PK Study of Ticagrelor in Children Aged Less Than 24 Months, With Sickle Cell Disease (HESTIA4) (HESTIA4)
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|ClinicalTrials.gov Identifier: NCT03492931|
Recruitment Status : Completed
First Posted : April 10, 2018
Last Update Posted : June 10, 2019
The purpose of this Phase I study is to investigate the pharmacokinetic properties of ticagrelor in pediatric patients from 0 to less than 24 months with sickle cell disease.
Ticagrelor dose level adjustment will require a Protocol amendment and regulatory approval.
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease||Drug: Ticagrelor||Phase 1|
Study design This Phase I paediatric study (in patients aged 0 to <24 months) with ticagrelor is planned to be a multi-centre, open-label, single dose study.
To determine the PK properties of ticagrelor after a single oral dose
To determine the PK properties of the active metabolite (AR-C124910XX) after a single oral dose To assess the acceptability and the palatability of a single oral dose of ticagrelor
To assess safety and tolerability of a single oral dose of ticagrelor
Duration of treatment At least 20 eligible patients will receive a single open label oral dose of ticagrelor on Day 1.
Statistical methods A population PK analysis approach will be used to determine the PK parameters of ticagrelor and its metabolite AR-C124910XX in paediatric patients aged 0 to <24 months eg, CL/F (oral clearance) (only for ticagrelor) and AUC.
The PK will also be described by presenting the observed plasma concentrations of Ticagrelor and its active metabolite for all individuals, as well as corresponding descriptive statistics.
No statistical comparisons are planned for the primary or secondary objectives, which will be summarised descriptively
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||21 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Masking Description:||Study has an Open-Label design.|
|Official 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)|
|Actual Study Start Date :||March 28, 2018|
|Actual Primary Completion Date :||May 7, 2019|
|Actual Study Completion Date :||May 7, 2019|
Experimental: Treatment arm
Single dose of ticagrelor based on age
Patients will receive a single dose of ticagrelor
Other Name: AR-C124910XX is an active metabolite of ticagrelor given orally in a single dose. It will be measured, but it won't be given directly to subjects.
- Peak Plasma Concentration (Cmax) of Ticagrelor [ Time Frame: 1,2,4,6 hours post dose ]This measure is obtained from observed plasma concentrations
- Area under plasma concentration curve [ Time Frame: 1,2,4,6 hours post dose ]This measure is obtained from the population PK analysis
- CL/F (Oral clearance) [ Time Frame: 1,2,4,6 hours post dose ]This measure is obtained from the population PK analysis.
- Peak Plasma Concentration (Cmax) for active metabolite (AR-C124910XX) [ Time Frame: 1,2,4,6 hours post dose ]
- Area under plasma concentration curve [ Time Frame: 1,2,4,6 hours post dose ]
- Assessment of ticagrelor suspension for palatability [ Time Frame: Day 1, single timepoint assessment ]Questionnaire with one five-options question reflecting different degrees of patients willingness to swallow, from "swallowed without problem" to "vomited up medication".
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03492931
|Edegem, Belgium, 2650|
|Genova, Italy, 16100|
|Kisumu, Kenya, 40100|
|Nairobi, Kenya, 00100|
|Beirut, Lebanon, 11-0236|
|Tripoli, Lebanon, 1434|
|Madrid, Spain, 28007|
|London, United Kingdom, SE1 7EH|