RecruitingPhase 3NCT07605429

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

A Randomized, Multi-center, Double-blind, Sham-controlled, Phase III Clinical Study to Evaluate the Efficacy and Safety of Intrathecally Administered Rugonersen in Pediatric and Adult Participants With Angelman Syndrome

Sponsor

OHB Pediatrics Ltd.

Enrollment

165 participants

Start Date

Jun 23, 2026

Study Type

INTERVENTIONAL

Conditions

Angelman Syndrome

Summary

Purpose of the study is to evaluate the efficacy and safety of intrathecally administered rugonersen in pediatric and adult participants with Angelman syndrome.

Eligibility

Min Age: 1 YearMax Age: 50 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying Sham procedure and a drug called rugonersen for people with angelman syndrome. The study is currently recruiting participants at 2 locations. People eligible for this study include aged 1 Year to 50 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGrugonersen

Study Drug

PROCEDURESham procedure

Sham Procedure

Locations(3)

Rady Children's Hospital

San Diego, California, United States

Rush University

Chicago, Illinois, United States

University of North Carolina at Chapel Hill School of Medicine

Chapel Hill, North Carolina, United States

View Full Details on ClinicalTrials.gov

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NCT07605429

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