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

RecruitingEARLY Phase 1Ages 4 Years - 8 YearsMaleShanghai Jiao Tong University School of Medicine
NCT06900049 on ClinicalTrials.gov โ†—
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In plain words

What the trial is, and whether it could suit you

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.

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๐Ÿ“‹ Eligibility rules (as published)
Key Inclusion Criteria: * Male๏ผŒ4-8 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%๏ผ› * The average FVC percentage of the predicted value is less than 40%๏ผ› * 12 lead ECG QT interval (QTc) \>0.45 seconds.
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What it tests

  • LE051
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Where

1 site
Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
Shanghai, China
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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