ClinicalTrials.gov record NCT06229769

Natural History Study for Patients With Angelman Syndrome

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

A Monocentric, Prospective, Longitudinal and Observational Natural History Study for Patients With Angelman Syndrome in CHR Citadelle Liège : NatHis-Angelman

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
LocationBelgium
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.

A single-center, long-term natural history study for people with Angelman Syndrome. The registry describes a 9-year programme (3-year recruitment, 5-year follow-up, 1 year analysis) with plans to include about 30 patients and regular visits every six months. Investigators will collect past and ongoing medical and developmental data using standard tests and caregiver questionnaires.

What it is studying. The study is describing the natural history of Angelman Syndrome by measuring motor skills, cognitive and communication development, quality of life, and daily movement patterns. It will collect retrospective and prospective clinical data and test whether standardized scales and continuous movement monitoring with ActiMyo® are suitable as outcome measures for future research.

What the researchers hope to learn. To characterize how motor, cognitive and communication abilities change over time in people with Angelman Syndrome. To collect and compare standard clinical scales and patient/caregiver measures. To evaluate movement-monitoring (ActiMyo®) and other assessment tools for use in future clinical trials.

What this study is about

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

This study is a 9-year natural history study for patients with Angelman syndrome in Belgium (a genetic neurodevelopmental disorder, affecting 500,000 individuals in the world). It includes a 3-year recruitment phase, a 5 year follow-up and a year to analyze the collected data. The investigators plan to include 30 patients with a semi-annual follow-up for 4 years. The investigators will collect relevant retrospective and prospective data using age-standardized scales and questionnaires for functional motor assessments and global developmental assessment.

Eligibility

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

Inclusion criteria

  • Genetically confirmed diagnosis of AS
  • 0-99 years
  • Male or Female
  • Participant's carer is willing to give IC/sign a "record of consultation" for participation in the study

Exclusion criteria

  • comorbidity that could potentially affect the results of the study coexists. This

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

Patients are seen every six months for five years. In these visits, patient have medical review (general medical examination, neurological examination), vital signs (height, weight, respiratory rate, heart rate, blood pressure), cognitive assessment (Bayley-IV), language assessment and questionaire (Bayley-IV, ORCA), motor assessments (Bayley-IV, FMS, Developmental milestones and HINE), quality of life questionnaire (PedsQL, CGI-CASS) and general development questionnaire (Vineland-II).

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 are seen every six months for up to five years for medical review and vital-sign measurements (height, weight, heart and respiratory rate, blood pressure). At visits they complete standardized cognitive, language and motor assessments (for example Bayley‑IV, Functional Mobility Scale, HINE for children under 2, and developmental milestones), caregiver interviews (Vineland‑II, ORCA) and quality-of-life questionnaires (PedsQL, CGI-CASS). Some participants will have continuous movement monitoring with a wearable device (ActiMyo®). Investigators will also collect relevant past medical information from records.

How the study is structured. A prospective, longitudinal, single-center observational (natural history) study. No treatments are given; participants are assessed at regular visits (about every six months) with medical exams, developmental tests (for example Bayley-IV, Vineland-II), caregiver questionnaires, and continuous movement monitoring.

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 Belgium. 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
Oct 10, 2021
Primary completion
January 2028 (estimated)
Study completion
January 2028 (estimated)
Target enrollment
30 participants (estimated)
Last updated on registry
May 20, 2025
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
Centre Hospitalier Universitaire de Liege
Principal investigator
Laurent Servais, Prof. Investigator of CRMN Liège, Principal investigator
Centre Hospitalier Universitaire de Liege
Collaborators
  • Centre Hospitalier Régional de la Citadelle
  • SYSNAV

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)

Terms used on this page · explanations by MedicalUpdates.ai

natural history study
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.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
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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