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

Launched by SHANGHAI JIAO TONG UNIVERSITY SCHOOL OF MEDICINE · Mar 23, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called LE051 for boys with Duchenne Muscular Dystrophy (DMD), a genetic condition that causes muscle weakness. The goal is to see if LE051 is safe and effective for these patients, especially those already receiving a specific type of therapy called exon 51 skipping. The trial is currently looking for male participants aged 4 to 8 years old who have been diagnosed with DMD and meet certain genetic criteria.

If your child is eligible and chooses to participate, they will receive the LE051 treatment through an intravenous (IV) infusion. Throughout the trial, the researchers will monitor the child's health and how well the treatment works. It's important to note that children with serious heart issues or specific lung function problems may not be able to join the study. This trial aims to find better ways to help manage DMD and improve the quality of life for those affected by it.

Gender

MALE

Eligibility criteria

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