Status: Recruiting
Safety and Dystrophin Expression of SPOT-03 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.
Participants needed: 9
Trial details
Phase: Early Phase 1Age: 2-7Biological sex: MaleType: InterventionalSponsor: Shanghai Siponuoyin Biotechnology Co LtdUpdated: May 7, 2026Locations: 1
Eligibility criteria
According to the requirements of the region/country and/or IRB/IEC, the patient... [+6]
Complications other than DMD that may cause muscle weakness and/or motor dysfunc... [+19]