Impact of Sulphonylureas on Neurodevelopmental Outcomes in KCNJ11-related Intermediate Developmental Delay, Epilepsy and Neonatal Diabetes (iDEND) Syndrome

Part of paid clinical trials in Chicago, Illinois.

Sponsor
Royal Devon and Exeter NHS Foundation Trust
Study ID
NCT05751525
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
2 Years - 50 Years
Healthy Volunteers
Not accepted

Interventions

  • Sulfonylurea — DRUG
    Glibenlclamide / glyburide

Study Details

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.

Key Dates

Start date
Jul 1, 2016
Status verified
Jun 2024
Primary completion
Aug 1, 2025
Completion
Dec 31, 2025

Study Design

Enrollment
21 participants (estimated)

Arms

  • Arm: Early SU treatment
    Patients with permanent neonatal diabetes (PNDM) due to the V59M mutation in the KCNJ11 gene who commenced sulfonylurea therapy in the first twelve months of life.
  • Arm: Late SU treatment
    Patients with permanent neonatal diabetes (PNDM) due to the V59M mutation in the KCNJ11 gene who commenced sulfonylurea older than the age of twelve months.

Primary Outcome Measure

Number and type of neurodevelopmental and psychiatric disorders [ Time Frame: At or up to 2 years after recruitment ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
University of ChicagoChicagoIllinois60637
Maria Salguero Bermonth

Find similar trials in Chicago, IL

By condition
ADHDAutism spectrum disorderEpilepsy
By specialty
PsychiatryMental healthBehavioral healthPediatricsNeurologyBrain disorders
By research site
University of Chicago· Chicago, IL

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