drugset / Trial / NCT06314490
Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A
Phase 1/2
Active not recruiting
1 enrolled
University of California, San Diego
California Institute for Regenerative Medicine (CIRM) · collabn-Lorem Foundation · collab
NaSingle-groupOpen-labelTreatment
Summary
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single pediatric participant with SCN2A associated developmental epileptic encephalopathy
Timeline
- Start
- 2024-02-16
- Primary completion
- 2026-02-16
- Completion
- 2026-02-16
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | nL-SCN2A-002 | Antisense oligonucleotide | — | — |