Duchenne Muscular Dystrophy (DMD)Early Phase 1NCT07188012
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
A Early Phase 1 study testing SPOT-03 in people with Duchenne Muscular Dystrophy (DMD), run by Shanghai Siponuoyin Biotechnology Co Ltd at 1 site in China.
Trial summary
The primary objective of this study is to evaluate the safety and tolerability of SPOT-03 administered by intravenous (IV) infusion to DMD patients. In addition, this study will preliminarily investigate the changes in dystrophin nucleic acid concentration, dystrophin protein expression and engraftment… Read more about this study
- Who is it for?
- Ages 2 to 7 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- What is being tested?
- SPOT-03
- 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
- 6 Aug 2025to Dec 2028
- 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 2 to 7 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- According to the requirements of the region/country and/or IRB/IEC, the patient and/or legal guardian have signed a written informed consent form and are aware of all relevant study content.
- Boys aged ≥ 2 years to < 8 years and capable of walking independently for at least 10 meters.
- The medical history includes clinical diagnosis of DMD and confirmed Duchenne mutations using validated genetic testing (MLPA and whole genome sequencing).
- Able to tolerate muscle biopsy under anesthesia and have no contraindications to biopsy.
- Heart, liver, lung, and kidney functions are sufficient:
- The left ventricular ejection fraction (LVEF) should be ≥ 50%;
- Forced vital capacity (FVC) > 50% of the expected value, and do not require nighttime ventilation;
- Patient's glomerular filtration rate (GFR)>30 mL/min/1.73 m2
Who cannot join
- Complications other than DMD that may cause muscle weakness and/or motor dysfunction.
- There are severe intellectual disabilities (such as severe autism, severe cognitive impairment, and severe behavioral disorders) that, according to the investigator's judgment, can affect the study.
- Hospitalization for respiratory failure within 8 weeks prior to screening.
- Asthma or underlying lung diseases that are poorly controlled, such as bronchitis, bronchiectasis, emphysema, or recurrent infectious pneumonia that investigator believes may affect respiratory function.
- Severe uncontrolled heart failure (NYHA III-IV), including any of the following conditions：
- Intravenous administration of diuretics or positive inotropic drugs is required within 8 weeks prior to screening.
Show all 21Show fewer
- Hospitalization due to worsening heart failure or arrhythmia within 8 weeks prior to screening.
- Abnormal laboratory values considered clinically significant:
- GGT > 3 × upper limit of normal
- Bilirubin ≥ 3.0 mg/dL
- Creatinine ≥ 1.8 mg/dL
- Hemoglobin < 8 or > 18 g/dL
- White blood cell count > 18,500/μL
- Arrhythmias that require anti-arrhythmic treatment.
- Subjects who are undergoing immunosuppressive therapy.
- Has used other gene therapy, investigational drugs, or any treatment aimed at increasing dystrophin expression.
- Subjects with a history of major surgeries within 12 weeks prior to the initial infusion or planning to undergo major surgeries (such as scoliosis surgery) during this study.
- Subjects who are allergic to investigational products or local aesthetic drugs or have a history of severe allergies or genetic allergic reactions.
- Within 6 months prior to the initial infusion, the subjects are exposed to another investigational drug or have participated in an intervention clinical trial.
- Subjects with positive hepatitis B core antibody or hepatitis C antibody or HIV antibody during screening.
- Investigator believes that the presence of any other serious diseases, medical conditions, or chronic drug treatment needs can pose unnecessary risks to gene transfer.
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
Shanghai, Shanghai Municipality
Winston Town, 212-920-5501, Winston.town@spotbiosystems.com
Wang Jiwen
About this study
Official title: A Pilot Study for the Safety and Expression of Dystrophin in Skeletal Muscle After SPOT-03 Administration in Duchenne Muscular Dystrophy (DMD) Patients
The primary objective of this study is to evaluate the safety and tolerability of SPOT-03 administered by intravenous (IV) infusion to DMD patients. In addition, this study will preliminarily investigate the changes in dystrophin nucleic acid concentration, dystrophin protein expression and engraftment, anti-dystrophin antibodies and cytokine profiles, as well as fat tissue mas and lean tissue mass following SPOT-03 administrations.
