Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Ticagrelor Dose 1a+ Dose 2a · 1 trial · 1 indication
The PK parameter presented was derived using a model based analysis and not from a non-compartmental (NCA) analysis.
The PK parameter presented was derived using a model based analysis and not from a non-compartmental (NCA) analysis.
| Arm | Type | Description |
|---|---|---|
| Ticagrelor Dose 1a + Dose 2a | OTHER | Part 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 2b | OTHER | Part A: Ticagrelor Dose 1b and ticagrelor Dose 2b single doses + 1 week repeated dosing. Part B: Ticagrelor or placebo 4 weeks repeated dosing. |
| Name | Type | Description |
|---|---|---|
| Ticagrelor Dose 1a + Dose 2a | 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 | DRUG | Ticagrelor Dose 1b and ticagrelor Dose 2b single doses + 1 week ticagrelor repeted dosing followed by 4 weeks repeated dosing ticagrelor or placebo. |
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...
Ticagrelor Dose 1a+ Dose 2a is being studied in pediatric patients with sickle cell disease, specifically to investigate platelet aggregation. It is a small molecule antiplatelet agent being evaluated in this hematology indication rather than for its approved cardiovascular uses.
Ticagrelor Dose 1a+ Dose 2a targets P2RY12, the platelet P2Y12 receptor. It acts as a negative allosteric modulator of this receptor, which is the mechanism behind its antiplatelet effect. Blocking P2RY12 signaling reduces platelet activation and aggregation.
Ticagrelor Dose 1a+ Dose 2a is being developed by AstraZeneca PLC, which trades under the ticker AZN. AstraZeneca is the sponsor behind the pediatric sickle cell disease program evaluating this dosing regimen.
Ticagrelor Dose 1a+ Dose 2a is in Phase 2 development. It is an investigational regimen and is not approved for the pediatric sickle cell disease indication. The Phase 2 study evaluating it has been completed.
Ticagrelor Dose 1a+ Dose 2a was studied in the Phase 2 trial NCT02214121, a pharmacokinetic and pharmacodynamic dose-ranging study in pediatric patients with sickle cell disease. That trial enrolled 46 patients across the United States, Canada, Kenya, Lebanon, South Africa, and the United Kingdom, and it is now completed.