drugset / Trial / NCT07197268
Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation
Phase 1/2
Active not recruiting
1 enrolled
n-Lorem Foundation
University of North Carolina, Chapel Hill · collab
NaSingle-groupOpen-labelTreatment
Summary
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Bainbridge-Ropers Syndrome (BRPS) due to a pathogenic, de novo nonsense variant in ASXL3
Timeline
- Start
- 2025-05-19
- Primary completion
- 2027-05
- Completion
- 2027-05
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | nL-ASXL3-001 | Antisense oligonucleotide | — | — |
Indications
No indication recorded.