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.
• 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