SPOT-03 IV gene therapy to deliver dystrophin in boys with Duchenne muscular dystrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a first-in-human, open-label, single-arm, single-center study testing muscle-targeted extracellular vesicles (SPOT-03) loaded with full-length dystrophin nucleic acid delivered by IV infusion. Six to nine ambulatory boys aged 2 to <8 will be enrolled into two dosing schedules that differ by total administrations, with concurrent oral tacrolimus to manage immune response. The primary focus is safety and tolerability, with planned measurements of dystrophin nucleic acid and protein expression from muscle biopsy, anti-dystrophin antibodies, cytokine profiles, and body composition changes. Dosing includes intensive repeat infusions and scheduled follow-up assessments to capture engraftment and biomarker changes.

Why it matters

Who can join: the full rules

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.

Where it's running

• Shanghai Children's Medical Center
  Shanghai, Shanghai Municipality, China Recruiting

Study contacts

• Principal investigator: Wang Jiwen — Shanghai Children's Medical Center
• Study coordinator: Winston Town
• Email: Winston.town@spotbiosystems.com
• Phone: 212-920-5501

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.