ClinicalTrials.gov record NCT05293184

The Global Angelman Syndrome Registry

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Aug 22, 2026

The Global Angelman Syndrome Registry

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
SexAll
LocationAustralia
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

An online, patient-organisation–driven registry that gathers information on the natural history of children and people with Angelman Syndrome. Caregivers or patients complete questionnaires over time to record diagnosis, clinical status, and patient-reported outcomes. The registry also supports recruitment for clinical trials and advances research and standards of care. It is available in multiple languages, including English, Spanish, Traditional Chinese, Italian, Polish, Hindi, and Brazilian Portuguese.

What it is studying. Whether and how health, symptoms, and quality-of-life change over time in people with Angelman Syndrome using repeated caregiver- and patient-reported information, and to create a resource that can support future research and trial recruitment.

What the researchers hope to learn. To create a long-term natural history dataset for Angelman Syndrome; to help researchers identify and recruit potential participants for studies; and to support research that may inform standards of care.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

The Global Angelman Syndrome Registry is an online patient organisation driven registry to collect information about the natural history of children and adults with Angelman Syndrome. The registry will facilitate 1) recruitment for clinical trials into therapies and interventions to benefit participants with Angelman Syndrome and their families, and 2) advancement of research and best standards of care for Angelman Syndrome. The registry is currently available in English, Spanish, Traditional Chinese, Italian, Polish, Hindi, and Brazilian Portuguese.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Other
  • Observational study only

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Diagnosis of Angelman Syndrome

Exclusion criteria

  • -

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants (or their parent/caregiver) complete online questionnaires about diagnosis, clinical status, and patient-reported outcomes. Parents/caregivers may be asked to complete additional questionnaire modules. Responses will be tracked over time, with a target follow-up period of up to 70 years. The registry is intended to support research and to facilitate recruitment for clinical trials.

How the study is structured. A prospective, observational online registry (case-only) run by a patient organisation. Participants (people with Angelman Syndrome, from birth through adulthood) or their caregivers join and complete questionnaires over many years (target duration 70 years). No experimental treatments are assigned—the study collects and stores longitudinal health and patient-/caregiver-reported data.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in Australia. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Angelman syndrome

Clinical trials11 trials listed for Angelman syndromeEvery trial the registry lists for Angelman syndrome, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Angelman syndrome researchLatest monthly update: September 2026. Includes: Seizure frequency. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Sep 28, 2016
Primary completion
Dec 31, 2099 (estimated)
Study completion
Dec 31, 2099 (estimated)
Target enrollment
5000 participants (estimated)
Last updated on registry
Sep 3, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Foundation for Angelman Syndrome Therapeutics, Australia
Principal investigator
Associate Professor Helen (Honey) Heussler, Associate Professor, Paediatrics and Child Health
The University of Queensland

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

  • Foundation for Angelman Syndrome Therapeutics, AustraliaRecruiting

    Cairns, Queensland, 4870 · Australia

Terms used on this page · explanations by MedicalUpdates.ai

Registry
An organised collection of information about people with a particular condition, gathered over time for research. Joining a registry usually means sharing medical information, not receiving a treatment.
natural history
A study that follows how a condition changes over time without assigning an experimental treatment. It may involve study visits and tests, and is often used to plan future trials.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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