Duchenne Muscular Dystrophy (DMD)Early Phase 1NCT06641895
Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy
A Early Phase 1 study testing BBM-D101 in people with Duchenne Muscular Dystrophy (DMD), run by Shanghai Jiao Tong University School of Medicine at 1 site in China.
Trial summary
The purpose of the study is to assess the safety, tolerability, and efficacy of BBM-D101 to treat patients with Duchenne Muscular Dystrophy. Read more about this study
- Who is it for?
- Ages 4 to 8 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- What is being tested?
- BBM-D101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteChina
- Study dates
- 25 Jul 2024to 31 Jul 2030
- Phase
- Early Phase 1
- 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 8 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- The legal guardian of the subject fully understands the purpose, nature, methods, and possible risks of the study, and signs a written informed consent form;
- The study includes ambulatory male subjects who are at least 4 years old and less than 8 years old (4 years old ≤ age \< 8 years old) ;
- Genetically confirmed diagnosis of DMD;
- Have at least 1 of the following typical clinical signs or laboratory abnormalities of DMD: proximal muscle weakness, waddling gait, pseudo gastrocnemius hypertrophy, Gower\'s sign, pterygoid scapula;
- Ability to cooperate with motor assessment testing, magnetic resonance imaging (MRI) and muscle biopsy according to the requirements of the study.
Who cannot join
- Hepatitis B surface antigen (HBsAg) positive, hepatitis B virus deoxyribonucleic acid (HBV-DNA) ≥1000U/mL, hepatitis C virus ribonucleic acid (HCV-RNA) positive or human immunodeficiency virus (HIV) positive;
- Receiving antiviral therapy for hepatitis B, hepatitis C, HIV, etc.;
- Left ventricular ejection fraction (LVEF) \<50% or ≥ class III cardiac function defined by New York Heart Association (NYHA);
- With severe or persistent arrhythmias and congenital heart disease.
- The subject\'s preventive treatment/cardiomyopathy treatment changes within 1 month before the start of the study treatment;
- With underlying liver disease, such as previous diagnosis of portal hypertension, splenomegaly, hepatic encephalopathy, or hepatic fibrosis ≥ stage 3; or nodules, cysts found by B-ultrasound in the past, or elevated alpha-fetoprotein in laboratory tests during the screening period, etc., and these abnormalities are judged by the investigator to be clinically significant;
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, 200127
Jiwen Wang, 18916613192, wangjiwen@scmc.com.cn
About this study
Official title: A Single-Arm, Open-Label, Single-Dose Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 Injection in Patients with Duchenne Muscular Dystrophy
The purpose of the study is to assess the safety, tolerability, and efficacy of BBM-D101 to treat patients 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 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
- Duchenne Muscular Dystrophy (DMD)
- Being tested
- BBM-D101
- Keywords
- Adeno-Associated Virus, gene therapy, Duchenne muscular dystrophy
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BBM-D101Genetic
BBM-D101 is a recombinant adeno-associated virus vector-based gene therapy for DMD treatment. It is a suspension for single intravenous (IV) infusion.
| Study group | Role | What they receive |
|---|---|---|
| Single dose intravenous injection of BBM-D101 | Experimental | Not described Receives: Genetic: 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 access the numbers of DLT events determined by the Safety Data Review Committee (SRC) in DLT observation period after BBM-D101 injection infusion.
Measured: 12 weeks
The incidence of adverse events (AEs) and serious adverse events (SAEs)
To assess the safety of BBM-D101 Injection by AEs and SAEs.
Measured: 52 weeks
Secondary outcomes (7)
Changes from baseline in the North Star Ambulatory Assessment (NSAA)
To assess changes in NSAA from baseline within 12 weeks, 26 weeks, and 52 weeks after BBM-D101 injection 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: 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 12 weeks, 26 weeks, and 52 weeks after BBM-D101 injection infusion
Measured: 52 weeks
Changes from baseline in the time to ascend time to rise (TTR) without assistance without assistance
To assess changes in TTR from baseline within 12 weeks, 26 weeks, and 52 weeks after BBM-D101 injection infusion
Measured: 52 weeks
Changes from baseline in the time to ascend 4 steps (4-stair climb, 4SC) without assistance
To assess changes in 4-SC from baseline within 12 weeks, 26 weeks, and 52 weeks after BBM-D101 injection infusion
Measured: 52 weeks
Changes from baseline in the time to ascend 100-meter walk/run test (100MWR) without assistance
To assess changes in 100MWR from baseline within 12 weeks, 26 weeks, and 52 weeks after BBM-D101 injection infusion
Measured: 52 weeks
Changes from baseline in BBM-D101 genome copies in muscle biopsy samples
To assess changes of BBM-D101 genome from baseline in muscle in 12 weeks and 52 weeks following BBM-D101 administration. BBM-D101 genome was detected by Quantitative Polymerase Chain Reaction (QPCR).
Measured: 52 weeks
Changes from baseline in BBM-D101 therapeutic protein level in muscle biopsy samples
To assess changes of BBM-D101 therapeutic protein from baseline in muscle in 12 weeks and 52 weeks following BBM-D101 administration. BBM-D101 therapeutic protein was detected by western blot (Jess) and tissue immunofluorescence.
