Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease

Summary

The purpose of the study is to Evaluate the Effect of Ticagrelor versus Placebo in Reducing the Rate of Vaso-Occlusive Crises in Paediatric Patients with Sickle Cell Disease

Timeline

Start

2018-09-26

Primary completion

2020-08-13

Completion

2020-08-13

Outcome

Missed primary endpoint

Stopped: “Recommendation from an independent data monitoring committee (DMC) and accepted by AstraZeneca.”

paper The primary end point was not met: PMID 35849650 ↗

registry analysis (superiority test); Ticagrelor 15/30/45 mg bd vs Placebo; p = 0.7597; Incidence rate ratio 1.06 (95% CI 0.75 to 1.50); Negative binomial model NCT03615924 ↗

Publications

• Heeney MM, Abboud MR, Githanga J, Inusa BPD, Kanter J, Michelson AD, Nduba V, Musiime V, Apte M, Inati A, Taksande AM, Andersson M, Astrand M, Maklad N, Niazi M, Himmelmann A, Berggren AR. Ticagrelor vs placebo for the reduction of vaso-occlusive crises in pediatric sickle cell disease: the HESTIA3 study. Blood. 2022 Sep 29;140(13):1470-1481. doi: 10.1182/blood.2021014095.
• Heeney MM, Abboud MR, Amilon C, Andersson M, Githanga J, Inusa B, Kanter J, Leonsson-Zachrisson M, Michelson AD, Berggren AR; HESTIA3 study investigators. 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). Contemp Clin Trials. 2019 Oct;85:105835. doi: 10.1016/j.cct.2019.105835. Epub 2019 Aug 22.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ticagrelor	Small molecule	15 mg	Oral
Subject	Ticagrelor	Small molecule	30 mg	Oral
Subject	Ticagrelor	Small molecule	45 mg	Oral

Indications