DMDPhase 1/2NCT07058662
A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.
A Phase 1/2 study testing Single dose intravenous of BBM-D101 in people with DMD, run by Belief BioMed (Beijing) Co., Ltd at 1 site in China.
Trial summary
The purpose of the study is to evaluate the safety, tolerability, and efficacy of BBM-D101 to treat participants with Duchenne Muscular Dystrophy. Read more about this study
- Who is it for?
- Ages 4 to 9 years, men only
- Conditions the study is for
- DMD
- What is being tested?
- Single dose intravenous of BBM-D101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 31 Jul 2025to 30 Jun 2031
- Phase
- Phase 1/2
- 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 4 to 9 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- The Participants and/or his legal guardian must fully understand the purpose, nature, methods, and potential risks of the study, and sign a written informed consent form.
- Ambulatory male subjects aged 4 years and above but under 9 years (4 years ≤ age < 9 years).
- Any mutation in the DMD gene confirmed by genetic testing
- Serum creatine kinase (CK) during the screening period meets the study requirements.
- Receiving stable, standard-dose glucocorticoids before screening.
- The subject's AAV capsid antibodies meet the clinical trial requirements.
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- Able to cooperate with motor function assessment, MRI, and muscle biopsy as required by the study.
- Laboratory test results during the screening period and at baseline meet the standards.
- The subject and/or his legal guardian must fully understand the study procedures, be willing to actively cooperate, commit to high compliance with the protocol, and ensure that the subject attends all scheduled visits.
Who cannot join
- Positive for hepatitis B surface antigen (HBsAg), hepatitis B virus deoxyribonucleic acid (HBV-DNA) ≥ 1000 U/mL, hepatitis C virus ribonucleic acid (HCV-RNA) positive, human immunodeficiency virus (HIV) positive, or positive for Treponema pallidum antibodies.
- Currently receiving antiviral therapy for hepatitis B, hepatitis C, HIV, etc.
- The investigator deems the subject has severe behavioral or cognitive disorders that may hinder participation in this study.
- Poorly controlled asthma, or Duchenne Muscular Dystrophy (DMD) leading to significant decline in lung function, or recurrent infectious pneumonia that the investigator considers may affect respiratory function.
- Left ventricular ejection fraction (LVEF) < 50% or New York Heart Association (NYHA) cardiac function class ≥ III.
- Severe or persistent arrhythmias (such as atrial fibrillation, frequent ventricular premature beats, ventricular bigeminy, ventricular trigeminy, severe bundle branch block, etc.), and congenital heart disease that is evaluated by the investigator as unsuitable for participation in this study.
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- Any changes in preventive/cardiomyopathy treatment (initiation of treatment, drug changes, dosing regimen changes, treatment interruption, termination, or restart) within 1 month before the infusion of the study drug.
- History of liver diseases such as portal hypertension, splenomegaly, hepatic encephalopathy, liver fibrosis ≥ stage 3, or hepatic nodules/cysts found by ultrasound during screening, or elevated alpha-fetoprotein with clinical significance as determined by the investigator.
- Severe infection (such as pneumonia, pyelonephritis, or meningitis) within 4 weeks before the treatment visit (enrollment may be postponed).
- History of gene therapy or cell therapy (such as stem cell transplantation).
- History of or current presence of autoimmune diseases, severe renal, gastrointestinal, neurological, or coagulation disorders, malignant tumors, or other diseases.
- Other diseases that the investigator deems unsuitable for participation in this study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
DMD Clinical Trial Team contact
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
Yi Dai, MD, +86-13811177531, pumchdy@sina.com
About this study
Official title: A Phase 1/2, Open-label Clinical Study to Evaluate the Safety, Tolerability and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.
The purpose of the study is to evaluate the safety, tolerability, and efficacy of BBM-D101 to treat participants with Duchenne Muscular Dystrophy.
Read the detailed description
This is a single-arm, open-label study to evaluate the safety, tolerability, efficacy, pharmacokinetic, pharmacodynamic, and immune response of BBM-D101 within 52 weeks after a single intravenous infusion in DMD boys, as well as the long-term safety and efficacy of BBM-D101 for up to 5 years post infusion.
BBM-D101 is a gene addition therapy based on engineered AAV delivery therapeutic protein gene cassette into muscle for treating DMD. Therapeutic protein could mediate the dystrophin-associated protein complex to prevent muscular dystrophy and to rescue the function of muscle.
