drugset / Trial / NCT04680065
GDNF Gene Therapy for Multiple System Atrophy
RandomizedParallel-groupQuadruple-blindTreatment
Summary
The objective of this randomized, double-blinded, placebo-controlled Phase 1 investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a possible or probable diagnosis of Multiple System Atrophy.
Timeline
- Start
- 2023-10-03
- Primary completion
- 2026-08
- Completion
- 2028-08
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AAV2-GDNF | Gene therapy (AAV / viral vector) | — | Other |