A registry for individuals with Angelman Syndrome
Official title: The Global Angelman Syndrome Registry
Run by Foundation for Angelman Syndrome Therapeutics, Australia · NCT05293184 · Data updated 2026-11-12
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.
Quick facts
| Study type | Observational |
|---|---|
| Sex | All |
| Enrollment | 5000 (estimated) |
| Sponsor | Foundation for Angelman Syndrome Therapeutics, Australia Academic / other |
| Locations | 1 site (Cairns, Queensland) |
| Status | Recruiting |
| Started | September 28, 2016 |
| Main results expected | December 31, 2099 |
| Study ends | December 31, 2099 |
| Registry record updated | September 3, 2026 |
| Trial ID | NCT05293184 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates for this registry are individuals diagnosed with Angelman Syndrome.
✕Probably not a fit
Patients without a diagnosis of Angelman Syndrome will not benefit from participation in this registry.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
The Global Angelman Syndrome Registry is an online platform designed to gather information about the natural history of individuals with Angelman Syndrome, both children and adults. This observational registry aims to facilitate recruitment for clinical trials focused on therapies and interventions that could benefit participants and their families. Additionally, it seeks to advance research and establish best practices for the care of those affected by Angelman Syndrome. The registry is accessible in multiple languages, including English, Spanish, Traditional Chinese, Italian, Polish, Hindi, and Brazilian Portuguese.
Why it matters
Potential benefit: If successful, this registry could enhance the understanding of Angelman Syndrome and improve recruitment for clinical trials, ultimately leading to better treatment options for patients.
How similar studies have gone: Other registries for rare diseases have shown success in facilitating research and improving patient outcomes, indicating that this approach is promising.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Diagnosis of Angelman Syndrome Exclusion Criteria: \-
Where it's running
-
Foundation for Angelman Syndrome Therapeutics, Australia
Cairns, Queensland, Australia Recruiting
Study contacts
- Principal investigator: Helen (Honey) Heussler, MBBS, FRACP DM — The University of Queensland
- Study coordinator: Megan Tones
- Email: curator@angelmanregistry.info
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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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.
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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.
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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.
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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.
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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.
-
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.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.