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RECRUITING
NCT07188012
EARLY_PHASE1

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

Sponsor: Shanghai Siponuoyin Biotechnology Co Ltd

View on ClinicalTrials.gov

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.

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

Key Details

Gender

MALE

Age Range

2 Years - 7 Years

Study Type

INTERVENTIONAL

Enrollment

9

Start Date

2025-08-06

Completion Date

2026-12

Last Updated

2026-05-07

Healthy Volunteers

No

Interventions

GENETIC

SPOT-03

SPOT-03 injection administered via IV infusion

Locations (1)

Shanghai Children's Medical Center

Shanghai, Shanghai Municipality, China