drugset / Trial / NCT07180355
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Non-randomizedParallel-groupOpen-labelTreatment
Summary
This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA). It will be delivered via dual intradentate nucleus (IDN) and intravenous (IV) administration to participants with FA. All participants will receive SGT-212 and will be enrolled in the study for approximately 5 years.
Timeline
- Start
- 2025-10-22
- Primary completion
- 2028-03-21
- Completion
- 2032-02-29
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | SGT-212 | Gene therapy (AAV / viral vector) | — | Other |