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Ticagrelor Dose 1a + Dose 2a

Phase 2

Investigation of Platelet Aggregation in Paediatric Patients With Sickle Cell Disease | Small molecule | Hematology |AstraZeneca PLC|Last Updated: Dec 14, 2018

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindCONTROLLED
Total Trials1
Total Enrollment46
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02214121A Pharmacokinetic (PK) and Pharmacodynamic (PD) Dose-ranging Phase II Study of Ticagrelor in Paediatric Patients With Sickle Cell DiseasePHASE2 COMPLETED 46 — — Sep 11, 2014Feb 27, 2017Dec 14, 201818 United States, Canada +4
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Study Endpoints
Primary Endpoints
P2Y12 Reaction Units (PRU) - Part A
PRU measurements are taken in conjunction with single doses at Visit 2 (Day 0) and Visit 3 (Day 7) and after repeated dosing at Visit 4 (Day 14). Up to 8 hours post-dose (6 hours following protocol amendment) Visit 2 and 3, and up to 2 hours Visit 4.
P2Y12 Reaction Units (PRU) - Part B
PRU measurements are taken after 4 weeks of double blind treatment at the end of Part B.
Maximum Plasma Concentration (Cmax) - Part A
PK measurements (up to 8 hours post-dose) are taken in conjunction with single doses at Visit 2 (Day 0) and Visit 3 (Day 7) and after repeated dosing at Visit 4 (Day 14).
Maximum Plasma Concentration (Cmax) - Part B
PK measurements (up to 4 hours post-dose) are taken after 4 weeks of double blind treatment at the end of Part B.
Area Under the Plasma Concentration Time Curve (AUC) - Part A
PK measurements (up to 8 hours post-dose) are taken in conjunction with single doses at Visit 2 (Day 0) and Visit 3 (Day 7) and after repeated dosing at Visit 4 (Day 14).

The PK parameter presented was derived using a model based analysis and not from a non-compartmental (NCA) analysis.

Area Under the Plasma Concentration Time Curve (AUC) - Part B
PK measurements (up to 4 hours post-dose) are taken after 4 weeks of double blind treatment at the end of Part B.

The PK parameter presented was derived using a model based analysis and not from a non-compartmental (NCA) analysis.

Secondary Endpoints
Assessment of Ticagrelor Concentration - Part A
In conjunction with single doses at Visit 2 (Day 0) and Visit 3 (Day 7), after repeated dosing at Visit 4 (Day 14). Up to 8h post-dose (6h following protocol amendment, no pre-dose) Visit 2 and 3, up to 2h Visit 4 (pre-dose, 1h added following amendment)
Assessment of Ticagrelor Concentration - Part B
PK measurements (up to 4 hours post-dose) are taken after 4 weeks of double blind treatment at the end of Part B.
Assessment of AR-C124910XX Concentration - Part A
In conjunction with single doses at Visit 2 (Day 0) and Visit 3 (Day 7), after repeated dosing at Visit 4 (Day 14). Up to 8h post-dose (6h following protocol amendment, no pre-dose) Visit 2 and 3, up to 2h Visit 4 (pre-dose, 1h added following amendment)
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Ticagrelor Dose 1a + Dose 2aOTHERPart A: Ticagrelor Dose 1a and ticagrelor Dose 2a single doses + 1 week repeated dosing Part B: Ticagrelor or placebo 4 weeks repeated dosing.
Ticagrelor Dose 1b + Dose 2bOTHERPart A: Ticagrelor Dose 1b and ticagrelor Dose 2b single doses + 1 week repeated dosing. Part B: Ticagrelor or placebo 4 weeks repeated dosing.
Interventions
NameTypeDescription
Ticagrelor Dose 1a + Dose 2aDRUGTicagrelor 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 2bDRUGTicagrelor Dose 1b and ticagrelor Dose 2b single doses + 1 week ticagrelor repeted dosing followed by 4 weeks repeated dosing ticagrelor or placebo.
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Eligibility Criteria
Age Range2 Years — 17 Years
SexALL
Healthy VolunteersNo
Study Sites18

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...

Countries:United StatesCanadaKenyaLebanonSouth AfricaUnited Kingdom
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