Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy
Recruiting now · Early Phase 1
Conditions studied: Duchenne Muscular Dystrophy (DMD)
In brief
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.
Key facts
- Study ID
- NCT06900049
- Run by
- Shanghai Jiao Tong University School of Medicine
- People needed
- 12
- Starts
- 2024-10-24
- Expected to finish
- 2026-12-31
- Last updated by the study team
- 2025-04-02
Who can join
Age: 4 and older, up to 8. Sex: male. Healthy volunteers: not accepted.
Where it is running
- Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine — Shanghai, Shanghai Municipality, China (enrolling)
Full record on ClinicalTrials.gov
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