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RECRUITING
NCT06900049
EARLY_PHASE1

Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy

Sponsor: Shanghai Jiao Tong University School of Medicine

View on ClinicalTrials.gov

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

Interventions

DRUG

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