drugset / Trial / NCT06888661
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy
NaSingle-groupOpen-labelTreatment
Summary
The purpose of this trial is to evaluate safety and efficacy of intrathecal delivery of EXG001-307 as a treatment of spinal muscular atrophy .
Timeline
- Start
- 2025-03-10
- Primary completion
- 2026-03
- Completion
- 2026-12-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | EXG001-307 | Gene therapy (AAV / viral vector) | — | Intrathecal |