Clinical Trials in China / NCT06900049
Recruiting
Early Phase 1
Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy
NCT06900049 · tracked via the Priya Life Science China tracker
Condition(s) studied
Duchenne Muscular Dystrophy (DMD)
Investigational drug(s) / intervention(s)
LE051
LE051: LE051 dose escalation : dose 1 and dose 2.
Study 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
Key Inclusion Criteria:
* 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.
Key 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.
Primary outcome measure(s)
- Frequency of AEs, SAEs — from day 1 to week 52 after treatment
Trial sites (1)
| Facility | City | Region | Status |
|---|---|---|---|
| Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine | Shanghai | Shanghai Municipality | Recruiting |
More Shanghai Jiao Tong University School of Medicine trials in China
Other trials for the same condition
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Recruiting
A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy.
Active, not recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06900049 on ClinicalTrials.gov ↗ ← All trials in China