6):2312-2313. doi: 10.1016/j.ymthe.2025.05.014. Epub 2025 May 28. No abstract available.
Results publication: PubMed 41180128Xue F, Hu Y, Yang R, Sun J, Yang L, Wang X, Yu Z, Zhu T, Zhou H, Zhou Z, Yan Z, Du X, Zheng C, Liu W, Zhou R, Dai J, Yin J, Wang H, Tang LV, Chen S, Cheng H, Zhu M, Wang S, Song Y, Zhang P, Zhang L. Chinese guidance for the clinical application of adeno-associated virus vector-based gene therapy for hemophilia B (2025). Blood Sci. 2025 Oct 29;7(4):e00257. doi: 10.1097/BS9.0000000000000257. eCollection 2025 Dec.
Results publication: PubMed 41266685Xue F, Ju M, Zhu T, Zhou Z, Sun J, Yang L, Yan Z, Zhou H, Du X, Zheng C, Zheng J, Wu X, Du Z, Jiang W, Yang C, Xiao X, Liu W, Yang R, Zhang L. Factor IX-Padua AAV gene therapy in hemophilia B: phases 1/2 and 3 trials. Nat Med. 2026 Jan;32(1):93-102. doi: 10.1038/s41591-025-04012-y. Epub 2025 Nov 20.
Prevalence of neutralizing antibodies against AAV8,AAV9, and AAV843 in a Chinese populationLink provided by the sponsor.
Individual participant dataSharing planned: No

Related trials for Hemophilia B

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
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
Not yet recruitingPhase 1
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Hemophilia B
RecruitingPhase 3
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
Hemophilia

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. This study is not recruiting at the moment.

Lei Zhang, MD study chair

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

Caifeng Yang, Master study director

Shanghai Xinzhi BioMed Co., Ltd.

9 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-H901 in people with Hemophilia B, run by Shanghai Xinzhi BioMed Co., Ltd. at 9 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 6 inclusion and 6 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: under way, not looking for participants, as of the sponsor's last update on 1 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 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?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Phase 1/2: The incidence of dose limiting toxicity (DLT) events (time frame: 10 weeks post-infusion). 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 NCT05203679, which also lists 5 linked publications.

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 1 Apr 2026.