RecruitingEarly Phase 1NCT06900049

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

Evaluation of the Safety, Tolerability, and Efficacy of a Single Intravenous Injection of LE051 in Patients With Duchenne Muscular Dystrophy (DMD)

Sponsor

Shanghai Jiao Tong University School of Medicine

Enrollment

12 participants

Start Date

Oct 24, 2024

Study Type

INTERVENTIONAL

Conditions

Duchenne Muscular Dystrophy (DMD)

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.

Eligibility

Sex: MALEMin Age: 4 YearsMax Age: 8 Years

Inclusion Criteria3

Male，4-8 years old at the beginning of screening (including boundary values;
DMD subjects with a clinical diagnosis of DMD referred to the Duchenne Clinical Practice Guidelines for Progressive Muscular Dystrophy (2020 edition) and whose genetic test results were confirmed to be applicable to exon skipping at No.51.
The subjects and/or his guardian voluntary participate in this trial and can comprehend and sign ICF.

Exclusion Criteria3

Clinical signs of heart failure: left ventricular ejection fraction (LVEF) <40%；
The average FVC percentage of the predicted value is less than 40%；
lead ECG QT interval (QTc) >0.45 seconds.

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