drugset / Trial / NCT07480564
Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome
NaSingle-groupOpen-labelTreatment
Summary
The primary objectives of this study are to evaluate the safety, tolerability and preliminary efficacy of a single intrathecal (IT) dose of TSHA-102 in pediatric females with typical Rett syndrome.
Timeline
- Start
- 2026-05-08
- Primary completion
- 2031-06
- Completion
- 2031-06
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | LONIMECGENE RENPARVOVEC | Gene therapy (AAV / viral vector) | 1e+15 vg | Intrathecal |