- Conditions
- DMD
- Being tested
- Single dose intravenous of BBM-D101
- Keywords
- DMD, gene therapy, Adeno-Associated Virus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Single dose intravenous of BBM-D101Genetic
BBM-D101 is a gene addition therapy based on engineered AAV delivery therapeutic protein gene cassette into muscle for treating DMD. Therapeutic protein could mediate the dystrophin-associated protein complex to prevent muscular dystrophy and to rescue the function of muscle.The administration is completed by a single intravenous infusion.
| Study group | Role | What they receive |
|---|---|---|
| Arm of BBM-D101 | Experimental | Single-dose treatment Receives: Genetic: Single dose intravenous of BBM-D101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of dose limiting toxicity (DLT) events
To evaluate the rate of incidence of DLT events determined by the Safety Data Review Committee (SRC) in DLT observation period after BBM-D101 infusion.
Measured: Within 12 weeks
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
To evaluate the safety of BBM-D101 by AEs and SAEs.
Measured: Within 12 weeks
Secondary outcomes (8)
Changes from baseline in BBM-D101 therapeutic protein level of muscle biopsy samples
To assess changes of therapatic protein from baseline in muscle after BBM-D101 Infusion.
Measured: Within 52 weeks
Changes from baseline in serum Creatine Kinase (CK) level
To assess changes of serum CK after BBM-D101 Infusion.
Measured: Within 52 weeks
Changes from baseline in the time to ascend time to rise (TTR) without assistance
To assess changes in TTR from baseline within 52 weeks after BBM-D101 infusion.
Measured: Within 52 weeks
Changes from baseline in the time to ascend 10-meter walk/run test (10MWR) without assistance
To assess changes in 10MWR from baseline within 52 weeks after BBM-D101 infusion.
Measured: Within 52 weeks
Changes from baseline in the time to ascend 4 steps (4-stair climb, 4-SC) without assistance.
To assess changes in 4-SC from baseline within 52 weeks after BBM-D101 infusion.
Measured: Within 52 weeks
Changes from baseline in the North Star Ambulatory Assessment (NSAA).
To assess changes in NSAA from baseline within 52 weeks after BBM-D101 infusion; The NSAA is a scale that rates performance of various motor abilities in ambulant children with Duchenne Muscular Dystrophy and is used to monitor disease progression and treatment effects. The NSAA total score is defined as the sum of all 17items, ranging from 0 (worst) to 34 (best).
Measured: Within 52 weeks
Changes from baseline in the TTR， 10MWR , 4-SC，NSAA.
Evaluate the changes from baseline in TTR, 10MWR, 4-SC and NSAA in Participants after Infusion of BBM-D101.
Measured: Within 5 years
Incidence of AEs and SAEs.
To assess the long-term safety of BBM-D101 by AEs and SAEs.
Measured: Within 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2025
- Study start31 Jul 2025
- Primary completion30 Jun 2027 (estimated)
- Study completion30 Jun 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DMDStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Belief BioMed (Beijing) Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Shanghai Mianyi Biopharmaceutical Co., Ltd., Shanghai Xinzhi BioMed Co., Ltd., Belief BioMed Limited
- Responsible party
- Sponsor
- Investigators
- Yi Dai, MD, Principal investigator, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07058662
- Sponsor's protocol ID
- BBM041-CLN1001
- Status last verified
- Nov 2025
- First submitted
- 1 Jul 2025
- Last updated
- 4 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Open-label Clinical Study to Evaluate the Safety, Tolerability and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.
- ClinicalTrials.gov ID
- NCT07058662
- Acronym / sponsor ID
- BBM041-CLN1001
- Conditions
- DMD
- Keywords
- DMD, gene therapy, Adeno-Associated Virus
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Belief BioMed (Beijing) Co., LtdIndustry (pharma, biotech, devices)
- Collaborators
- Shanghai Mianyi Biopharmaceutical Co., Ltd., Shanghai Xinzhi BioMed Co., Ltd., Belief BioMed Limited
- Study start
- 31 Jul 2025 (actual)
- Primary completion
- 30 Jun 2027 (estimated)
- Study completion
- 30 Jun 2031 (estimated)
- First posted
- 10 Jul 2025 (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.