Read the detailed description
This is a FIH, open-label, single-arm, and single-center exploratory clinical study of SPOT-03 administered via IV infusion for DMD patients. SPOT-03 is a muscle-targeted extracellular vesicles (EVs) loaded with full-length dystrophin nucleic acid. The targeting ability of SPOT-03 is conferred by molecular targeting peptides on the EVs membrane, enabling the delivery of dystrophin nucleic acid as a gene therapy product for DMD.
The study has two dose groups:
Group I: 4.0E+11 dystrophin nucleic acid copies/kg, administered 8 times Group II: 4.0E+11 dystrophin nucleic acid copies/kg, administered 32 times The study will have a Screening Period of 30 days, during which patients or their legal guardian written informed consent will be obtained before Screening assessments and eligibility will be determined. A total of 6 to 9 DMD patients aged 2 to less than 8 years will be enrolled in this study according to the inclusion criteria. Complete data from at least 3 subjects will be obtained for Group II. All subjects will begin oral tacrolimus (0.05-0.2 mg/kg/d, adjustable according to actual clinical conditions) 3 days before the initial administration of SPOT-03 (D-3) and, with the duration aligned to the treatment schedule: 4 weeks for Group I and 16 weeks for Group II. In group I, the first dose of SPOT-03 will be administered by intravenous infusion on D1, followed by twice a week administration (once every 4 days) for a total of 8 doses. In group II, the first dose of SPOT-03 will be administered by intravenous infusion on D1, followed by twice a week administration (once every 4 days) for a total of 32 doses.
After all subjects in the previous dose group have completed SPOT-03 administrations, the next dose group may proceed after the investigators and sponsors have discussed and determined that there are no serious adverse reactions related to the drug.
Safety tests and evaluations will be conducted for the patients during each administration and follow-up.
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Being tested
- SPOT-03
- Keywords
- Duchenne Muscular Dystrophy, North Star Ambulatory Assessment, Dystrophin, Gene Therapy, Extracellular Vesicles
- 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.
SPOT-03Genetic
SPOT-03 injection administered via IV infusion
| Study group | Role | What they receive |
|---|---|---|
| 8-Dose and 32-Dose | Experimental | In Group I, 3 patients will be administered SPOT-03 by intravenous infusion on D1, followed by twice a week administration (once every 4 days) for a total of 8 doses. In Group II, at least 3 patients will be administered SPOT-03 by intravenous infusion on D1, followed by twice a week administration (once every 4 days) for a total of 32 doses. Receives: Genetic: SPOT-03 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Treatment-Related Adverse Events Following Intravenous (IV) Infusion of SPOT-03 in DMD patients
Safety and tolerability of SPOT-03 will be assessed by collection and quantification of all adverse events, graded according to Common Terminology Criteria for Adverse Events (CTCAE) v5.0.
Measured: From enrollment through 1-year post-treatment
Secondary outcomes (7)
Concentration of Dystrophin Nucleic Acid in Blood
Dystrophin nucleic acid concentration measured in blood of DMD patients before and after IV infusion of SPOT-03. Quantification is performed by qPCR.
Measured: 70 weeks
Concentration of Dystrophin Nucleic Acid in Muscles
Dystrophin nucleic acid concentration measured in muscles of DMD patients at baseline, during the treatment (only Group II) and after the last IV infusion (dose 8 for Group I and dose 32 for Group II) of SPOT-03. Quantification is performed by qPCR.
Measured: 16 weeks
Percent of Normal Dystrophin Protein Expression in Muscles
Dystrophin protein measured in muscles of DMD patients at baseline, during the treatment (only Group II) and after the last IV infusion (dose 8 for Group I and dose 32 for Group II) of SPOT-03, expressed as percent of the mean wild-type level in healthy individuals without DMD or Becker muscular dystrophy. Quantification is performed by Western Blot.
Measured: 16 weeks
Percentage of Dystrophin-Positive Fibers in Muscles
Fiber intensity and dystrophin-positive fibers measured in muscles of DMD patients at baseline and after the last IV infusion (dose 8 for Group I and dose 32 for Group II) of SPOT-03. Quantification is performed by Immunofluorescence (IF) / Immunohistochemistry (IHC).
