| Primary | P2Y12 Reaction Units (PRU) - Part A | | The PD analysis set is a subset of the safety analysis set, including all patients having at least one PRU measured. All patients who received at least one single dose of ticagrelor were included in the safety population (SAF) for Part A. Analysis on the SAF was based on the study medication actually received. | Posted | | Mean | Standard Deviation | P2Y12 reaction units | | 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. | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 0.125 mg/kg | Single dose received at Visit 2. | | OG001 | Ticagrelor 0.75 mg/kg | Single dose received at Visit 2. | | OG002 | Ticagrelor 0.375 mg/kg | Single dose received at Visit 3. | | OG003 | Ticagrelor 0.563 mg/kg | Single dose received at Visit 3 | | OG004 | Ticagrelor 1.125 mg/kg | Single dose received at Visit 3. | | OG005 | Ticagrelor 2.25 mg/kg | Single dose received at Visit 3. | | OG006 | Ticagrelor 0.125 mg/kg Bid | Repeated bid treatment between Visit 3 and Visit 4. | | OG007 | Ticagrelor 0.563 mg/kg Bid | Repeated bid treatment between Visit 3 and Visit 4. | | OG008 | Ticagrelor 0.75 mg/kg Bid | Repeated bid treatment between Visit 3 and Visit 4. |
| | Units | Counts |
|---|
| Participants | - OG00014
- OG00131
- OG0027
- OG003
|
| | Title | Denominators | Categories |
|---|
| Pre-dose | - ParticipantsOG00014
- ParticipantsOG00130
- ParticipantsOG0027
- ParticipantsOG003
|
| |
| Primary | P2Y12 Reaction Units (PRU) - Part B | | The PD analysis set is a subset of the safety analysis set, including all patients having at least one PRU measured. All patients who received at least one dose of randomised study drug, ticagrelor or placebo, will be included in the SAF for Part B. Analysis on the SAF was based on the study medication actually received. | Posted | | Mean | Standard Deviation | P2Y12 reaction units | | PRU measurements are taken after 4 weeks of double blind treatment at the end of Part B. | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 0.125 mg/kg Bid | Repeated bid treatment during Part B. | | OG001 | Placebo | Repeated bid treatment during Part B. |
| |
| Primary | Maximum Plasma Concentration (Cmax) - Part A | | The PK analysis set is a subset of the safety analysis set, including all patients having at least one PK variable calculated. All patients who received at least one single dose of ticagrelor were included in the safety population (SAF) for Part A. Analysis on the SAF was based on the study medication actually received. | Posted | | Geometric Mean | Standard Deviation | ng/mL | | 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). | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 0.125 mg/kg | Single dose received at Visit 2. | | OG001 | Ticagrelor 0.75 mg/kg | Single dose received at Visit 2. | | OG002 | Ticagrelor 0.375 mg/kg | Single dose received at Visit 3. | | OG003 | Ticagrelor 0.563 mg/kg | Single dose received at Visit 3 | | OG004 |
|
| Primary | Maximum Plasma Concentration (Cmax) - Part B | | The PK analysis set is a subset of the safety analysis set, including all patients having at least one PK variable calculated. All patients who received at least one dose of randomised study drug, ticagrelor or placebo, will be included in the SAF for Part B. Analysis on the SAF was based on the study medication actually received. | Posted | | Geometric Mean | Standard Deviation | ng/mL | | PK measurements (up to 4 hours post-dose) are taken after 4 weeks of double blind treatment at the end of Part B. | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 0.125 mg/kg Bid | Repeated bid treatment during Part B. |
| |
| Primary | Area Under the Plasma Concentration Time Curve (AUC) - Part A | The PK parameter presented was derived using a model based analysis and not from a non-compartmental (NCA) analysis. | The PK analysis set is a subset of the safety analysis set, including all patients having at least one PK variable calculated. All patients who received at least one single dose of ticagrelor were included in the safety population (SAF) for Part A. Analysis on the SAF was based on the study medication actually received. | Posted | | Geometric Mean | Standard Deviation | ng*h/mL | | 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). | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 0.125 mg/kg | Single dose received at Visit 2. | | OG001 | Ticagrelor 0.75 mg/kg | Single dose received at Visit 2. | | OG002 | Ticagrelor 0.375 mg/kg | Single dose received at Visit 3. | | OG003 | Ticagrelor 0.563 mg/kg | |
