Study of ION582 for people with Angelman syndrome
Part of Brain & nervous system, Genetic & congenital clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You or your child must have Angelman syndrome caused by a deletion or mutation in the UBE3A gene.
- You must be between 2 and 50 years old.
- You must be medically stable and able to have sedation or general anesthesia without a breathing tube.
- Medicines for seizures, behavior, sleep, or special diets must have been stable for at least 8 weeks before starting the study.
- You cannot have had previous treatment with gene therapy, gene editing, or another oligonucleotide (similar to ION582).
- You cannot have certain other brain or spinal conditions that would make a lumbar puncture unsafe.
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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