Measured: 16 weeks
Concentration of Cytokines in Serum
Immunogenicity assessment will be measured by evaluating the changes of cytokines levels (TNF-α, INF-γ, IL-2, IL-6, and IL-10) in the serum of DMD patients before and after IV infusion of SPOT-03.
Measured: 70 weeks
Concentration of Anti-Dystrophin Antibody in Serum
Anti-dystrophin antibody concentration measured in the serum of DMD patients before and after IV infusion of SPOT-03. Quantification is performed by enzyme-linked immunosorbent assay (ELISA).
Measured: 70 weeks
Adipose Tissue and Lean Tissue Mass (only Group II)
Adipose tissue and lean tissue mass of DMD patients measured at baseline, after IV infusion of SPOT-03, and during follow-up period. Quantification is performed by Dual-Energy X-ray Absorptiometry (DEXA).
Measured: 70 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2025
- Study start6 Aug 2025
- Primary completionDec 2028 (estimated)
- Study completionDec 2028 (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.
- 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
- Model description
- In Group I, 3 patients will be administered SPOT-03 by intravenous infusion on D1, followed by twice a week administration (once every 4 days) for a total of 8 doses. In Group II, at least 3 patients will be administered SPOT-03 by intravenous infusion on D1, followed by twice a week administration (once every 4 days) for a total of 32 doses.
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Siponuoyin Biotechnology Co Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Shanghai Children's Medical Center
- Responsible party
- Sponsor
- Investigators
- Wang Jiwen, Principal investigator, Shanghai Children's Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07188012
- Sponsor's protocol ID
- FM-T3-SH
- Status last verified
- Nov 2026
- First submitted
- 22 Aug 2025
- Last updated
- 5 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Individual participant data will not be shared as this is an early-phase pilot study
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study for the Safety and Expression of Dystrophin in Skeletal Muscle After SPOT-03 Administration in Duchenne Muscular Dystrophy (DMD) Patients
- ClinicalTrials.gov ID
- NCT07188012
- Acronym / sponsor ID
- FM-T3-SH
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Keywords
- Duchenne Muscular Dystrophy, North Star Ambulatory Assessment, Dystrophin, Gene Therapy, Extracellular Vesicles
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Shanghai Siponuoyin Biotechnology Co LtdIndustry (pharma, biotech, devices)
- Collaborators
- Shanghai Children's Medical Center
- Study start
- 6 Aug 2025 (actual)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 23 Sep 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 primary objective of this study is to evaluate the safety and tolerability of SPOT-03 administered by intravenous (IV) infusion to DMD patients. In addition, this study will preliminarily investigate the changes in dystrophin nucleic acid concentration, dystrophin protein expression and engraftment, anti-dystrophin antibodies and cytokine profiles, as well as fat tissue mas and lean tissue mass following SPOT-03 administrations.
Detailed description
This is a FIH, open-label, single-arm, and single-center exploratory clinical study of SPOT-03 administered via IV infusion for DMD patients. SPOT-03 is a muscle-targeted extracellular vesicles (EVs) loaded with full-length dystrophin nucleic acid. The targeting ability of SPOT-03 is conferred by molecular targeting peptides on the EVs membrane, enabling the delivery of dystrophin nucleic acid as a gene therapy product for DMD.
The study has two dose groups:
Group I: 4.0E+11 dystrophin nucleic acid copies/kg, administered 8 times Group II: 4.0E+11 dystrophin nucleic acid copies/kg, administered 32 times The study will have a Screening Period of 30 days, during which patients or their legal guardian written informed consent will be obtained before Screening assessments and eligibility will be determined. A total of 6 to 9 DMD patients aged 2 to less than 8 years will be enrolled in this study according to the inclusion criteria. Complete data from at least 3 subjects will be obtained for Group II. All subjects will begin oral tacrolimus (0.05-0.2 mg/kg/d, adjustable according to actual clinical conditions) 3 days before the initial administration of SPOT-03 (D-3) and, with the duration aligned to the treatment schedule: 4 weeks for Group I and 16 weeks for Group II. In group I, the first dose of SPOT-03 will be administered by intravenous infusion on D1, followed by twice a week administration (once every 4 days) for a total of 8 doses. In group II, the first dose of SPOT-03 will be administered by intravenous infusion on D1, followed by twice a week administration (once every 4 days) for a total of 32 doses.
