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Study identifier: NCT07605429 Synced from ClinicalTrials.gov · July 29, 2026
● Recruiting

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

Condition: Angelman Syndrome  ·  Sponsor: OHB Pediatrics Ltd.

PhasePhase 3
Planned participants165
Who can joinAll sexes, 1 Year to 50 Years
Healthy volunteersNo

About this study

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

This description comes directly from the study's public registry record.

Talk to the study team

Brenda Vincenzi, MD  ·  +18573022294  ·  patientadvocacy@oakhillbio.com

Always discuss trial participation with your own doctor first.

Locations (2)

Rady Children's HospitalSan Diego, California, United StatesRecruiting
University of North Carolina at Chapel Hill School of MedicineChapel Hill, North Carolina, United StatesRecruiting

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Source record: clinicaltrials.gov/study/NCT07605429