drugset / Trial / NCT05606614
A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)
Non-randomizedSequentialSingle-blindTreatment
Summary
The primary objectives of this study are to evaluate the safety of a single intrathecal (IT) dose of TSHA-102 in females with typical Rett syndrome, to select the TSHA-102 dose with the best benefit/risk profile based on the totality of safety and efficacy data and to evaluate the efficacy and safety of TSHA-102 at the selected dose.
Timeline
- Start
- 2023-03-06
- Primary completion
- 2031-06
- Completion
- 2031-06
Outcome
Met primary endpoint
release “Part A data from REVEAL Phase 1/2 trials presented at IRSF Scientific Meeting showed 100% response rate for pivotal trial primary endpoint” tayshagtx.com ↗
release “100% response rate post-TSHA-102 (May 2025 data cutoff) for the pivotal trial primary endpoint of the gain/regain of ≥ one defined developmental milestone” tayshagtx.com ↗
Publications
- Jagadeeswaran I, Oh J, Sinnett SE. Preclinical Milestones in MECP2 Gene Transfer for Treating Rett Syndrome. Dev Neurosci. 2025;47(2):147-156. doi: 10.1159/000539267. Epub 2024 May 9.
- Sadhu C, Lyons C, Oh J, Jagadeeswaran I, Gray SJ, Sinnett SE. The Efficacy of a Human-Ready miniMECP2 Gene Therapy in a Pre-Clinical Model of Rett Syndrome. Genes (Basel). 2023 Dec 24;15(1):31. doi: 10.3390/genes15010031.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | LONIMECGENE RENPARVOVEC | Gene therapy (AAV / viral vector) | 5.7e+14 vg | Intrathecal |
| Subject | LONIMECGENE RENPARVOVEC | Gene therapy (AAV / viral vector) | 1e+15 vg | Intrathecal |