| Primary | Number of Vaso-Occlusive Crisis Events | A VOC is the composite of a painful crisis and/or an acute chest syndrome (ACS) event. The number of VOC events is defined as the count of VOC events experienced by a participant throughout the treatment period. | The FAS included all randomized participants regardless of treatment received. | Posted | | Number | | VOC events | | From randomization (Day 0) up to end of study (EOS) visit or date of premature study discontinuation, up to approximately 20 months | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
| | OG001 | Placebo | Pediatric participants received placebo matching with ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The number of placebo tablets was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of placebo matching with ticagrelor 15 mg bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of placebo matching with ticagrelor 30 mg bd.
- Participants with a body weight >48 kg received 3 tablets of placebo matching with ticagrelor 45 mg bd.
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| | | Title | Denominators | Categories |
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| | Group IDs | Group Description | Statistical Method | Statistical Comment | P-Value | P-Value Comment | Parameter Type | Parameter Value | Dispersion Type | Dispersion Value | Confidence Interval Sides | Confidence Interval % | CI Lower Limit | CI Upper Limit | CI Lower Limit Comment | CI Upper Limit Comment | Estimate Comment | Tested Non-Inferiority | Non-Inferiority Type | Non-Inferiority Comment | Other Analysis Description |
|---|
| Incidence rates, incidence rate ratios, and p-values are from a negative binomial model analysis, with treatment group and baseline hydroxyurea use included in the model as covariates. | Negative binomial model | | 0.7597 | | Incidence rate ratio | 1.06 | | | 2-Sided | 95 | 0.75 | 1.50 | | | | | Superiority | | |
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| Secondary | Number of Painful Crisis Events | A painful crisis is an onset or worsening of pain that lasts at least 2 hours, for which there is no explanation other than vaso-occlusion and which requires therapy with oral or parenteral opioids, parenteral non-steroidal anti-inflammatory drugs, or other analgesics prescribed by a healthcare provider in a medical setting (such as a hospital, clinic or emergency room visit) or at home. Events with an onset date <=7 days of the previous event onset date are not counted as new events. | The FAS included all randomized participants regardless of treatment received. | Posted | | Number | | painful crisis events | | From randomization (Day 0) up to EOS visit or date of premature study discontinuation, up to approximately 20 months | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
| | OG001 | Placebo | Pediatric participants received placebo matching with ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The number of placebo tablets was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of placebo matching with ticagrelor 15 mg bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of placebo matching with ticagrelor 30 mg bd.
- Participants with a body weight >48 kg received 3 tablets of placebo matching with ticagrelor 45 mg bd.
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| Secondary | Number of Acute Chest Syndrome Events | The ACS is an acute illness characterized by fever and/or respiratory symptoms, accompanied by a new pulmonary infiltrate on a chest X-ray. Events with an onset date <=7 days of the previous event onset date are not counted as new events. | The FAS included all randomized participants regardless of treatment received. | Posted | | Number | | ACS events | | From randomization (Day 0) up to EOS visit or date of premature study discontinuation, up to approximately 20 months | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
| | OG001 | Placebo | Pediatric participants received placebo matching with ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The number of placebo tablets was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of placebo matching with ticagrelor 15 mg bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of placebo matching with ticagrelor 30 mg bd.
- Participants with a body weight >48 kg received 3 tablets of placebo matching with ticagrelor 45 mg bd.
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| Secondary | Duration of Painful Crises | The duration of painful crises is defined as the sum of the duration of painful crises experienced by a participant over the defined treatment period. If two or more events have overlapping durations, the overlapping days were counted only once. | The FAS included all randomized participants regardless of treatment received. | Posted | | Number | | days | | From randomization (Day 0) up to EOS visit or date of premature study discontinuation, up to approximately 20 months | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
| | OG001 | Placebo | Pediatric participants received placebo matching with ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The number of placebo tablets was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of placebo matching with ticagrelor 15 mg bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of placebo matching with ticagrelor 30 mg bd.
- Participants with a body weight >48 kg received 3 tablets of placebo matching with ticagrelor 45 mg bd.
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| Secondary | Number of Vaso-Occlusive Crisis Events Requiring Hospitalization or Emergency Department Visits | The number of VOC events requiring hospitalization or emergency department visits is defined as the count of VOC events experienced by a participant over the treatment period, for which the primary setting for VOC treatment was in-patient hospitalization or emergency department. Events with an onset date <=7 days of the previous event onset date are not counted as new events. | The FAS included all randomized participants regardless of treatment received. | Posted | | Number | | VOC events requiring hospitalization | | From randomization (Day 0) up to EOS visit or date of premature study discontinuation, up to approximately 20 months | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
| | OG001 | Placebo | Pediatric participants received placebo matching with ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The number of placebo tablets was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of placebo matching with ticagrelor 15 mg bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of placebo matching with ticagrelor 30 mg bd.
- Participants with a body weight >48 kg received 3 tablets of placebo matching with ticagrelor 45 mg bd.
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| Secondary | Number of Days Hospitalized for Vaso-Occlusive Crisis Events | The number of days hospitalized for all individual VOC events experienced by a participant during the treatment period is defined as the sum of the duration of all individual hospitalizations (taking into account potential overlapping hospitalization days of VOC components) during VOC events experienced by a participant over the treatment period for which the primary setting for VOC treatment was in-patient hospitalization. | The FAS included all randomized participants regardless of treatment received. | Posted | | Number | | days | | From randomization (Day 0) up to EOS visit or date of premature study discontinuation, up to approximately 20 months | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
| | OG001 | Placebo | Pediatric participants received placebo matching with ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The number of placebo tablets was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of placebo matching with ticagrelor 15 mg bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of placebo matching with ticagrelor 30 mg bd.
