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A Pharmacokinetic (PK) and Pharmacodynamic (PD) Dose-ranging Phase II Study of Ticagrelor in Paediatric Patients With Sickle Cell Disease

NCT ID: NCT02214121

Last Updated: 2018-12-14

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

46 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-09-11

Study Completion Date

2017-02-27

Brief Summary

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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

Detailed Description

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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.

Conditions

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Investigation of Platelet Aggregation in Paediatric Patients With Sickle Cell Disease

Keywords

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sickle cell anemia paediatric

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Ticagrelor Dose 1a + Dose 2a

Part A: Ticagrelor Dose 1a and ticagrelor Dose 2a single doses + 1 week repeated dosing Part B: Ticagrelor or placebo 4 weeks repeated dosing.

Group Type OTHER

Ticagrelor Dose 1a + Dose 2a

Intervention Type DRUG

Ticagrelor Dose 1a and ticagrelor Dose 2a single doses + 1 week ticagrelor repeated dosing followed by 4 weeks repeated dosing ticagrelor or placebo.

Ticagrelor Dose 1b + Dose 2b

Part A: Ticagrelor Dose 1b and ticagrelor Dose 2b single doses + 1 week repeated dosing. Part B: Ticagrelor or placebo 4 weeks repeated dosing.

Group Type OTHER

Ticagrelor Dose 1b + Dose 2b

Intervention Type DRUG

Ticagrelor Dose 1b and ticagrelor Dose 2b single doses + 1 week ticagrelor repeted dosing followed by 4 weeks repeated dosing ticagrelor or placebo.

Interventions

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Ticagrelor Dose 1a + Dose 2a

Ticagrelor Dose 1a and ticagrelor Dose 2a single doses + 1 week ticagrelor repeated dosing followed by 4 weeks repeated dosing ticagrelor or placebo.

Intervention Type DRUG

Ticagrelor Dose 1b + Dose 2b

Ticagrelor Dose 1b and ticagrelor Dose 2b single doses + 1 week ticagrelor repeted dosing followed by 4 weeks repeated dosing ticagrelor or placebo.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Children aged ≥2 to \<18 years of age
* Diagnosed with homozygous sickle cell (HbSS) or sickle beta-zero-thalassaemia (HbS/β0)

Exclusion Criteria

* At risk for haemorrhagic or bradycardic events
* Significant hepatic impairment
* Renal failure requiring dialysis
* Concomitant oral or intravenous therapy with strong CYP3A4 (cytochrome) inhibitors, CYP3A4 substrates with narrow therapeutic indices, or strong CYP3A4 inducers.
* Surgical procedure planned to occur during the study.
* Patients who are currently pregnant or breastfeeding or planning to become pregnant during the study.
* Patients who have known hypersensitivity or contraindication to ticagrelor.
Minimum Eligible Age

2 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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AstraZeneca

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Research Site

Orange, California, United States

Site Status

Research Site

Chicago, Illinois, United States

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Detroit, Michigan, United States

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Research Site

Hershey, Pennsylvania, United States

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Research Site

Philadelphia, Pennsylvania, United States

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Research Site

Charleston, South Carolina, United States

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Research Site

Toronto, Ontario, Canada

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Research Site

Kisian, , Kenya

Site Status

Research Site

Nairobi, , Kenya

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Research Site

Beirut, , Lebanon

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Research Site

Beirut, , Lebanon

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Research Site

Tripoli, , Lebanon

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Research Site

Parow, , South Africa

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Research Site

Rondebosch, , South Africa

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Cardiff, , United Kingdom

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Research Site

London, , United Kingdom

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Research Site

London, , United Kingdom

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Research Site

Manchester, , United Kingdom

Site Status

Countries

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Egypt Ghana Italy Turkey (Türkiye) United States Canada Kenya Lebanon South Africa United Kingdom

References

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Hsu LL, Sarnaik S, Williams S, Amilon C, Wissmar J, Berggren A; HESTIA1 Investigators. A dose-ranging study of ticagrelor in children aged 3-17 years with sickle cell disease: A 2-part phase 2 study. Am J Hematol. 2018 Dec;93(12):1493-1500. doi: 10.1002/ajh.25273. Epub 2018 Oct 2.

Reference Type BACKGROUND
PMID: 30187935 (View on PubMed)

Amilon C, Niazi M, Berggren A, Astrand M, Hamren B. Population Pharmacokinetics/Pharmacodynamics of Ticagrelor in Children with Sickle Cell Disease. Clin Pharmacokinet. 2019 Oct;58(10):1295-1307. doi: 10.1007/s40262-019-00758-0.

Reference Type DERIVED
PMID: 30972696 (View on PubMed)

Provided Documents

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Document Type: Statistical Analysis Plan

View Document

Document Type: Study Protocol

View Document

Other Identifiers

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2014-001006-18

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

D5136C00007

Identifier Type: -

Identifier Source: org_study_id