Long-term extension of GTX-102 in Angelman syndrome
Part of Brain & nervous system, Genetic & congenital clinical trials.
This study is a long-term follow-up for people with Angelman syndrome who have already taken part in a GTX-102 trial. It tests whether the treatment continues to be safe and helpful over a longer period.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You or your child must have completed the final visit in a previous GTX-102 trial.
- The screening visit must happen within 6 months of that last visit.
- If you have any ongoing side effects from the prior trial, the study doctor must approve continuing.
- Females who can become pregnant must use effective birth control or avoid sex during the study and for 6 months after the last dose.
- Males must agree to use birth control or avoid sex during the study and for 3 months after the last dose.
- You cannot join if you stopped early from a previous GTX-102 trial.
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 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.
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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.
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 follows people with Angelman syndrome to understand how their symptoms and development change over time. It may help researchers better describe the condition and support future treatments.
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.
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