NCT06900049RecruitingEarly phase 1
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)
Source: ClinicalTrials.gov · Last updated 2 Apr 2025 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Early phase 1
- Study type
- Interventional
- Enrollment
- 12 (estimated)
- First submitted
- 23 Mar 2025
- Start date
- 24 Oct 2024
- Primary completion date
- 20 Sept 2026
- Lead sponsor
- Shanghai Jiao Tong University School of Medicine
- Diseases
- Duchenne muscular dystrophy
- NCT number
- NCT06900049
What you commit to
- Study type: Interventional · Early phase 1
- Planned participants: 12
- From 24 Oct 2024 to the primary completion date, 20 Sept 2026
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Lab values
- Clinical signs of heart failure: left ventricular ejection fraction (LVEF) \<40%；
- 12 lead ECG QT interval (QTc) \>0.45 seconds.
Other
- The average FVC percentage of the predicted value is less than 40%；
Who the study is looking for
- 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.
Age in the registry: 4 years to 8 years · Sex: Male · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine Coordinator of this site? Confirm its status | Shanghai | China | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.