Actively Recruiting
Evaluation of the Safety, Tolerability, and Efficacy of a Single Intravenous Injection of LE051 in Patients With Duchenne Muscular Dystrophy (DMD)
Led by Shanghai Jiao Tong University School of Medicine · Updated on 2025-04-02
12
Participants Needed
1
Research Sites
14 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and effectiveness of LE051 intravenous therapy in male patients with Duchenne Muscular Dystrophy (DMD) aged 4 to 8 years. DMD is a progressive muscle disorder caused by mutations in the dystrophin gene, leading to muscle weakness and disability. LE051 is an investigational gene therapy designed to induce exon 51 skipping, potentially benefiting about 13% of DMD patients. This is an early phase 1, single-arm, open-label study sponsored by Shanghai Jiao Tong University School of Medicine. Participants will receive a single intravenous infusion of LE051, which contains a genetic cassette delivered by an adeno-associated virus to target exon 51. The study monitors the pharmacokinetics, pharmacodynamics, immune response, safety, and long-term effects of this single dose. There is no placebo or comparison group, and the treatment is given once at the beginning of the study. During the 52 weeks following treatment, participants will have regular assessments including safety monitoring for adverse events, muscle function tests such as the North Star Ambulatory Assessment and various timed walk and climb tests, and muscle biopsies to measure dystrophin protein levels. Researchers will track changes compared to baseline to evaluate treatment impact. The study also collects data on serious adverse events and overall tolerability throughout the year.
CONDITIONS
Brief 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 beginning of screening
- Clinical diagnosis of Duchenne Muscular Dystrophy confirmed by genetic testing applicable to exon 51 skipping
- Voluntary participation with ability to understand and sign informed consent form
You will not qualify if you...
- Clinical signs of heart failure with left ventricular ejection fraction less than 40%
- Average forced vital capacity (FVC) percentage less than 40%
- QT interval longer than 0.45 seconds on 12-lead ECG
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Day 1 to Week 52 after treatment
Participants receive a single intravenous injection of LE051 and are monitored for safety, tolerability, and efficacy.
Regular visits during the 52 weeks after treatment for assessments
Duration - Up to Week 52 after treatment
Participants continue to be monitored for long-term safety and efficacy after treatment.
Ongoing assessments during the 52-week follow-up period
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
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here