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Ticagrelor

Phase 2

Sickle Cell Disease | Small molecule | Hematology |AstraZeneca PLC|Last Updated: May 10, 2021

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-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials2
Total Enrollment108
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02482298A Study to Assess the Effect of Ticagrelor in Reducing the Number of Days With Pain in Patients With Sickle Cell DiseasePHASE2 COMPLETED 87Jul 9, 2015Nov 16, 2016Dec 19, 201820 United States, Egypt +6
NCT03492931PK Study of Ticagrelor in Children Aged Less Than 24 Months, With Sickle Cell Disease (HESTIA4)PHASE1 COMPLETED 21Mar 28, 2018May 7, 2019May 10, 20218 Belgium, Italy +4
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Study Endpoints
Primary Endpoints
Change in Proportion of Days With Pain Due to Sickle Cell Disease as Measured by an eDiary
Baseline through Week 12

To investigate the efficacy of 2 different doses of ticagrelor versus placebo in reducing the number of days with pain due to sickle cell disease.

Peak Plasma Concentration (Cmax) of Ticagrelor
1,2,4,6 hours post dose

This measure is obtained from observed plasma concentrations

Area under plasma concentration curve
1,2,4,6 hours post dose

This measure is obtained from the population PK analysis

CL/F (Oral clearance)
1,2,4,6 hours post dose

This measure is obtained from the population PK analysis.

Secondary Endpoints
Average of the Daily Worst Pain Values Reported Via eDiary
Baseline through Week 12
Change in Proportion of Days With Analgesic Use Measured by an eDiary
Baseline through Week 12
Peak Plasma Concentration (Cmax) for active metabolite (AR-C124910XX)
1,2,4,6 hours post dose
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposePREVENTION
Treatment Arms
ArmTypeDescription
Dose AEXPERIMENTAL -
Dose BEXPERIMENTAL -
PlaceboPLACEBO_COMPARATOR -
Treatment armEXPERIMENTALSingle dose of ticagrelor based on age
Interventions
NameTypeDescription
TicagrelorDRUGTwo arms: 1) 10 mg ticagrelor + 45 mg ticagrelor placebo or 2) 45 mg ticagrelor + 10 mg ticagrelor placebo. Drugs taken orally, twice a day (morning and evening, at least 12 hours apart) from randomization until the end of treatment.
PlaceboDRUG10 mg ticagrelor placebo + 45 mg ticagrelor placebo. Drugs taken orally, twice a day (morning and evening at least 12 hours apart) from randomization until the end of treatment
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Eligibility Criteria
Age Range18 Years — 30 Years
SexALL
Healthy VolunteersNo
Study Sites20

Inclusion Criteria: * Confirmed medical history or diagnosis of homozygous sickle cell (HbSS) or sickle beta-zero-thalassaemia (HbS/β0) by HPLC * If treated with hydroxyurea, the dose must have been stable for 3 months Exclusion Criteria: * History of transient ischaemic attack or clinically over...

Countries:United StatesEgyptFranceItalyKenyaLebanonTurkey (Türkiye)United KingdomBelgiumSpain
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