Actively Recruiting
Impact of Sulphonylureas on Neurodevelopmental Outcomes in KCNJ11-related Intermediate Developmental Delay, Epilepsy and Neonatal Diabetes (iDEND) Syndrome
Led by Royal Devon and Exeter NHS Foundation Trust · Updated on 2024-06-13
21
Participants Needed
4
Research Sites
495 weeks
Total Duration
On this page
Sponsors
R
Royal Devon and Exeter NHS Foundation Trust
Lead Sponsor
U
University of Chicago
Collaborating Sponsor
AI-Summary
What this Trial Is About
The goal of this observational study is to learn about the impact of the diabetes drug glibenclamide (glyburide) on neurodevelopment in individuals with iDEND (developmental delay, epilepsy and neonatal diabetes) due to the V59M mutation in the KCNJ11 gene. The main question it aims to answer is whether initiating sulphonylurea (SU) therapy in the first year of life results in better neurodevelopmental outcomes in affected individuals, in comparison to starting therapy later than 12 months of age. Participants will undergo a neurodevelopmental assessment comprising parental and teacher completion of standardised questionnaires, and where possible face to face neuropsychological testing. Researchers will compare the outcomes of these standardised tests in the individuals who started SU therapy \<12 months of age in comparison to those who started \>12 months of age.
CONDITIONS
Official Title
Impact of Sulphonylureas on Neurodevelopmental Outcomes in KCNJ11-related Intermediate Developmental Delay, Epilepsy and Neonatal Diabetes (iDEND) Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Current age 2 years or older
- Heterozygous for the V59M mutation in the KCNJ11 gene
- Successfully transferred to oral sulphonylurea therapy
- Willing to participate in the study
You will not qualify if you...
- Never able to transfer to oral sulphonylurea therapy
- Unwilling to participate in the study
AI-Screening
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Trial Site Locations
Total: 4 locations
1
University of Chicago
Chicago, Illinois, United States, 60637
Actively Recruiting
2
University of Rome
Rome, Italy
Actively Recruiting
3
University of Bergen
Bergen, Norway
Actively Recruiting
4
University of Exeter
Exeter, United Kingdom
Actively Recruiting
Research Team
D
Dr Pamela Bowman, MBBS MSc PhD
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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