D5136C00009 - Hestia 3, Phase 3 study of Ticagrelor in Paediatric SCD
Research type
Research Study
Full title
A Randomised, Double-Blind, Parallel-Group, Multicentre, Phase III Study to Evaluate the Effect of Ticagrelor versus Placebo in Reducing the Rate of Vaso-Occlusive Crises in Paediatric Patients with Sickle Cell Disease (HESTIA3)
IRAS ID
242665
Contact name
Banu Kaya
Contact email
Sponsor organisation
AstraZeneca AB
Eudract number
2017-002421-38
Duration of Study in the UK
2 years, 5 months, 17 days
Research summary
This is an international, multi-centre, double-blind, randomised, parallel-group, placebo-controlled Phase 3 study to evaluate the effect of the study drug, ticagrelor, versus placebo in reducing the rate of vaso-occlusive crises (VOCs) and/or acute chest syndrome (ACS),which are common painful complications in children and teenagers with sickle cell disease (SCD). \n\nIn SCD cells in the blood called platelets are more active, causing them to become “sticky” and clump together which can obstruct blood vessels. This can lead to reduced blood flow to different parts of the body and can cause VOCs. VOCs are severe painful episodes that occur when these sickle-shaped red blood cells obstruct vessels, stopping oxygen reaching organs and/or tissue which can result in organ damage and cell death. Acute chest syndrome (ACS) can occur when sickle-shaped red blood cells obstruct the blood vessels of the lungs, causing difficulty with breathing, cough and fever.\n\nTicagrelor is an “antiplatelet” medication which is taken by mouth and aims to reduce the number of VOCs and ACS in the target population. Patients enrolled in the study will be randomly allocated to one of two treatment arms. Half of the patients enrolled will receive Ticagrelor and the other half will receive a placebo. Patients will take the study treatment (ticagrelor or placebo) twice daily. Each dose will be 15mg, 30mg or 45mg, the dose each patient takes will be dependent on their body weight. Patients will take the study treatment for at least 12 months, and up to approximately 24 months. \n
REC name
Wales REC 3
REC reference
18/WA/0138
Date of REC Opinion
27 Jun 2018
REC opinion
Further Information Favourable Opinion