Condition

Angelman Syndrome clinical trials and research

• 10Clinical trials
• 12NIH research projects
• 8Countries

Newest Angelman Syndrome clinical trials

All recruiting trials (10)

• Obudanersen for Angelman Syndrome in UPD/ID patients

  This study tests obudanersen in children and adults with Angelman syndrome caused by UPD or imprinting defects to see if it improves expressive communication.

  Recruiting Phase 3 Ionis Pharmaceuticals, Inc. · Carrboro, North Carolina · NCT07782827

• Rugonersen in Angelman Syndrome (phase 3)

  This trial tests whether rugonersen given into the spine can help people with Angelman syndrome, compared with a sham procedure.

  Recruiting at 10 sites Phase 3 OHB Pediatrics Ltd. · Los Angeles, California and 9 other locations · NCT07605429

• Tracking changes in Angelman syndrome over 12 months

  This study will see how developmental skills, behavior, sleep, and brain activity change over one year in children and adults with a genetically confirmed diagnosis of Angelman syndrome.

  Recruiting Massachusetts General Hospital · Lexington, Massachusetts · NCT07417137

• MVX-220 gene therapy for Angelman syndrome

  This study will try a single dose of MVX-220 gene therapy injected into the cisterna magna for children and adults with Angelman syndrome caused by UBE3A deletion, uniparental disomy, or imprinting center defects.

  Recruiting at 3 sites Phase 1/2 MavriX Bio, LLC · Los Angeles, California and 2 other locations · NCT07181837

• Using eye tracking to understand social perception in children with Angelman syndrome

  This study is testing how children with Angelman syndrome see and understand social situations compared to healthy kids, using eye tracking technology to help find ways to measure their progress in future treatments.

  Recruiting Assistance Publique - Hôpitaux de Paris · Paris · NCT06737718

• Understanding brain connectivity in drug-resistant epilepsy and neurodevelopmental syndromes

  This study is testing how the brain connects in people with drug-resistant epilepsy and certain developmental disorders to see if it can help improve their treatment options.

  Recruiting IRCCS Eugenio Medea · Conegliano, Treviso · NCT06353620

• Natural history observation for Angelman syndrome patients

  This study is trying to learn more about how Angelman syndrome affects patients over nine years by regularly checking their motor skills, thinking abilities, and quality of life.

  Recruiting Centre Hospitalier Universitaire de Liege · Liège, Liège · NCT06229769

• Observing metabolic profiles in patients with imprinting disorders

  This study looks at the metabolic health of people, both adults and children, with imprinting disorders to see if there are common patterns and how these conditions affect their overall health.

  Recruiting at 8 sites Institut National de la Santé Et de la Recherche Médicale, France · Angers and 19 other locations · NCT05945576

• A registry for individuals with Angelman Syndrome

  This study is creating an online registry to collect information from people with Angelman Syndrome to help find better treatments and improve care for them and their families.

  Recruiting Foundation for Angelman Syndrome Therapeutics, Australia · Cairns, Queensland · NCT05293184

• Natural history study of Angelman Syndrome

  This study is trying to learn more about how Angelman Syndrome affects children and adults over time to help improve care and future treatments.

  Recruiting at 10 sites Boston Children's Hospital · Los Angeles, California and 10 other locations · NCT04507997

NIH-funded research (12)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Genome editor therapy for Angelman syndrome in young children

  A University of North Carolina team is exploring a gene-editing approach to turn on the silent UBE3A gene in people with Angelman syndrome to reduce symptoms.

  NIH research Univ of North Carolina Chapel Hill · Chapel Hill, United States · NIH-11512551

• MicroRNA pathways in Angelman Syndrome: novel targets for therapy

  A small study explores how specific microRNAs may influence Angelman syndrome and whether targeting them could lead to new treatments.

  NIH research Ohio State University · Columbus, UNITED STATES · NIH-11453242

• Delta brain rhythms in Angelman syndrome

  Scientists will study unusual brain wave patterns in Angelman syndrome to understand how these signals relate to behavior and guide future treatments for affected children and adults.

  NIH research Children's Research Institute · Washington, United States · NIH-11445746

• Targeting the stress response in Angelman syndrome

  Aims to understand how loss of a brain gene called UBE3A disrupts pathways in Angelman syndrome and to design therapies that calm the stress response in affected neurons.

  NIH research Washington University · Saint Louis, United States · NIH-11358877

• Small molecule therapy to turn on the paternal UBE3A gene in Angelman syndrome

  A small molecule drug is being developed to turn on the paternal UBE3A gene in Angelman syndrome, with the goal of improving brain function.

  NIH research Univ of North Carolina Chapel Hill · Chapel Hill, United States · NIH-11353907

• UBE3A mosaicism and Angelman syndrome phenotypes

  A study from UNC Chapel Hill will explore how the mix of neurons with active paternal UBE3A affects Angelman syndrome symptoms using a special mouse model and gene therapy tools.

  NIH research Univ of North Carolina Chapel Hill · Chapel Hill, United States · NIH-11343229

• How body‑sensing nerves affect walking in Angelman syndrome

  This project looks at whether problems with a nerve sensor called PIEZO2 cause balance and walking problems in people with Angelman syndrome.

  NIH research University of Texas Hlth Sci Ctr Houston · Houston, United States · NIH-11284016

• Understanding brain changes in Angelman syndrome

  This work explores how certain proteins and tiny cell packages called extracellular vesicles affect brain development in people with Angelman syndrome.

  NIH research Western University of Health Sciences · Pomona, United States · NIH-11130279

• Developing a small molecule treatment for Angelman syndrome

  This study is looking at a new treatment for Angelman syndrome that could help improve the lives of those affected by it, by testing a special molecule in mice to see how it affects their behavior and brain function before moving on to trials with people.

  NIH research Univ of North Carolina Chapel Hill · Chapel Hill, United States · NIH-11123115

• Understanding how sensory receptors affect movement in Angelman Syndrome

  This study is looking at how a specific protein called PIEZO2 affects movement and walking in people with Angelman Syndrome, hoping to find new ways to help improve their motor skills.

  NIH research University of Texas Hlth Sci Ctr Houston · Houston, United States · NIH-11030311

• Understanding how brain circuits affect seizures in Angelman syndrome

  This study is looking at how seizures happen in mice with Angelman syndrome, a condition that affects brain development, to find new ways to help people with this syndrome who often struggle with seizures.

  NIH research Univ of North Carolina Chapel Hill · Chapel Hill, United States · NIH-11025893

• Developing a new treatment for Angelman Syndrome using modified RNA technology

  This study is testing a new way to treat Angelman Syndrome by using special tools to help turn on a gene that’s not working properly in the brain, with the hope of improving development for those affected.

  NIH research Couragene, INC. · Edison, United States · NIH-10918729

Related topics

Quick answers

How many Angelman Syndrome clinical trials are recruiting right now?

10, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 8 countries. The most sites are in United States (5 trials), France (2 trials), Belgium (1 trial), Australia (1 trial), Canada (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 3: 2, Phase 1/2: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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