Ticagrelor versus placebo for the reduction of vaso-occlusive crises in pediatric sickle cell disease: Rationale and design of a randomized, double-blind, parallel-group, multicenter phase 3 study (HESTIA3)

An unmet need for therapies exists to reduce sickle cell disease (SCD) complications in pediatric patients. Activated platelets contribute to the formation of cellular aggregates during sickling and vaso-occlusive crises (VOCs). Ticagrelor is an oral, direct-acting, and reversible adenosine diphosphate P2Y12 receptor antagonist that inhibits platelet activation and aggregation. Although ticagrelor was well tolerated in two phase 2 studies in children and young adults with SCD, larger and longer-term treatment studies are needed to assess ticagrelor's efficacy to reduce VOCs.

Source: Contemporary Clinical Trials - August 21, 2019 Category: Radiology Authors: Matthew M. Heeney, Miguel R. Abboud, Carl Amilon, Marielle Andersson, Jessie Githanga, Baba Inusa, Julie Kanter, Maria Leonsson-Zachrisson, Alan D. Michelson, Anders R. Berggren, on behalf of the HESTIA3 study investigators Source Type: research