drugset / Trial / NCT07605429
BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome.
RandomizedParallel-groupQuadruple-blindTreatment
Summary
Purpose of the study is to evaluate the efficacy and safety of intrathecally administered rugonersen in pediatric and adult participants with Angelman syndrome.
Timeline
- Start
- 2026-06
- Primary completion
- 2029-03
- Completion
- 2031-03
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Rugonersen | Antisense oligonucleotide | — | Intrathecal |