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Tests treatment safety and results for Duchenne Muscular Dystrophy (DMD)
Official title Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy
ClinicalTrials.gov ID: NCT06900049
What this study is testing
What is LE051?
LE051 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for duchenne muscular dystrophy (dmd).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 to 8, men only
You may be able to join if
- Male，4-8 years old at the beginning of screening (including boundary values;
- DMD people with a clinical diagnosis of DMD referred to the Duchenne Clinical Practice Guidelines for Progressive Muscular Dystrophy (2020 edition)...
- The people and/or his guardian voluntary participate in this trial and can comprehend and sign ICF. Key
You likely can't join if
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Shanghai, Shanghai Municipality, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 4 years to 8 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shanghai, Shanghai Municipality, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.