drugset / Trial / NCT07221760
Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation
Phase 1/2
Active not recruiting
1 enrolled
n-Lorem Foundation
Dell Children's Medical Center of Central Texas · collab
NaSingle-groupOpen-labelTreatment
Summary
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with dentatorubral-pallidoluysian atrophy (DRPLA) due to a heterozygous pathogenic CAG trinucleotide expansion in ATN1
Timeline
- Start
- 2025-11-06
- Primary completion
- 2027-11
- Completion
- 2027-11
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | nL-ATN1-001 | Antisense oligonucleotide | — | — |