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
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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Interventions
DRUGLE051
LE051 dose escalation : dose 1 and dose 2.
Locations(1)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06900049
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