International Clinical Trial: A Single-Center, Single-Arm, Open-Label Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of RG001 Injection in the Treatment of Duchenne Muscular Dystrophy (DMD)

Geneva, April 20 -- International Clinical Trials Registry received information related to the study (ChiCTR2600121625) titled 'A Single-Center, Single-Arm, Open-Label Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of RG001 Injection in the Treatment of Duchenne Muscular Dystrophy (DMD)' on April 1.

Study Type: Interventional study

Study Design: Single arm

Primary Sponsor: Chinese PLA General Hospital

Condition: Duchenne Muscular Dystrophy,DMD

Intervention: Low-dose group (1.2x10^13 vg/kg):RG001 injection, single intravenous infusion, dose 1.2x10^13 vg/kg

Recruitment Status: Recruiting

Phase: 0

Date of First Enrollment: 2026-04-01

Target Sample Size: Low-dose group (1.2x10^13 vg/kg):3;High-dos...