REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Angelman Syndrome

In brief

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.

Key facts

Study ID

NCT06914609

Run by

Ionis Pharmaceuticals, Inc.

People needed

158

Starts

2025-06-10

Expected to finish

2030-04-01

Last updated by the study team

2026-07-29

Who can join

Age: 2 and older, up to 50. Sex: any. Healthy volunteers: not accepted.

Where it is running

• John Radcliffe Hospital — Oxford, United Kingdom (enrolling)
• UCLA Clinical & Translational Research Center (CTRC) — Los Angeles, California, United States (enrolling)
• Rady Children's Hospital — San Diego, California, United States (enrolling)
• Colorado Children's Hospital Research Institute — Aurora, Colorado, United States (enrolling)
• Children's National Hospital — Washington D.C., District of Columbia, United States (enrolling)
• Nicklaus Children's Hospital — Miami, Florida, United States (enrolling)
• Children's Healthcare of Atlanta — Atlanta, Georgia, United States (enrolling)
• Rush University Medical Center — Chicago, Illinois, United States (enrolling)
• Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
• Children's Mercy — Kansas City, Missouri, United States (enrolling)
• Ichan School of Medicine at Mount Sinai — New York, New York, United States (enrolling)
• Columbia University Irving Medical Center — New York, New York, United States (enrolling)
• University of North Carolina at Chapel Hill School of Medicine — Carrboro, North Carolina, United States (enrolling)
• Cincinnati Children's Hospital — Cincinnati, Ohio, United States (enrolling)
• Nationwide Children's Hospital — Columbus, Ohio, United States (enrolling)
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
• Vanderbilt Clinical Research Center — Nashville, Tennessee, United States (enrolling)
• Texas Children's Hospital — Houston, Texas, United States (enrolling)
• Queensland Children's Hospital — South Brisbane, Queensland, Australia (enrolling)
• Austin Hospital — Heidelberg, Australia (enrolling)
• Perth Children's Hospital — Nedlands, Australia (enrolling)
• Sydney Children's Hospital — Randwick, Australia (enrolling)
• London Health Science Centre - Children's Hospital — London, Ontario, Canada (enrolling)
• McGill University Health Centre — Montreal, Quebec, Canada (enrolling)
• University of Alberta Hospital — Edmonton, Canada (enrolling)

Full record on ClinicalTrials.gov

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