Sickle Cell Disease MedDRA version: 20.0 Level: LLT Classification code 10040644 Term: Sickle cell disease System Organ Class: 100000004850
Conditions
Sponsors
AstraZeneca AB
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female paediatric patients aged >=2 to =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. History of transient ischaemic attack (TIA) or cerebrovascular accident (ischaemic or haemorrhagic), severe head trauma, intracranial haemorrhage, intracranial neoplasm, arteriovenous malformation, aneurysm, or proliferative retinopathy. 2. Findings on TCD: Current or previous values for time averaged mean of the maximum velocity (TAMMV) that are Conditional or Abnormal. Patients with Conditional TAMMV values or higher (>=153 cm/sec using TCD imaging technique which is corresponding to >=170 cm/sec by the non-imaging technique). Both the middle cerebral artery and the internal carotid artery should be considered. Any other criteria that would locally be considered as TCD indications for chronic transfusion would also exclude the patient. 3. Active pathological bleeding or increased risk of bleeding complications according to Investigator 4. Haemoglobin 3 days per week that cannot be discontinued 8. Receiving chronic treatment with anticoagulants or antiplatelet drugs that cannot be discontinued 9. Moderate or severe hepatic impairment defined as laboratory values of alanine aminotransferase (ALT) >2 × upper limits of normal (ULN), total bilirubin (TB) >2 × ULN (unless judged by the Investigator to be caused by haemolysis), albumin 1.4, or symptoms of liver disease (eg, ascites) from test performed at Screening (Visit 1). 10. Renal failure requiring dialysis 11. Patient considered to be at risk of bradycardic events (eg, known sick sinus syndrome or second or third degree atrioventricular block) unless already treated with a permanent pacemaker. 12. Concomitant oral or intravenous therapy with strong or moderate cytochrome P450 3A4 (CYP3A4) inhibitors, CYP3A4 substrates with narrow therapeutic indices, or strong CYP3A4 inducers (see full list in Appendix G), which cannot be stopped at least 5 half-lives before randomisation. 13. Active untreated malaria. Patients with suspected malaria at Screening (Visit 1) will be tested. 14. Known hypersensitivity or contraindication to ticagrelor 15. Patients who are currently pregnant or breastfeeding, or planning to become pregnant during the study or have given birth less than 3 months prior to Screening (Visit 1) 16. Any condition which, in the opinion of the Investigator, would make it unsafe or unsuitable for the patient to participate in this study 17. Concern for the inability of the patient or caregiver (defined as legally authorized representative) to comply with study procedures and/or follow-up 18. Previous randomisation in the present study 19. Participation in another clinical study with an IP or device during the last 30 days preceding enrolment. 20. Involvement of member of patient’s family, or patient self, in the planning and/or conduct of the study (applies to both AstraZeneca staff and/or staff at the study site).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To compare the effect of ticagrelor vs placebo for the reduction of VOCs, which is the composite of painful crisis and/or ACS, in paediatric patients with SCD;Secondary Objective: - compare the effect of ticagrelor vs placebo for reduction of; painful crises, ACS, duration of painful crises, days hospitalised for VOC, days hospitalised for acute SCD complications - compare the effect of ticagrelor vs placebo on the number of VOCs requiring hospitalisation or emergency department visits - compare the effect of ticagrelor vs placebo on the number of acute SCD complications - compare the effect of ticagrelor vs placebo on the number of sickle cell-related red blood cell (RBC) transfusions - describe the health-related quality of life (HRQL) and fatigue - describe intensity of pain during VOC;Primary end point(s): Number of VOCs;Timepoint(s) of evaluation of this end point: Up to End of Study Visit {12 to 24 months} | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Number of painful crises - Number of ACSs - Duration of painful crises - Number of VOCs requiring hospitalisation or emergency department visits - Number of days hospitalised for VOC - Number of acute SCD complications - Number of days hospitalised for acute SCD complications - Number of sickle cell-related RBC transfusions - HRQL total score and by dimension using Paediatric Quality of Life Inventory (PedsQL) SCD Module and Fatigue total score and by dimension using the PedsQL Multidimensional Fatigue Scale - Intensity of worst pain daily during VOC;Timepoint(s) of evaluation of this end point: - Secondary end points will be measured up to Safety Follow up Visit (End of Study + 14 days) - HRQL (PedsQL) assessment will be performed at visits on day 0, 6mo, 12mo, 18mo, EOS (12-24 months) | — |
Countries
Belgium, Brazil, Canada, Egypt, Ghana, Greece, India, Italy, Kenya, Lebanon, Saudi Arabia, South Africa, Spain, Tanzania, United Republic of, Turkey, Uganda, United Kingdom, United States
Contacts
Public ContactInformation Centre
AstraZeneca AB
Outcome results
None listed