drugset / Trial / NCT06283212
A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome
Non-randomizedSequentialOpen-labelTreatment
Summary
EXPEDITION is a Phase 1/2 study in the UK to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet Syndrome aged 6 to \< 48 months. The study follows and open-label, dose-escalation design.
Timeline
- Start
- 2024-05-09
- Primary completion
- 2030-10
- Completion
- 2030-10
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | ETX101 | Gene therapy (AAV / viral vector) | — | — |