LE051 Exon 51 Skipping Therapy for Duchenne: LE051 Clinical Trial for DMD
Actively RecruitingEarly Phase 1Exon Skipping
Study Overview
- Age
- 4–8 years
- Phase
- Early Phase 1
- Sponsor
- Shanghai Jiao Tong University School of Medicine
- Therapeutic Approach
- Exon Skipping
- Variant Requirement
- 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.
- Eligible Sex
- Male
- Ambulation
- Ambulatory
- Study Start (Actual)
- 2024-10-24
- Primary Completion (Estimated)
- 2026-09-20
- Study Completion (Estimated)
- 2026-12-31
- Enrollment (Estimated)
- 12
- Countries
- China
Study Requirements and Criteria
Inclusion Criteria
- Male，4-8 years old at the beginning of screening (including boundary values;
- The subjects and/or his guardian voluntary participate in this trial and can comprehend and sign ICF.
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.
Contact Information
This section provides contact details for people who can answer questions about joining this study
- Name: Jiwen Wang
- Phone Number: +86 189 1661 3192
- Email: [email protected]
Clinical Trial Registry
NCT ID
NCT06900049This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.