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.
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.
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.
Shanghai Siponuoyin Biotechnology Co Ltd
Lead sponsor
Shanghai Children's Medical Center
Collaborator