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Oct 2024
- Study start25 Jul 2024
- Primary completion31 Jul 2026 (estimated)
- Study completion31 Jul 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies of the same condition, open and under way.
- More trials run by Shanghai Jiao Tong University School of MedicineThe sponsor's other registered studies.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Jiao Tong University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Shanghai Mianyi Biopharmaceutical Co., Ltd.
- Responsible party
- Principal investigator, Jiwen Wang, Shanghai Jiao Tong University School of Medicine
- Investigators
- Jiwen Wang, Study chair, Shanghai Children's Medical Center, affiliated to Shanghai Jiao Tong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06641895
- Sponsor's protocol ID
- BBM041-IIT1001
- Status last verified
- Oct 2024
- First submitted
- 8 Oct 2024
- Last updated
- 25 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Arm, Open-Label, Single-Dose Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 Injection in Patients with Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT06641895
- Acronym / sponsor ID
- BBM041-IIT1001
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Keywords
- Adeno-Associated Virus, gene therapy, Duchenne muscular dystrophy
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Shanghai Jiao Tong University School of MedicineUniversity, hospital or other
- Collaborators
- Shanghai Mianyi Biopharmaceutical Co., Ltd.
- Study start
- 25 Jul 2024 (actual)
- Primary completion
- 31 Jul 2026 (estimated)
- Study completion
- 31 Jul 2030 (estimated)
- First posted
- 15 Oct 2024 (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 assess the safety, tolerability, and efficacy of BBM-D101 to treat patients 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 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 |
|---|---|---|
| Single dose intravenous injection of BBM-D101 | Experimental | Genetic: BBM-D101 |
Who may qualify
- Ages
- 4 to 8 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- The legal guardian of the subject fully understands the purpose, nature, methods, and possible risks of the study, and signs a written informed consent form;
- The study includes ambulatory male subjects who are at least 4 years old and less than 8 years old (4 years old ≤ age \< 8 years old) ;
- Genetically confirmed diagnosis of DMD;
- Have at least 1 of the following typical clinical signs or laboratory abnormalities of DMD: proximal muscle weakness, waddling gait, pseudo gastrocnemius hypertrophy, Gower\'s sign, pterygoid scapula;
- Ability to cooperate with motor assessment testing, magnetic resonance imaging (MRI) and muscle biopsy according to the requirements of the study.
Key exclusion criteria (6 listed)
- Hepatitis B surface antigen (HBsAg) positive, hepatitis B virus deoxyribonucleic acid (HBV-DNA) ≥1000U/mL, hepatitis C virus ribonucleic acid (HCV-RNA) positive or human immunodeficiency virus (HIV) positive;
- Receiving antiviral therapy for hepatitis B, hepatitis C, HIV, etc.;
- Left ventricular ejection fraction (LVEF) \<50% or ≥ class III cardiac function defined by New York Heart Association (NYHA);
- With severe or persistent arrhythmias and congenital heart disease.
- The subject\'s preventive treatment/cardiomyopathy treatment changes within 1 month before the start of the study treatment;
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 |
|---|---|---|
| BBM-D101BBM-D101 is a recombinant adeno-associated virus vector-based gene therapy for DMD treatment. It is a suspension for single intravenous (IV) infusion. | Genetic | Single dose intravenous injection of BBM-D101 |
Primary outcome
Incidence of dose limiting toxicity (DLT) events
Time frame: 12 weeks
To access the numbers of DLT events determined by the Safety Data Review Committee (SRC) in DLT observation period after BBM-D101 injection infusion.
The incidence of adverse events (AEs) and serious adverse events (SAEs)
Time frame: 52 weeks
To assess the safety of BBM-D101 Injection by AEs and SAEs.
7 secondary outcomes
Changes from baseline in the North Star Ambulatory Assessment (NSAA)
Time frame: 52 weeks
Changes from baseline in the time to ascend 10-meter walk/run test (10MWR) without assistance
Time frame: 52 weeks
Changes from baseline in the time to ascend time to rise (TTR) without assistance without assistance
Time frame: 52 weeks
Changes from baseline in the time to ascend 4 steps (4-stair climb, 4SC) without assistance
Time frame: 52 weeks
Changes from baseline in the time to ascend 100-meter walk/run test (100MWR) without assistance
Time frame: 52 weeks
Changes from baseline in BBM-D101 genome copies in muscle biopsy samples
Time frame: 52 weeks
Changes from baseline in BBM-D101 therapeutic protein level in muscle biopsy samples
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 25 Mar 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.
Jiwen Wang study chair
Shanghai Children's Medical Center, affiliated to Shanghai Jiao Tong University School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing BBM-D101 in people with Duchenne Muscular Dystrophy (DMD), run by Shanghai Jiao Tong University School of Medicine 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 5 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: looking for participants now, as of the sponsor's last update on 25 Mar 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 phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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 Jiwen Wang (18916613192, wangjiwen@scmc.com.cn), 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: 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 25 Mar 2025.