A Pilot Study for the Safety and Expression of Dystrophin in Skeletal Muscle After SPOT-mRNA03 Administration in Duchenne Muscular Dystrophy (DMD) Patients

Study Summary

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.

Interventions

Study Locations

Facility	City	State	Country
Shanghai Children's Medical Center	Shanghai	Shanghai Municipality	China

Official Trial Information

View on ClinicalTrials.gov