A Pilot Study for the Safety and Expression of Dystrophin in Skeletal Muscle After SPOT-mRNA03 Administration in Duchenne Muscular Dystrophy (DMD) Patients
The primary objective of this study is to evaluate the safety and and tolerability of SPOT-mRNA03 administered by intravenous (IV) infusion to DMD patients. In addition, this study will preliminarily investigate the concentration changes in dystrophin mRNA concentration, dystrophin protein expression and engraftment, as well as cytokine profiles and immunogenicity.
• 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.
• Ambulatory boys aged between 2 to 6 years of age, inclusive who can work without assistance 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