drugset / Trial / NCT06900049
Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2024-10-24
- Primary completion
- 2026-09-20
- Completion
- 2026-12-31
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | LE051 | Gene therapy (AAV / viral vector) | — | Intravenous |