D5136C00010 Hestia 4 - Phase I, open-label study of ticagrelor
Research type
Research Study
Full title
A Multi-centre, Phase I, Open-label, Single-dose Study to Investigate Pharmacokinetics (PK) of Ticagrelor in Infants and Toddlers, Aged 0 to less than 24 Months, with Sickle Cell Disease (HESTIA4)
IRAS ID
238440
Contact name
Baba Inusa
Contact email
Sponsor organisation
Astra Zeneca
Eudract number
2017-003641-14
Duration of Study in the UK
2 years, 5 months, 29 days
Research summary
This is a Phase I, multi-centre, open-label study for young children with Sickle Cell Disease (SCD), aged 0 to <24 months. SCD is an inherited genetic disorder that affects red blood cells, causing them to be sickle-shaped. Ticagrelor (BRILINTA) is an oral medication that prevents particular cells (platelets) in the blood from activating and causing blood clots. A vaso-occlusive crisis (VOC) is a severe painful episode (with a quick onset) that occurs when these sickle-shaped red blood cells block blood vessels, stopping oxygen reaching organs and/or tissue, resulting in organ damage and cell death.
This study will measure the level of Ticagrelor being absorbed by a child’s body (pharmacokinetics), after administration of a single dose, as well as the drugs safety and its tolerability. Only one single dose will be given to participants, meaning that no direct therapeutical benefit can be expected. The doses that have been selected for this study are based on the previous Ticagrelor studies, where no safety concerns were raised. In addition, patients participating in the study will not be withheld from any other standard of care treatments that may be used in SCD (e.g. hydroxyurea). Previous studies in SCD populations with anti-platelet medications have suggested potential beneficial effects.
REC name
London - Central Research Ethics Committee
REC reference
18/LO/0075
Date of REC Opinion
28 Feb 2018
REC opinion
Further Information Favourable Opinion