Babies and children with adrenal insufficiency (in most cases they have the genetic condition congenital adrenal hyperplasia (CAH) need treatment with hydrocortisone because their bodies do not produce enough cortisol. There are currently no licensed therapies available for children aged 6 and under in Europe, so clinicians must resort to adapting adult formulations. TAIN is a clinical trial funded by the European Commission of an innovative new formulation of hydrocortisone specifically designed for young children.
TAIN is coordinated by the University of Sheffield and includes short term safety studies and a Phase 3 clinical study in neonates and infants. Further information can be found on the project website.
As part of this trial, and with additional funding from Diurnal, Genetic Alliance UK is working with parents of children affected by adrenal insufficiency to ensure that their experiences and opinions inform the development and introduction of treatments for children.
Our research consists of the following stages:
1. Literature review of the psychosocial impact of adrenal insufficiency in children.
2. Qualitative interviews with parents of affected children (UK), to explore the impact of the condition and current treatments.
3. Quantitative online survey to test the psychosocial findings more widely, and to gather health economic data. The survey was developed with the health economist Professor Simon Dixon (University of Sheffield).
4. Dissemination.
We have been delighted to work with the patient organisations Living with CAH (a Genetic Alliance UK member) and the Addison’s Disease Self-Help Group and the Dutch Adrenal Society (NVACP)
For further information please contact our Director of Research, Amy Hunter, or our Research Associate, Amy Simpson.
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