drugset / Trial / NCT07157254
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
RandomizedParallel-groupOpen-labelTreatment
Summary
The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.
Timeline
- Start
- 2025-10-13
- Primary completion
- 2030-01
- Completion
- 2030-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | GTX-102 | Antisense oligonucleotide | — | Intrathecal |