Angel syndrome treatment study for children and adults
Part of Brain & nervous system, Genetic & congenital clinical trials.
This study tests an experimental medicine for Angelman syndrome (AS), a genetic condition affecting the nervous system. The medicine is given by lumbar puncture and aims to improve symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of Angelman syndrome due to either paternal UPD or imprinting defect, but not due to a UBE3A deletion or mutation.
- You must be between 2 and 50 years old, depending on the study group.
- You must be medically stable enough to undergo sedation or general anesthesia without needing a breathing tube.
- If you are on medications for AS symptoms (like anti-seizure or sleep aids), they must have been stable for at least 8 weeks before the study starts.
- You must not have had prior treatment with gene therapy or similar experimental medicines.
- You must agree that your caregiver(s) will not share your personal medical information on social media during the study.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
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.
This study follows people with Angelman syndrome over time to learn more about the condition. It does not test any new treatments, but you need a parent or caregiver to come with you to all visits.
This study tests a new medicine called rugonersen for people with Angelman syndrome. The medicine is given as an injection into the fluid around the spinal cord and may help improve symptoms by targeting the genetic cause of the condition.
This study tests an experimental drug called GTX-102 for people with Angelman syndrome. It aims to see if the drug is safe and can help with symptoms. The trial includes different age groups and genetic types of Angelman syndrome.
This study follows people with Angelman syndrome over time to learn more about the condition. You can join if you have a confirmed genetic diagnosis and your caregiver agrees to take part.
This study tests an experimental medicine called MVX-220 to see if it is safe and effective for people with Angelman Syndrome. It includes both children (ages 4-8) and adults (ages 18-50) who can walk independently and have stable seizure medications.
Hear when a new Angelman Syndrome trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.