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Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy
Sponsor: Shanghai Jiao Tong University School of Medicine
Summary
The purpose of this study is to evaluate the safety, tolerability, and efficacy of LE051 intravenous therapy in DMD patients treated with exon 51 skipping therapy.
Official title: Evaluation of the Safety, Tolerability, and Efficacy of a Single Intravenous Injection of LE051 in Patients With Duchenne Muscular Dystrophy (DMD)
Key Details
Gender
MALE
Age Range
4 Years - 8 Years
Study Type
INTERVENTIONAL
Enrollment
12
Start Date
2024-10-24
Completion Date
2026-12-31
Last Updated
2025-04-02
Healthy Volunteers
No
Conditions
Interventions
LE051
LE051 dose escalation : dose 1 and dose 2.
Locations (1)
Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China