|
| Primary | Area Under the Plasma Concentration Time Curve (AUC) - Part B | The PK parameter presented was derived using a model based analysis and not from a non-compartmental (NCA) analysis. | The PK analysis set is a subset of the safety analysis set, including all patients having at least one PK variable calculated. All patients who received at least one dose of randomised study drug, ticagrelor or placebo, will be included in the SAF for Part B. Analysis on the SAF was based on the study medication actually received. | Posted | | Geometric Mean | Standard Deviation | ng*h/mL | | PK measurements (up to 4 hours post-dose) are taken after 4 weeks of double blind treatment at the end of Part B. | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 0.125 mg/kg Bid | Repeated bid treatment during Part B. |
| |
| Secondary | Assessment of Ticagrelor Concentration - Part A | | The PK analysis set is a subset of the safety analysis set, including all patients having at least one PK variable calculated. All patients who received at least one single dose of ticagrelor were included in the safety population (SAF) for Part A. Analysis on the SAF was based on the study medication actually received. | Posted | | Geometric Mean | Standard Deviation | ng/mL | | 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) | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 0.125 mg/kg | Single dose received at Visit 2. | | OG001 | Ticagrelor 0.75 mg/kg | Single dose received at Visit 2. | | OG002 | Ticagrelor 0.375 mg/kg | Single dose received at Visit 3. | | OG003 | Ticagrelor 0.563 mg/kg | Single dose received at Visit 3 |
|
| Secondary | Assessment of Ticagrelor Concentration - Part B | | The PK analysis set is a subset of the safety analysis set, including all patients having at least one PK variable calculated. All patients who received at least one single dose of ticagrelor were included in the safety population (SAF) for Part A. Analysis on the SAF was based on the study medication actually received. | Posted | | Geometric Mean | Standard Deviation | ng/mL | | PK measurements (up to 4 hours post-dose) are taken after 4 weeks of double blind treatment at the end of Part B. | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 0.125 mg/kg Bid | Repeated bid treatment during Part B. |
| |
| Secondary | Assessment of AR-C124910XX Concentration - Part A | AR-C124910XX is the active metabolite of Ticagrelor | The PK analysis set is a subset of the safety analysis set, including all patients having at least one PK variable calculated. All patients who received at least one single dose of ticagrelor were included in the safety population (SAF) for Part A. Analysis on the SAF was based on the study medication actually received. | Posted | | Geometric Mean | Standard Deviation | ng/mL | | 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) | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 0.125 mg/kg | Single dose received at Visit 2. | | OG001 | Ticagrelor 0.75 mg/kg | Single dose received at Visit 2. | | OG002 | Ticagrelor 0.375 mg/kg | Single dose received at Visit 3. | | OG003 | Ticagrelor 0.563 mg/kg | |
|
| Secondary | Assessment of AR-C124910XX Concentration - Part B | AR-C124910XX is the active metabolite of Ticagrelor | The PK analysis set is a subset of the safety analysis set, including all patients having at least one PK variable calculated. All patients who received at least one single dose of ticagrelor were included in the safety population (SAF) for Part A. Analysis on the SAF was based on the study medication actually received. | Posted | | Geometric Mean | Standard Deviation | ng/mL | | PK measurements (up to 4 hours post-dose) are taken after 4 weeks of double blind treatment at the end of Part B. | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 0.125 mg/kg Bid | Repeated bid treatment during Part B. |
| |
