Evaluation of the Safety, Tolerability, and Efficacy of a Single Intravenous Injection of LE051 in Patients With Duchenne Muscular Dystrophy (DMD)
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Early Phase 1
SUMMARY
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.
Eligibility
Participation Requirements
Sex: Male
Minimum Age: 4
Maximum Age: 8
Healthy Volunteers: f
View:
• 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.
Locations
Other Locations
China
Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
RECRUITING
Shanghai
Contact Information
Primary
Jiwen Wang
wangjiwen@scmc.com.cn
+86 189 1661 3192
Time Frame
Start Date: 2024-10-24
Estimated Completion Date: 2026-12-31
Participants
Target number of participants: 12
Treatments
Experimental: LE051 treatment
Single dose intravenous injection of LE051
Related Therapeutic Areas
Sponsors
Leads: Shanghai Jiao Tong University School of Medicine