Ignite Creation Date:
2024-05-06 @ 11:50 AM
Last Modification Date:
2024-10-26 @ 12:51 PM
Study NCT ID:
NCT03615924
Status:
TERMINATED
Last Update Posted:
2021-04-09
First Post:
2018-06-22
Brief Title:
Effect of Ticagrelor vs Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease
Sponsor:
AstraZeneca
Organization:
AstraZeneca
Study Overview
Official 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
Status:
TERMINATED
Status Verified Date:
2021-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Recommendation from an independent data monitoring committee DMC and accepted by AstraZeneca
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
HESTIA3
Brief 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
Detailed Description:
Hestia3 will evaluate the efficacy safety and tolerability of ticagrelor versus placebo in children with SCD during treatment for at least 12 months and up to approximately 24 months
The target population are children aged 2 to 18 years of age and body weight of 12 kg diagnosed with HbSS or HbSβ0 confirmed by high-performance liquid chromatography or hemoglobin electrophoresis At least 50 evaluable patients should be recruited in each of the age groups 2 years to 12 years and 12 years to 18 years
To be eligible for the study patients must have experienced at least 2 VOCs defined as painful crisis andor ACS events in the past 12 months prior to Visit 1 indicating that the severity of the patients disease justifies preventive chronic long-term treatment The intent is to enroll only children aged 2 years or above since VOCs become more frequent with age
Study participants should receive standard of care for SCD adjusted to the individual patient at the discretion of the investigator including routine health care screening examinations and immunizations according to local guidelines and health care programmers Study drug will be given on the background of standard treatments for SCD Study participants are not withheld from any other treatments that may be used in SCD eg hydroxyurea during the trial which is important considering the use of a placebo control group However restrictions apply to some medications and interventions that may be necessary for the patients health and well-being during the study
Patients are to be followed up to 24 months or until a common study end date is reached defined as 12 months after the last patient is randomised The expected average follow-up is 18 months Considering inclusion of patients with at least 2 VOC events in the past year this treatment duration is considered long enough to evaluate effects on VOC events as well as to capture safety and tolerability data supporting a potential future long term use of ticagrelor
Due to ticagrelor mechanism of action and the potential to reduce symptoms caused by ischemia during a vaso-occlusion a composite endpoint with painful crises andor ACS has been selected for the primary endpoint Painful crisis is the most common reason for emergency department visits for patients with SCD with a significant impact on young patients lives affecting them physically and emotionally Secondary endpoints are included to broaden the understanding of effects in patients with SCD and to also assess potential benefits on symptomatic disease burden and health-related quality of life HRQL
Patients will be treated with 15 30 and 45 mg bd or matching placebo depending on body weight
The target population are children aged 2 to 18 years of age and body weight of 12 kg diagnosed with HbSS or HbSβ0 confirmed by high-performance liquid chromatography or hemoglobin electrophoresis At least 50 evaluable patients should be recruited in each of the age groups 2 years to 12 years and 12 years to 18 years
To be eligible for the study patients must have experienced at least 2 VOCs defined as painful crisis andor ACS events in the past 12 months prior to Visit 1 indicating that the severity of the patients disease justifies preventive chronic long-term treatment The intent is to enroll only children aged 2 years or above since VOCs become more frequent with age
Study participants should receive standard of care for SCD adjusted to the individual patient at the discretion of the investigator including routine health care screening examinations and immunizations according to local guidelines and health care programmers Study drug will be given on the background of standard treatments for SCD Study participants are not withheld from any other treatments that may be used in SCD eg hydroxyurea during the trial which is important considering the use of a placebo control group However restrictions apply to some medications and interventions that may be necessary for the patients health and well-being during the study
Patients are to be followed up to 24 months or until a common study end date is reached defined as 12 months after the last patient is randomised The expected average follow-up is 18 months Considering inclusion of patients with at least 2 VOC events in the past year this treatment duration is considered long enough to evaluate effects on VOC events as well as to capture safety and tolerability data supporting a potential future long term use of ticagrelor
Due to ticagrelor mechanism of action and the potential to reduce symptoms caused by ischemia during a vaso-occlusion a composite endpoint with painful crises andor ACS has been selected for the primary endpoint Painful crisis is the most common reason for emergency department visits for patients with SCD with a significant impact on young patients lives affecting them physically and emotionally Secondary endpoints are included to broaden the understanding of effects in patients with SCD and to also assess potential benefits on symptomatic disease burden and health-related quality of life HRQL
Patients will be treated with 15 30 and 45 mg bd or matching placebo depending on body weight
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
None