TAIN

Treatment of Adrenal Insufficiency in neonates- Development of a Hydrocortisone Preparation for the treatment of Adrenal Insufficiency in neonatesand infants

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 Obiettivo del progetto (Objective)

The aim of TAIN is to develop a neonatal formulation of hydrocortisone, a drug included in the EMA priority list that needs specific evaluation in the age range 0 – 2 years (neonates & infants). Hydrocortisone is an essential glucocorticoid hormone used as replacement therapy for the treatment of congenital and acquired adrenal insufficiency as well playing an important therapeutic role in oncology in infants, specifically brain tumours and leukaemia’s. TAIN involves European leaders in neonatology, paediatric pharmacology, methodology and SMEs that will establish links with ethical bodies and regulatory authorities. The programme will perform in silico experiments and evaluate formulations for neonates. The phase 3 clinical trial comparing the neonatal hydrocortisone versus current (unlicensed) therapy will be optimized using age-appropriate state-of-the-art methods adapted to neonates (including in silico experiments and pharmacokinetics) to validate the components of a Paediatric Investigation Plan. It will be performed by neonatologists trained in paediatric pharmacology and clinical research in line with guidelines on Good Clinical Practice. All the ethical issues will be considered, including pain and distress, blood sampling (number and volume) and informed consent. Parent information sheets and consent form will be submitted to patient and parents’ associations for approval. TAIN will include short term safety studies and Phase 3 clinical studies in neonates and infants. Results will be reported in order to allow a PUMA application to be submitted and to improve neonatal and infant care. Therefore, TAIN will validate the appropriate use of hydrocortisone in neonates and infants which will be of direct benefit to children, their families and health professionals. TAIN will strengthen paediatric drug evaluation across Europe and build up a network of units with experience in clinical research that will be used for additional drug evaluation in neonates.

Introduzione (Teaser)

EU-funded researchers have developed a new hydrocortisone formulation that can be safely administered to infants and children under six years of age suffering with adrenal insufficiency.

Descrizione progetto (Article)

Adrenal insufficiency is a rare disorder affecting approximately 1 in 14 000 people in Europe. Patients are unable to produce sufficient amounts of the hormone cortisol which is essential for physiological responses to challenges such as infection, injury or surgery.

The most common type of adrenal insufficiency in children is due to congenital adrenal hyperplasia where a genetic mutation blocks the production of cortisol. Although the glucocorticoid hormone hydrocortisone (the synthetic version of cortisol) has been successfully used as a replacement therapy for adrenal insufficiency, the drug formulation is not suitable for young children.

Seeking to address this issue, the http://www.tain-project.org (TAIN) project has developed a new hydrocortisone formulation that can be used in children from birth and up to six years of age. The formulation will be registered under the name Infacort and its characteristics have been adapted specifically to paediatric use, namely dosage, taste-masking and shelf life.

Given the very young target patient group of Infacort, the consortium will evaluate drug behaviour using computational models to minimise patient testing. Partners have obtained approval for their Paediatric Investigation Plan (PIP) from the European Medicines Agency, and are in the final stages of testing prior to drug approval.

GMP-grade Infacort formulation is currently being tested for safety and efficacy in a clinical trial involving paediatric patients. The outcome of this trial will reveal the suitability of this formulation for treatment of childhood adrenal insufficiency.