Status:
RECRUITING
Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy
Lead Sponsor:
Shanghai Jiao Tong University School of Medicine
Conditions:
Duchenne Muscular Dystrophy (DMD)
Eligibility:
MALE
4-8 years
Phase:
EARLY_PHASE1
Brief 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.
Detailed Description
This is a single-arm, open-label study to evaluate the safety, tolerability, efficacy, pharmacokinetic, pharmacodynamic, and immune response of LE051 after a single intravenous infusion in DMD patient...
Eligibility Criteria
Inclusion
- Key
- 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
- 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.
Key Trial Info
Start Date :
October 24 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 31 2026
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT06900049
Start Date
October 24 2024
End Date
December 31 2026
Last Update
April 2 2025
Active Locations (1)
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1
Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China, 200127