RECRUITINGEarly Phase 1INTERVENTIONAL
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)
About This Trial
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.
Who May Be Eligible (Plain English)
Key Who May Qualify:
- 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 Who Should NOT Join This Trial:
- 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.
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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.
Treatments Being Tested
DRUG
LE051
LE051 dose escalation : dose 1 and dose 2.
Locations (1)
Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China