Evaluating LE051 for treating Duchenne Muscular Dystrophy
Evaluation of the Safety, Tolerability, and Efficacy of a Single Intravenous Injection of LE051 in Patients With Duchenne Muscular Dystrophy (DMD)
This study is testing a new intravenous treatment called LE051 to see if it is safe and effective for people with Duchenne Muscular Dystrophy who can benefit from a specific type of therapy.
Quick facts
| Phase | Early Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 4 Years to 8 Years |
| Sex | Male |
| Sponsor | Shanghai Jiao Tong University School of Medicine Academic / other |
| Locations | 1 site (Shanghai, Shanghai Municipality) |
| Trial ID | NCT06900049 on ClinicalTrials.gov |
What this trial studies
This study evaluates the safety, tolerability, and efficacy of LE051, an intravenous therapy, in patients with Duchenne Muscular Dystrophy (DMD) who are eligible for exon 51 skipping therapy. It is a single-arm, open-label trial that will assess the pharmacokinetics, pharmacodynamics, and immune response following a single infusion of LE051. The study aims to provide insights into the long-term safety and efficacy of this investigational treatment for DMD.
Who should consider this trial
Good fit: Ideal candidates are male patients aged 4-8 years with a confirmed clinical diagnosis of DMD and genetic eligibility for exon 51 skipping.
Not a fit: Patients with severe heart failure or significant respiratory impairment may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve muscle function and quality of life for patients with DMD who qualify for exon 51 skipping.
How similar studies have performed: Other studies targeting exon skipping in DMD have shown promise, indicating potential for success with this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Shanghai, Shanghai Municipality
- Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine — Shanghai, Shanghai Municipality, China (Recruiting)
Study contacts
- Study coordinator: Jiwen Wang
- Email: wangjiwen@scmc.com.cn
- Phone: +86 189 1661 3192
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.