SPOT-03 gene therapy for children with Duchenne muscular dystrophy
Tests the safety of SPOT-03, given by infusion, in children with Duchenne muscular dystrophy.
Plain-language title generated from the registry record
Registry title: Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Key facts
- Where
- Shanghai, ChinaSee the site
- Phase
- Early phase 1
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for boys aged 2 to under 8 years with Duchenne muscular dystrophy (DMD) who can walk on their own. They must have a confirmed Duchenne gene mutation.
- Age
- 2 years to under 8 years
- Sex
- Boys only
- Disease stage
- Duchenne muscular dystrophy confirmed by a clinical diagnosis. The child must be able to walk at least 10 meters without help.
- Earlier treatment
- Cannot join if they had other gene therapy, an investigational drug or any treatment meant to raise dystrophin. Cannot join if they are on immunosuppressive therapy.
- Biomarker or test result
- Duchenne mutation confirmed by genetic testing (MLPA and whole genome sequencing).
Main reasons for not taking part
- Other conditions that may cause muscle weakness or movement problems
- Hospital stay for breathing failure in the 8 weeks before screening
- Severe uncontrolled heart failure (NYHA III-IV) or arrhythmias that need treatment
- Positive test for hepatitis B core antibody, hepatitis C antibody or HIV antibody
- Another investigational drug or clinical trial in the 6 months before the first infusion
Read by machine on 9 November 2026.
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 2 and 7 years old?
Source: NCT07188012, who can take part
Where in China
- Shanghai Children's Medical CenterShanghai, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Shanghai Siponuoyin Biotechnology Co Ltd
- Trial code
- NCT07188012
- Record updated
- 5 November 2026
- Original description
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 full original descriptionShow less
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.
Inclusion: 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.
Exclusion: Hospitalization for respiratory failure within 8 weeks prior to screening; 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; Hospitalization due to worsening heart failure or arrhythmia within 8 weeks prior to screening; 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; 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; 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; Arrhythmias that require anti-arrhythmic treatment; 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; 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; Complications other than DMD that may cause muscle weakness and/or motor dysfunction.