Actively Recruiting
A Monocentric, Prospective, Longitudinal and Observational Natural History Study for Patients With Angelman Syndrome in CHR Citadelle Li e8ge NatHis-Angelman
Led by Centre Hospitalier Universitaire de Liege · Updated on 2025-05-20
30
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
Sponsors
C
Centre Hospitalier Universitaire de Liege
Lead Sponsor
C
Centre Hospitalier Régional de la Citadelle
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are conducting a 9-year natural history study for patients with Angelman syndrome, a rare genetic neurodevelopmental disorder. This study is based in Belgium and aims to better understand the progression and characteristics of Angelman syndrome by following patients over time. The study plans to enroll about 30 patients and track their development using standardized assessments and questionnaires. Participants will be seen every six months for five years. During these visits, they will undergo medical reviews including general and neurological examinations, as well as vital sign measurements like height, weight, heart rate, and blood pressure. Cognitive, language, and motor skills will be assessed using various age-appropriate tools such as Bayley-IV and HINE for younger children. Quality of life and developmental progress will also be evaluated through questionnaires. Throughout the study, researchers will collect detailed data on motor function, cognitive abilities, communication skills, and overall development. Continuous movement monitoring with a device called ActiMyo® will be used for two years. The study assesses multiple outcomes including functional mobility, developmental milestones, and quality of life measures. This observational study does not involve treatment but focuses on gathering information to enhance understanding of Angelman syndrome.
CONDITIONS
Brief Title
Natural History Study for Patients With Angelman Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Genetically confirmed diagnosis of Angelman syndrome
- Age from 0 to 99 years
- Male or female
- Participant's caregiver is willing to provide consent for participation
You will not qualify if you...
- Presence of a comorbidity that could affect the study results
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 5 years
Participants are observed over five years with medical and developmental assessments every six months.
Visits every 6 months for up to 5 years
Trial Site Locations
Total: 1 location
1
CHR Citadelle Liège
Liège, Liège, Belgium, 4000
Actively Recruiting
Research Team
N
Noor Benmhammed
T
Tamara Dangouloff, PhD
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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