Evaluating LE051 for treating Duchenne Muscular Dystrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study evaluates the safety, tolerability, and efficacy of LE051, an intravenous therapy, in patients with Duchenne Muscular Dystrophy (DMD) who are eligible for exon 51 skipping therapy. It is a single-arm, open-label trial that will assess the pharmacokinetics, pharmacodynamics, and immune response following a single infusion of LE051. The study aims to provide insights into the long-term safety and efficacy of this investigational treatment for DMD.

Why it matters

Who can join: the full rules

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.

Where it's running

• Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
  Shanghai, Shanghai Municipality, China Recruiting

Study contacts

• Study coordinator: Jiwen Wang
• Email: wangjiwen@scmc.com.cn
• Phone: +86 189 1661 3192

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.