Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients (NCT07188012) | Clinical Trial Compass
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
China9 participantsStarted 2025-08-06
Plain-language 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, anti-dystrophin antibodies and cytokine profiles, as well as fat tissue mas and lean tissue mass following SPOT-03 administrations.
Who can participate
Age range2 Years – 7 Years
SexMALE
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Inclusion criteria
✓. 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;
✕. 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.
What they're measuring
1
Number of Participants with Treatment-Related Adverse Events Following Intravenous (IV) Infusion of SPOT-03 in DMD patients
Timeframe: From enrollment through 1-year post-treatment
. 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.
✕. Hospitalization due to worsening heart failure or arrhythmia within 8 weeks prior to screening.
✕. Abnormal laboratory values considered clinically significant: