drugset / Trial / NCT07410143
Investigation of Individualised Antisense Oligonucleotides (ASOs) in People With Unique Genetic Variants Causing Severely Debilitating, Life Threatening (SDLT) Central Nervous System (CNS) Conditions
NaSingle-groupOpen-labelTreatment
Summary
This study is being conducted to evaluate individualised antisense oligonucleotides (ASOs) in participants with severely debilitating, life threatening (SDLT) central nervous system (CNS) conditions caused by unique genetic variants amenable to correction by an ASO.
Timeline
- Start
- 2026-01-13
- Primary completion
- 2026-09-30
- Completion
- 2026-10-31
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Individualized ASO | Antisense oligonucleotide | — | — |
Indications
No indication recorded.