Protocol No: ECCT/18/06/02 Date of Protocol: 05-01-2018

Study 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)

Study Objectives:
Laymans Summary:
Abstract of Study:

Background: Sickle cell disease (SCD) is an autosomal recessive inherited disorder in which polymerization of deoxygenated sickle haemoglobin leads to decreased deformability of red blood cells (RBCs) and obstruction of the vasculature, producing episodes of pain, organ ischaemia and infarction and early mortality. Activated platelets promote the adherence of sickle cells to endothelial cells and thus participate in the vaso-occlusive process. Inhibition of platelet activation has been proposed as a potential therapeutic option in the treatment of children and adults with SCD. General Aim: The primary study objective is to determine the pharmacokinetic (PK) properties of ticagrelor, an anti-platelet agent, after a single oral dose in young children with SCD, aged 0 to <24 months. Methodology: This is a Phase I, multicentre, open-label, study of ticagrelor in children less than age 24 months diagnosed with SCD (Hb SS or Hb S). The treatment period consists of a single dose of ticagrelor (Day 1). Following the dose of ticagrelor, four PK samples will be taken from each patient at 1, 2, 4 and 6 hours post-dose. There will be a subsequent follow up visit between Day 4 and Day 8. During the study, patients will be evaluated for adverse events (AEs)/serious adverse events (SAEs), laboratory safety samples and vital signs. Expected Outcome: The study results will provide PK data on ticagrelor in young children with SCD, as an important step towards determining the usefulness of ticagrelor in young children with SCD.