Clinical trials
Angelman Syndrome clinical trials
Below are recruiting angelman syndrome clinical trials, each written for real people, not researchers. We’re tracking 13 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07417137Recruiting
Long-term study of Angelman syndrome
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.
Lexington, MassachusettsAges 1 year+ - NCT06914609RecruitingPhase 3
Study of ION582 for people with Angelman syndrome
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.
Los Angeles, CaliforniaAges 2–50 - NCT07605429RecruitingPhase 3
Rugonersen treatment for Angelman syndrome
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.
San Diego, CaliforniaAges 1–50 - NCT06415344Enrolling by invitationPhase 3
Long-term extension of GTX-102 in Angelman syndrome
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.
Los Angeles, CaliforniaAges Any age - NCT07181837RecruitingPhase 1/Phase 2
Testing MVX-220 for Angelman Syndrome
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.
Los Angeles, CaliforniaAges 4–50 - NCT05293184Recruiting
Angelman syndrome registry to track symptoms worldwide
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.
Brisbane, QueenslandAges Any age - NCT06229769Recruiting
Angelman syndrome natural history study
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.
Liège, LiègeAges Any age - NCT06737718Recruiting
Eye tracking study for children with Angelman syndrome
This trial uses eye tracking to understand how children with Angelman syndrome perceive social cues. Participation helps researchers learn more about social perception for future treatments.
ParisAges 3–17 - NCT07157254RecruitingPhase 2
Study of GTX-102 for People with Angelman Syndrome
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.
Los Angeles, CaliforniaAges 1–64 - NCT04507997Recruiting
Study of the natural course of Angelman syndrome
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.
Los Angeles, CaliforniaAges Any age - NCT03836300Enrolling by invitation
Parent-infant coaching for families with young diagnoses
This study tests a parent-and-infant coaching program for babies and caregivers. It aims to see whether the program helps after an early diagnosis when English is used at home.
Research Triangle Park, North CarolinaAges Up to 99 years - NCT05945576Recruiting
RaDiCo IDMet study for people with inherited ID
This study enrolls people of all ages who have a confirmed inherited condition called ID. It collects information to better understand the condition and support future care.
AngersAges Any age - NCT06353620Recruiting
Brain connections study for certain epilepsies and genetic syndromes
This study uses MRI and a special EEG to understand brain connections in people with specific types of epilepsy or genetic conditions like Angelman syndrome and Dup15q. It may help find better ways to treat these conditions.
Conegliano, TrevisoAges 6–75
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Angelman Syndrome trials by city
Studies with a site in or near these metro areas.
Angelman Syndrome trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for angelman syndrome?
- Yes. Clin2 currently lists 13 recruiting angelman syndrome studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a angelman syndrome trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a angelman syndrome trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.