| Secondary | Oral Clearance (CL/F) - Part A | The PK parameter presented were derived using a model based analysis and not from a non-compartmental (NCA) analysis. | The PK analysis set is a subset of the safety analysis set, including all patients having at least one PK variable calculated. All patients who received at least one single dose of ticagrelor were included in the safety population (SAF) for Part A. Analysis on the SAF was based on the study medication actually received. | Posted | | Geometric Mean | Standard Deviation | L/h | | 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). | | | | ID | Title | Description |
|---|
| OG000 | Overall | All patients receiving Ticagrelor during Part A. |
| |
| Secondary | Oral Clearance (CL/F) - Part B | The PK parameter presented was derived using a model based analysis and not from a non-compartmental (NCA) analysis. | The PK analysis set is a subset of the safety analysis set, including all patients having at least one PK variable calculated. All patients who received at least one dose of randomised study drug, ticagrelor or placebo, will be included in the SAF for Part B. Analysis on the SAF was based on the study medication actually received. | Posted | | Geometric Mean | Standard Deviation | L/h | | PK measurements (up to 4 hours post-dose) are taken after 4 weeks of double blind treatment at the end of Part B. | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 0.125 mg/kg Bid | Repeated bid treatment during Part B. |
| |
| Secondary | Number of Vaso-occlusive Crises - Part B | | All patients randomised in Part B were to be included in the efficacy analysis set (EAS). Patients were analysed according to their randomised study medication. | Posted | | Mean | Standard Deviation | Number of events | | During 4 weeks of study treatment starting from randomization in Part B (week 2) up to 4 weeks (week 6). | | | | ID | Title | Description |
|---|
| OG000 | Part B - Ticagrelor | Randomised treatment group for Part B of the study. Relevant for the Part B period. | | OG001 | Part B - Placebo | Randomised treatment group for Part B of the study. Relevant for the Part B period. |
| |
| Secondary | Number of Vaso-occlusive Crises Requiring Hospitalization or Emergency Department Visits - Part B | | All patients randomised in Part B were to be included in the efficacy analysis set (EAS). Patients were analysed according to their randomised study medication. | Posted | | Mean | Standard Deviation | Number of events | | During 4 weeks of study treatment starting from randomization in Part B (week 2) up to 4 weeks (week 6). | | | | ID | Title | Description |
|---|
| OG000 | Part B - Ticagrelor | Randomised treatment group for Part B of the study. Relevant for the Part B period. | | OG001 | Part B - Placebo | Randomised treatment group for Part B of the study. Relevant for the Part B period. |
| |
| Secondary | Percentage of Days Hospitalized for Vaso-occlusice Crisis or Other Complications of Sickle Cell Disease - Part B | | All patients randomised in Part B were to be included in the efficacy analysis set (EAS). Patients were analysed according to their randomised study medication. | Posted | | Mean | Standard Deviation | Percentage of days | | During 4 weeks of study treatment starting from randomization in Part B (week 2) up to 4 weeks (week 6). | | | | ID | Title | Description |
|---|
| OG000 | Part B - Ticagrelor | Randomised treatment group for Part B of the study. Relevant for the Part B period. | | OG001 | Part B - Placebo | Randomised treatment group for Part B of the study. Relevant for the Part B period. |
| |
| Secondary | Percentage of Days With Pain (Age >=4) - Part B | Pain measured using the Faces Pain Scale, range 0-10 (0, 2, 4, 6, 8, 10), where 0 is no pain | All patients randomised in Part B were to be included in the efficacy analysis set (EAS). Patients were analysed according to their randomised study medication. | Posted | | Mean | Standard Deviation | Percentage of days | | During 4 weeks of study treatment starting from randomization in Part B (week 2) up to 4 weeks (week 6). | | | | ID | Title | Description |
|---|
