drugset / Trial / NCT07095712
Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS
Phase 1/2
Completed
1 enrolled
n-Lorem Foundation
Columbia University · collabThe Methodist Hospital Research Institute · collab
NaSingle-groupOpen-labelTreatment
Summary
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.
Timeline
- Start
- 2024-11-25
- Primary completion
- 2025-12-16
- Completion
- 2025-12-16
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | nL-TARD-001 | Antisense oligonucleotide | — | — |