Dragonfly Study: An International, Prospective, Longitudinal, Observational Natural History Study of Children and Adults Living With CTNNB1 Neurodevelopmental Syndrome
Status: Recruiting
Location: See all (2) locations...
Intervention Type: Diagnostic test
Study Type: Observational
SUMMARY
The aim of the Dragonfly study is to characterise and monitor the neurodevelopment of children and adults diagnosed with CTNNB1 syndrome through an international collaborative effort. Gaining comprehensive understanding of the mental, physical and social development of people with CTNNB1 neurodevelopmental syndrome and how their symptoms and abilities change over time will help improve and standardize care for these patients, as well as facilitate future research and clinical trials design.
Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:
• Clinically and genetically confirmed diagnosis of CTNNB1 syndrome.
• Age 0-99 years.
• Written informed consent/online consent to participate in study from a primary carer (parent or legal guardian).
Locations
Other Locations
Australia
Sydney Children's Hospital
NOT_YET_RECRUITING
Sydney
Slovenia
University Medical Centre Ljubljana
RECRUITING
Ljubljana
Contact Information
Primary
Damjan Osredkar, MD, PhD
damjan.osredkar@kclj.si
0038615229273
Time Frame
Start Date: 2024-06-14
Estimated Completion Date: 2030-01
Participants
Target number of participants: 250
Treatments
Children and Adults with CTNNB1 Neurodevelopmental Syndrome
Related Therapeutic Areas
Sponsors
Collaborators: University of Ljubljana School of Medicine, Slovenia, The University of New South Wales, CTNNB1 Foundation
Leads: University Medical Centre Ljubljana