Continued access to rugonersen for angelman syndrome
Part of Brain & nervous system, Genetic & congenital clinical trials.
This program provides continued access to the investigational drug rugonersen for people with Angelman syndrome who were previously in the TANGELO study. The goal is to keep offering treatment that may help manage symptoms, while closely monitoring safety.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You or your loved one must have completed the TANGELO study's optional open-label extension and its final safety visit.
- You must have a caregiver or legal representative who is at least 18, can come to clinic visits, and can answer questions.
- You must be able to have blood draws and spinal taps (lumbar punctures) with sedation or anesthesia if needed.
- You must not have taken any other experimental drug for Angelman syndrome since the last TANGELO dose.
- You must not have a history of severe headaches after a spinal tap or have had major surgery with general anesthesia in the last 12 weeks.
- Your medications for sleep, seizures, or mental health must be stable, and you must not use blood-thinning drugs within 2 weeks of starting.
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 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 medicine for Angelman syndrome (AS), a genetic condition affecting the nervous system. The medicine is given by lumbar puncture and aims to improve symptoms.
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 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 trial is building a global registry for people diagnosed with Angelman syndrome. It helps researchers learn more about how symptoms change over time, which can support future studies and treatments.
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.