After all subjects in the previous dose group have completed SPOT-03 administrations, the next dose group may proceed after the investigators and sponsors have discussed and determined that there are no serious adverse reactions related to the drug.
Safety tests and evaluations will be conducted for the patients during each administration and follow-up.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- In Group I, 3 patients will be administered SPOT-03 by intravenous infusion on D1, followed by twice a week administration (once every 4 days) for a total of 8 doses. In Group II, at least 3 patients will be administered SPOT-03 by intravenous infusion on D1, followed by twice a week administration (once every 4 days) for a total of 32 doses.
| Arm | Type | Receives |
|---|---|---|
| 8-Dose and 32-Dose | Experimental | Genetic: SPOT-03In Group I, 3 patients will be administered SPOT-03 by intravenous infusion on D1, followed by twice a week administration (once every 4 days) for a total of 8 doses. In Group II, at least 3 patients will be administered SPOT-03 by intravenous infusion on D1, followed by twice a week administration (once every 4 days) for a total of 32 doses. |
Who may qualify
- Ages
- 2 to 7 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- According to the requirements of the region/country and/or IRB/IEC, the patient and/or legal guardian have signed a written informed consent form and are aware of all relevant study content.
- Boys aged ≥ 2 years to < 8 years and capable of walking independently for at least 10 meters.
- The medical history includes clinical diagnosis of DMD and confirmed Duchenne mutations using validated genetic testing (MLPA and whole genome sequencing).
- Able to tolerate muscle biopsy under anesthesia and have no contraindications to biopsy.
- Heart, liver, lung, and kidney functions are sufficient:
Key exclusion criteria (21 listed)
- Complications other than DMD that may cause muscle weakness and/or motor dysfunction.
- There are severe intellectual disabilities (such as severe autism, severe cognitive impairment, and severe behavioral disorders) that, according to the investigator's judgment, can affect the study.
- Hospitalization for respiratory failure within 8 weeks prior to screening.
- Asthma or underlying lung diseases that are poorly controlled, such as bronchitis, bronchiectasis, emphysema, or recurrent infectious pneumonia that investigator believes may affect respiratory function.
- Severe uncontrolled heart failure (NYHA III-IV), including any of the following conditions：
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 |
|---|---|---|
| SPOT-03SPOT-03 injection administered via IV infusion | Genetic | 8-Dose and 32-Dose |
Primary outcome
Number of Participants with Treatment-Related Adverse Events Following Intravenous (IV) Infusion of SPOT-03 in DMD patients
Time frame: From enrollment through 1-year post-treatment
Safety and tolerability of SPOT-03 will be assessed by collection and quantification of all adverse events, graded according to Common Terminology Criteria for Adverse Events (CTCAE) v5.0.
7 secondary outcomes
Concentration of Dystrophin Nucleic Acid in Blood
Time frame: 70 weeks
Concentration of Dystrophin Nucleic Acid in Muscles
Time frame: 16 weeks
Percent of Normal Dystrophin Protein Expression in Muscles
Time frame: 16 weeks
Percentage of Dystrophin-Positive Fibers in Muscles
Time frame: 16 weeks
Concentration of Cytokines in Serum
Time frame: 70 weeks
Concentration of Anti-Dystrophin Antibody in Serum
Time frame: 70 weeks
Adipose Tissue and Lean Tissue Mass (only Group II)
Time frame: 70 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 5 Nov 2026.
- Individual participant dataSharing planned: No. Individual participant data will not be shared as this is an early-phase pilot study
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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.
Wang Jiwen principal investigator
Shanghai Children's Medical Center
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 SPOT-03 in people with Duchenne Muscular Dystrophy (DMD), run by Shanghai Siponuoyin Biotechnology 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 8 inclusion and 21 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 5 Nov 2026. 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 Winston Town (212-920-5501, Winston.town@spotbiosystems.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Treatment-Related Adverse Events Following Intravenous (IV) Infusion of SPOT-03 in DMD patients (time frame: From enrollment through 1-year post-treatment). 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 5 Nov 2026.