RecruitingPhase 3NCT06914609

REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome

Phase 3 Study of the Efficacy and Safety of ION582 in Children and Adults With Angelman Syndrome

Sponsor

Ionis Pharmaceuticals, Inc.

Enrollment

158 participants

Start Date

Jun 10, 2025

Study Type

INTERVENTIONAL

Conditions

Angelman Syndrome

Summary

The purpose of this study is to evaluate the efficacy and safety of ION582 in children and adults with Angelman syndrome caused by a deletion or mutation of the UBE3A gene.

Eligibility

Min Age: 2 YearsMax Age: 50 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called ION582 and a drug called Placebo for people with angelman syndrome. The study is currently recruiting participants at 40 locations. People eligible for this study include aged 2 Years 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

DRUGION582

ION582 will be administered by IT injection.

DRUGPlacebo

ION582 matching placebo will be administered by IT injection.

Locations(40)

Cedars-Sinai Medical Center

Los Angeles, California, United States

Rady Children's Hospital

San Diego, California, United States

Colorado Children's Hospital Research Institute

Aurora, Colorado, United States

Children's National Hospital

Washington D.C., District of Columbia, United States

Nicklaus Children's Hospital

Miami, Florida, United States

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Rush University Medical Center

Chicago, Illinois, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Children's Mercy

Kansas City, Missouri, United States

Ichan School of Medicine at Mount Sinai

New York, New York, United States

Columbia University Irving Medical Center

New York, New York, United States

University of North Carolina at Chapel Hill School of Medicine

Carrboro, North Carolina, United States

Cincinnati Children's Hospital

Cincinnati, Ohio, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Vanderbilt Clinical Research Center

Nashville, Tennessee, United States

Texas Children's Hospital

Houston, Texas, United States

Queensland Children's Hospital

South Brisbane, Queensland, Australia

Perth Children's Hospital

Nedlands, Australia

Sydney Children's Hospital

Randwick, Australia

London Health Science Centre - Children's Hospital

London, Ontario, Canada

McGill University Health Centre

Montreal, Quebec, Canada

University of Alberta Hospital

Edmonton, Canada

British Columbia Children's Hospital

Vancouver, Canada

Klinikum der Ludwig-Maximilians-Universitaet Muenchen

München, Germany

Sheba Medical Center

Ramat Gan, Israel

Associazione La Nostra Famiglia - IRCCS Eugenio Medea

Conegliano, Italy

Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, Italy

Azienda Ospedaliero Universitaria Pisana

Pisa, Italy

Ospedale Pediatrico Bambino Gesù

Roma, Italy

Osaka Women's and Children's Hospital

Izumi, Osaka, Japan

National Center of Neurology and Psychiatry

Kodaira, Tokyo, Japan

Uniwersyteckie Centrum Kliniczne

Gdansk, Poland

KK Women's and Children's Hospital

Singapore, Singapore

Seoul National University Hospital

Seoul, South Korea

Samsung Medical Center

Seoul, South Korea

Hospital Sant Joan de Deu

Barcelona, Spain

Corporacio Sanitaria Parc Tauli - Hospital de Sabadell

Sabadell, Spain

Great Ormond Street Hospital for Children - NHS Foundation Trust

London, United Kingdom

John Radcliffe Hospital

Oxford, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT06914609

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