Rugonersen

DRUG3 trials

Sponsors

F. Hoffmann-La Roche AG, Oak Hill Bio Ltd, OHB Pediatrics Ltd.

Conditions

Angelman SyndromeAngelman Syndrome (AS)

Phase 1

An Open-Label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO7248824 In Participants with Angelman Syndrome

CompletedCTIS2024-514797-45-00

F. Hoffmann-La Roche AGAngelman Syndrome (AS)

Start: 2020-08-25End: 2025-07-18Target: 28Updated: 2024-12-11

Phase 3

BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome.

RecruitingNCT07605429

OHB Pediatrics Ltd.Angelman Syndrome

Start: 2026-06-23End: 2031-03-01Target: 165Updated: 2026-09-10

Unknown Phase

Expanded Access/Compassionate Use of Rugonersen in Patients With Angelman Syndrome

Oak Hill Bio LtdAngelman Syndrome

Updated: 2025-08-22