Natural history study of Angelman Syndrome

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study aims to gather comprehensive data on the long-term natural history of Angelman Syndrome in children and adults. By utilizing both investigator-observed and parent-reported outcome measures, the study seeks to establish Angelman-specific norms that can inform future clinical trials. The findings will enhance the understanding of the condition and ultimately improve care for individuals affected by Angelman Syndrome.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Molecular diagnosis of Angelman syndrome

Exclusion Criteria:

* Presence of another condition, unrelated to Angelman syndrome, that affects neurodevelopment

Where it's running

• Rady Children's Hospital, San Diego
  San Diego, California, United States Recruiting
• Children's Hospital Colorado, Anschutz Medical Campus
  Aurora, Colorado, United States Recruiting
• Emory University School of Medicine
  Atlanta, Georgia, United States Recruiting
• Rush University Children's Hospital
  Chicago, Illinois, United States Recruiting
• Boston Children's Hospital
  Boston, Massachusetts, United States Recruiting
• The Carolina Institute for Developmental Disabilities
  Carrboro, North Carolina, United States Recruiting

Study contacts

• Study coordinator: Wen-Hann Tan, BMBS
• Email: wen-hann.tan@childrens.harvard.edu
• Phone: 617-355 6394

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• 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

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.