LE051 Injection for Duchenne Muscular Dystrophy
LE051
Muscular Diseases+7
+ Muscular Dystrophies
+ Musculoskeletal Diseases
Treatment Study
Summary
Study start date: October 24, 2024
Actual date on which the first participant was enrolled.Duchenne Muscular Dystrophy (DMD) is a serious condition that causes muscles to weaken over time, often leading to disability and early death. This study is trying to find out if a new treatment called LE051 is safe and effective for DMD patients. LE051 is a special kind of therapy that targets a specific part of the gene that is faulty in about 13% of DMD cases. The goal is to help improve muscle function and slow down the progression of the disease by skipping over the problematic part of the gene. In this study, patients with DMD receive a single dose of LE051 through an intravenous infusion, which means it is given directly into the bloodstream. The research checks how well the body tolerates this medicine and if it helps with the symptoms of DMD. Researchers also study how the body processes the drug and how the immune system responds to it. They monitor the patients over a long period to ensure that the treatment is safe in the long run and to see if it continues to be effective.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.12 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Male
Biological sex of participants that are eligible to enroll.From 4 to 8 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Criteria
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.One single intervention group is designated in this study
This study does not include a placebo group
Treatment Groups
Group I
ExperimentalStudy Objectives
Primary Objectives
Secondary Objectives
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 1 location
Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
Shanghai, ChinaOpen Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine in Google Maps