Actively Recruiting
Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy
Led by Shanghai Jiao Tong University School of Medicine · Updated on 2025-04-02
12
Participants Needed
1
Research Sites
114 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male, 4 to 8 years old at the start of screening
- Clinical diagnosis of Duchenne Muscular Dystrophy confirmed by genetic testing applicable to exon 51 skipping
- Voluntary participation with comprehension and signed informed consent by the subject or guardian
You will not qualify if you...
- Signs of heart failure with left ventricular ejection fraction below 40%
- Average forced vital capacity less than 40% of the predicted value
- QT interval corrected (QTc) longer than 0.45 seconds on 12-lead ECG
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China, 200127
Actively Recruiting
Research Team
J
Jiwen Wang
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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