Test drug safety for Angelman syndrome in young children
Part of Brain & nervous system, Genetic & congenital clinical trials.
This early-phase study tests a new experimental medicine called ION582 to see if it is safe, tolerable, and how the body processes it in people with Angelman syndrome. It may also look at early signs of how the medicine affects the brain and body, which could guide future treatments.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed, certified diagnosis of Angelman syndrome (from UBE3A deletion or mutation)
- You are between ages 0 and 50, and a parent/guardian can provide consent if needed
- You have been on stable usual care for at least 3 months (for example seizure medicines, sleep medicines, behavioral meds, gabapentin, cannabidiol, and/or diet or supplements)
- You will follow study rules and not post or share study details on social media until the study ends
- You do NOT have Angelman syndrome caused by certain specific genetic situations (paternal UPD or imprinting defect)
- You don’t have major uncontrolled health problems—especially poorly controlled seizures or recent status epilepticus
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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This trial tests a new medicine called ION582 for people with Angelman syndrome. It aims to see if it can help with symptoms and is given as a lumbar puncture (spinal tap). You may be able to join if you have a genetic diagnosis of Angelman syndrome and are between 2 and 50 years old.
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