| OG000 | Part B - Ticagrelor | Randomised treatment group for Part B of the study. Relevant for the Part B period. | | OG001 | Part B - Placebo | Randomised treatment group for Part B of the study. Relevant for the Part B period. |
| |
| Secondary | Mean Intensity of Pain (Age >=4) - Part B | Pain measured using the Faces Pain Scale, range 0-10 (0, 2, 4, 6, 8, 10), where 0 is no pain | All patients randomised in Part B were to be included in the efficacy analysis set (EAS). Patients were analysed according to their randomised study medication. | Posted | | Mean | Standard Deviation | Mean score on scale | | During 4 weeks of study treatment starting from randomization in Part B (week 2) up to 4 weeks (week 6). | | | | ID | Title | Description |
|---|
| OG000 | Part B - Ticagrelor | Randomised treatment group for Part B of the study. Relevant for the Part B period. | | OG001 | Part B - Placebo | Randomised treatment group for Part B of the study. Relevant for the Part B period. |
| |
| Secondary | Percentage of Days of Analgesic Use (Age >= 4) - Part B | | All patients randomised in Part B were to be included in the efficacy analysis set (EAS). Patients were analysed according to their randomised study medication. | Posted | | Mean | Standard Deviation | Percentage of days | | During 4 weeks of study treatment starting from randomization in Part B (week 2) up to 4 weeks (week 6). | | | | ID | Title | Description |
|---|
| OG000 | Part B - Ticagrelor | Randomised treatment group for Part B of the study. Relevant for the Part B period. | | OG001 | Part B - Placebo | Randomised treatment group for Part B of the study. Relevant for the Part B period. |
| |
| Secondary | Percentage of Days of Opioid Analgesic Use (Age >=4) - Part B | | All patients randomised in Part B were to be included in the efficacy analysis set (EAS). Patients were analysed according to their randomised study medication. | Posted | | Mean | Standard Deviation | Percentage of days | | During 4 weeks of study treatment starting from randomization in Part B (week 2) up to 4 weeks (week 6). | | | | ID | Title | Description |
|---|
| OG000 | Part B - Ticagrelor | Randomised treatment group for Part B of the study. Relevant for the Part B period. | | OG001 | Part B - Placebo | Randomised treatment group for Part B of the study. Relevant for the Part B period. |
| |
| Secondary | Percentage of Days of Absence From School or Work (Age >=6) - Part B | | All patients randomised in Part B were to be included in the efficacy analysis set (EAS). Patients were analysed according to their randomised study medication. | Posted | | Mean | Standard Deviation | Percentage of days | | During 4 weeks of study treatment starting from randomization in Part B (week 2) up to 4 weeks (week 6). | | | | ID | Title | Description |
|---|
| OG000 | Part B - Ticagrelor | Randomised treatment group for Part B of the study. Relevant for the Part B period. | | OG001 | Part B - Placebo | Randomised treatment group for Part B of the study. Relevant for the Part B period. |
| |
| Other Pre-specified | Haemorrhagic Events - Part A | | All patients who received at least one single dose of ticagrelor were included in the safety population (SAF) for Part A. Analysis on the SAF was based on the study medication actually received. | Posted | | Number | | Number of events | | From randomisation to Part A (week 0) through Visit 4 (week 2) | | | | ID | Title | Description |
|---|
| OG000 | Part A - Ticagrelor | Randomised treatment group for Part A of the study. Relevant for the Part A period. |
| | |
| Other Pre-specified | Haemorrhagic Events - Part B | | All patients who received at least one dose of randomised study drug, ticagrelor or placebo, will be included in the SAF for Part B. Analysis on the SAF was based on the study medication actually received. Erroneously treated patients will be accounted for in the actual treatment group. | Posted | | Number | | Number of events | | During 4 weeks of study treatment starting from randomization in Part B (week 2) up to 4 weeks (week 6). | | | | ID | Title | Description |
|---|
| OG000 | Part B - Ticagrelor | Randomised treatment group for Part B of the study. Relevant for the Part B period. | | OG001 | Part B - Placebo | Randomised treatment group for Part B of the study. Relevant for the Part B period. |
| |