Clinical Trial

Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients

Recruiting Early Phase 1
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Summary
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.
Protocol Amendment History 1 amendment
This ClinicalTrials.gov record has been amended once since 2025-09-15.
Trial Details
NCT Number NCT07188012
Lead Sponsor Shanghai Siponuoyin Biotechnology Co Ltd
Collaborators: Shanghai Children's Medical Center
Conditions Duchenne Muscular Dystrophy (DMD)
Enrollment 9 participants
Start Date 2025-08-06
Primary Completion 2026-12 (estimated)
Study Completion 2026-12 (estimated)
Updated on ClinicalTrials.gov 2026-05-07