Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients

Plain-language summary

This study examines the safety of SPOT-03 in patients with Duchenne muscular dystrophy (DMD) and whether it increases production of the dystrophin protein in muscle. It is an Early Phase 1 study and is currently recruiting.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Early Phase 1

Status

Recruiting

Sponsor

Shanghai Siponuoyin Biotechnology Co Ltd

Intervention

SPOT-03

Start date

8/6/2025

Estimated completion

12/1/2026

Countries

China

Related genes

Locations

• Shanghai, China

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

1. 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.
2. Boys aged ≥ 2 years to \< 8 years and capable of walking independently for at least 10 meters.
3. The medical history includes clinical diagnosis of DMD and confirmed Duchenne mutations using validated genetic testing (MLPA and whole genome sequencing).
4. Able to tolerate muscle biopsy under anesthesia and have no contraindications to biopsy.
5. Heart, liver, lung, and kidney functions are sufficient:

   1. The left ventricular ejection fraction (LVEF) should be ≥ 50%;
   2. Forced vital capacity (FVC) \> 50% of the expected value, and do not require nighttime ventilation;
   3. Patient's glomerular filtration rate (GFR)\>30 mL/min/1.73 m2

Exclusion Criteria:

1. Complications other than DMD that may cause muscle weakness and/or motor dysfunction.
2. 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.
3. Hospitalization for respiratory failure within 8 weeks prior to screening.
4. 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.
5. Severe uncontrolled heart failure (NYHA III-IV), including any of the following conditions：

   1. Intravenous administration of diuretics or positive inotropic drugs is required within 8 weeks prior to screening.
   2. Hospitalization due to worsening heart failure or arrhythmia within 8 weeks prior to screening.
6. Abnormal laboratory values considered clinically significant:

   1. GGT \> 3 × upper limit of normal
   2. Bilirubin ≥ 3.0 mg/dL
   3. Creatinine ≥ 1.8 mg/dL
   4. Hemoglobin \< 8 or \> 18 g/dL
   5. White blood cell count \> 18,500/μL
7. Arrhythmias that require anti-arrhythmic treatment.
8. Subjects who are undergoing immunosuppressive therapy.
9. Has used other gene therapy, investigational drugs, or any treatment aimed at increasing dystrophin expression.
10. 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.
11. Subjects who are allergic to investigational products or local aesthetic drugs or have a history of severe allergies or genetic allergic reactions.
12. Within 6 months prior to the initial infusion, the subjects are exposed to another investigational drug or have participated in an intervention clinical trial.
13. Subjects with positive hepatitis B core antibody or hepatitis C antibody or HIV antibody during screening.
14. Investigator believes that the presence of any other serious diseases, medical conditions, or chronic drug treatment needs can pose unnecessary risks to gene transfer.