drugset / Trial / NCT06369974
Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy
Phase 1/2
Enrolling by invitation
1 enrolled
Massachusetts General Hospital
n-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 TUBB4A associated leukodystrophy.
Timeline
- Start
- 2024-09-18
- Primary completion
- 2026-06
- Completion
- 2026-06
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | nL-TUBB4-001 | Antisense oligonucleotide | — | — |