Investigation of Platelet Aggregation in Paediatric Patients With Sickle Cell Disease Clinical Trial
Official title:
Multicenter, Open-label, Randomised, Pharmacokinetic (PK) and Pharmacodynamic (PD) Dose-ranging Phase II Study of Ticagrelor Followed by a Double-blind, Randomised, Parallel-group, Placebo-controlled 4 Weeks Extension Phase in Paediatric Patients With Sickle Cell Disease
The purpose of this Phase II dose-ranging study is to investigate pharmacokinetic (PK) and pharmacodynamic (PD) properties of various doses of ticagrelor followed by 4 weeks of twice-daily treatment in paediatric patients with sickle cell disease
This is a multicenter, open-label, dose-ranging study of ticagrelor followed by a double
blind, placebo-controlled extension phase in paediatric patients with sickle cell disease
(SCD).
Part A: Patients will be randomised 1:1 to receive one of two dosing schedules consisting of
two single weight-adjusted doses of ticagrelor. Pharmacokinetic (PK) parameters and
pharmacodynamic (PD) measurements will be determined following each dose. Platelet
aggregation will be measured using the VerifyNow™ P2Y12 assay.
Following these 2 single doses, all patients will receive open-label one-week treatment with
ticagrelor twice daily to determine tolerability prior to randomisation into Part B.
Part B: In this part patients will be randomised (2:1 ratio) to ticagrelor twice daily or
placebo for a 4-week treatment phase.
During the study, patients will be followed for the occurrence of vaso-occlusive crisis (VOC)
and for other disease manifestations such as daily pain, analgesic use and complications of
SCD.
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