The purpose of the study is to evaluate the safety, tolerability, and efficacy of BBM-D101 to treat participants with Duchenne Muscular Dystrophy.
Detailed description
This is a single-arm, open-label study to evaluate the safety, tolerability, efficacy, pharmacokinetic, pharmacodynamic, and immune response of BBM-D101 within 52 weeks after a single intravenous infusion in DMD boys, as well as the long-term safety and efficacy of BBM-D101 for up to 5 years post infusion.
BBM-D101 is a gene addition therapy based on engineered AAV delivery therapeutic protein gene cassette into muscle for treating DMD. Therapeutic protein could mediate the dystrophin-associated protein complex to prevent muscular dystrophy and to rescue the function of muscle.
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-D101 | Experimental | Genetic: Single dose intravenous of BBM-D101Single-dose treatment |
Who may qualify
- Ages
- 4 to 9 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- The Participants and/or his legal guardian must fully understand the purpose, nature, methods, and potential risks of the study, and sign a written informed consent form.
- Ambulatory male subjects aged 4 years and above but under 9 years (4 years ≤ age < 9 years).
- Any mutation in the DMD gene confirmed by genetic testing
- Serum creatine kinase (CK) during the screening period meets the study requirements.
- Receiving stable, standard-dose glucocorticoids before screening.
Key exclusion criteria (12 listed)
- Positive for hepatitis B surface antigen (HBsAg), hepatitis B virus deoxyribonucleic acid (HBV-DNA) ≥ 1000 U/mL, hepatitis C virus ribonucleic acid (HCV-RNA) positive, human immunodeficiency virus (HIV) positive, or positive for Treponema pallidum antibodies.
- Currently receiving antiviral therapy for hepatitis B, hepatitis C, HIV, etc.
- The investigator deems the subject has severe behavioral or cognitive disorders that may hinder participation in this study.
- Poorly controlled asthma, or Duchenne Muscular Dystrophy (DMD) leading to significant decline in lung function, or recurrent infectious pneumonia that the investigator considers may affect respiratory function.
- Left ventricular ejection fraction (LVEF) < 50% or New York Heart Association (NYHA) cardiac function class ≥ III.
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 of BBM-D101BBM-D101 is a gene addition therapy based on engineered AAV delivery therapeutic protein gene cassette into muscle for treating DMD. Therapeutic protein could mediate the dystrophin-associated protein complex to prevent muscular dystrophy and to rescue the function of muscle.The administration is completed by a single intravenous infusion. | Genetic | Arm of BBM-D101 |
Primary outcome
Incidence of dose limiting toxicity (DLT) events
Time frame: Within 12 weeks
To evaluate the rate of incidence of DLT events determined by the Safety Data Review Committee (SRC) in DLT observation period after BBM-D101 infusion.
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Within 12 weeks
To evaluate the safety of BBM-D101 by AEs and SAEs.
8 secondary outcomes
Changes from baseline in BBM-D101 therapeutic protein level of muscle biopsy samples
Time frame: Within 52 weeks
Changes from baseline in serum Creatine Kinase (CK) level
Time frame: Within 52 weeks
Changes from baseline in the time to ascend time to rise (TTR) without assistance
Time frame: Within 52 weeks
Changes from baseline in the time to ascend 10-meter walk/run test (10MWR) without assistance
Time frame: Within 52 weeks
Changes from baseline in the time to ascend 4 steps (4-stair climb, 4-SC) without assistance.
Time frame: Within 52 weeks
Changes from baseline in the North Star Ambulatory Assessment (NSAA).
Time frame: Within 52 weeks
Changes from baseline in the TTR， 10MWR , 4-SC，NSAA.
Time frame: Within 5 years
Incidence of AEs and SAEs.
Time frame: Within 5 years
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 4 Dec 2025.
- Individual participant dataSharing planned: No
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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.
DMD Clinical Trial Team contact
Yi Dai, MD principal investigator
Peking Union Medical College Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Single dose intravenous of BBM-D101 in people with DMD, run by Belief BioMed (Beijing) Co., Ltd at 1 site 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 9 inclusion and 12 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 4 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Hanyang Hu, Ph.D (+86-021-33588288, huhanyang@beliefbiomed.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of dose limiting toxicity (DLT) events (time frame: Within 12 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 4 Dec 2025.