RecruitingEarly Phase 1NCT07188012

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

A Pilot Study for the Safety and Expression of Dystrophin in Skeletal Muscle After SPOT-03 Administration in Duchenne Muscular Dystrophy (DMD) Patients

Sponsor

Shanghai Siponuoyin Biotechnology Co Ltd

Enrollment

9 participants

Start Date

Aug 6, 2025

Study Type

INTERVENTIONAL

Conditions

Duchenne Muscular Dystrophy (DMD)

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.

Eligibility

Sex: MALEMin Age: 2 YearsMax Age: 7 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying SPOT-03 for people with duchenne muscular dystrophy (dmd). The study is currently recruiting participants at 1 location. People eligible for this study include men aged 2 Years to 7 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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