drugset / Trial / NCT07181837
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
Non-randomizedSingle-groupOpen-labelTreatment
Summary
The purpose of this study is to evaluate the safety and efficacy of MVX-220 gene therapy in children and adults with Angelman syndrome with UBE3A gene deletion, uniparental disomy, or imprinting center defect genotypes.
Timeline
- Start
- 2025-10-29
- Primary completion
- 2028-03-31
- Completion
- 2031-05-31
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | MVX-220 | Unknown | — | Other |