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REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome

Join the REVEAL trial to study ION582 for Angelman Syndrome.

Recruiting
2-50 years
All
Phase 3
**Angelman Syndrome Study: ION582 Trial** This study is exploring a new treatment called **ION582** for Angelman syndrome, a genetic disorder affecting the brain caused by changes in the **UBE3A gene**. The study is conducted in four stages: a screening period, a 60-week treatment phase with either ION582 or a **placebo** (a fake treatment), a 25-month period where all get ION582, and an 8-month follow-up. Participants are split into two groups: one for ages 2-17 and another for ages 18-50. After the initial phase, everyone will get the real drug, ION582. The study ensures safety and checks how well ION582 works. **Key Points:** - The study lasts several years with different stages, including a long-term treatment period. - Participants will visit the clinic regularly for check-ups and treatments. - To join, participants must be stable on their current medications and have no severe health issues. Candidates must have Angelman syndrome confirmed by a specific genetic test and be between 2 and 50 years old. Previous gene therapies or major surgeries may exclude participation.
Study details
    Angelman Syndrome

NCT06914609

Ionis Pharmaceuticals, Inc.

2 May 2026

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