- Participants with a body weight >48 kg received 3 tablets of placebo matching with ticagrelor 45 mg bd.
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| Secondary | Number of Acute Sickle Cell Disease Complications | The number of acute SCD complications is defined as the count of all individual acute SCD complications experienced by a participant over the treatment period. Acute SCD complications are defined as any one or more of the following individual complications: Transient ischaemic attack/ischaemic stroke, hepatic sequestration, splenic sequestration, priapism, and dactylitis. | The FAS included all randomized participants regardless of treatment received. | Posted | | Number | | acute SCD complications | | From randomization (Day 0) up to EOS visit or date of premature study discontinuation, up to approximately 20 months | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
| | OG001 | Placebo | Pediatric participants received placebo matching with ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The number of placebo tablets was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of placebo matching with ticagrelor 15 mg bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of placebo matching with ticagrelor 30 mg bd.
- Participants with a body weight >48 kg received 3 tablets of placebo matching with ticagrelor 45 mg bd.
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| Secondary | Number of Days Hospitalized for Acute Sickle Cell Disease Complications | The number of days hospitalized for acute SCD complications is defined as the sum of the duration of all individual hospitalizations (taking into account potential overlapping hospitalization days) due to acute SCD complications experienced by a participant over the treatment period, for which hospitalization was reported. | The FAS included all randomized participants regardless of treatment received. | Posted | | Number | | days | | From randomization (Day 0) up to EOS visit or date of premature study discontinuation, up to approximately 20 months | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
| | OG001 | Placebo | Pediatric participants received placebo matching with ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The number of placebo tablets was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of placebo matching with ticagrelor 15 mg bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of placebo matching with ticagrelor 30 mg bd.
- Participants with a body weight >48 kg received 3 tablets of placebo matching with ticagrelor 45 mg bd.
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| Secondary | Number of Sickle Cell-Related Red Blood Cell (RBC) Transfusions | The number of participants with at least one sickle cell-related RBC transfusion reported. Adverse events resulting in the need for RBC transfusions were captured prior to database lock to determine if the transfusion was sickle cell-related or not. | The FAS included all randomized participants regardless of treatment received. | Posted | | Number | | RBC transfusions | | From randomization (Day 0) up to EOS visit or date of premature study discontinuation, up to approximately 20 months | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
| | OG001 | Placebo | Pediatric participants received placebo matching with ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The number of placebo tablets was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of placebo matching with ticagrelor 15 mg bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of placebo matching with ticagrelor 30 mg bd.
- Participants with a body weight >48 kg received 3 tablets of placebo matching with ticagrelor 45 mg bd.
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| Secondary | Health-Related Quality of Life Total Score Using the Pediatric Quality of Life Inventory (PedsQL) Sickle Cell Disease Module | The PedsQL SCD module instrument developed using a 5-point Likert scale (where 0= never and 4= almost always) for the participant self-report forms for ages ≥5 to <8 years, ≥8 to <13 years, and ≥13 to ≤18 years and the caregiver proxy-report form specific for ≥2 to <5 years was used. The PedsQL SCD module measures problems in the following categories:
- Pain: 3 sub-scales
- Worry: 2 sub-scales
- Emotions: 1 sub-scale
- Treatment: 1 sub-scale
- Communication: 2 sub-scales
- Total score PedsQL SCD module items were reverse-scored and linearly transformed to a 0 to 100 scale (0= 100, 1= 75, 2= 50, 3= 25, 4= 0) so that higher scores indicate better quality of life. To create the PedsQL SCD module total score (43/42/40 items - depending on version completed) the arithmetic mean of the transformed scores was computed as the sum of the items transformed scores divided by the number of items answered. Baseline values are the closest observation prior to and including the randomization visit.
| The FAS included all randomized participants regardless of treatment received. Only number of participants included in analysis at each time point are reported. | Posted | | Mean | Standard Deviation | score on a scale | | For ages ≥2 to <5 years and ≥5 to <8 years: Baseline (observation prior to and including the randomization visit) and Months 6, and 12; For ages ≥8 to <13 years and ≥13 to ≤18 years: Baseline and Months 6, 12, and 18 | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
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| Secondary | Fatigue Total Score Using Pediatric Quality of Life Inventory Multidimensional Fatigue Scale | The PedsQL multidimensional fatigue scale instrument developed using a 5-point Likert scale (where 0= never and 4= almost always) for the participant self-report forms for ages ≥5 to <8 years, ≥8 to <13 years, and ≥13 to ≤18 years and the caregiver proxy-report form specific for ≥2 to <5 years was used. The PedsQL multidimensional fatigue scale measures problems in the following categories:
- General (6 items)
- Sleep/rest (6 items)
- Cognitive fatigue (6 items)
- Total score (18 items) PedsQL multidimensional fatigue scale items were reverse-scored and linearly transformed to a 0 to 100 scale (0= 100, 1= 75, 2= 50, 3= 25, 4= 0) so that higher scores indicate better quality of life. To create the PedsQL multidimensional fatigue scale total score (18 items), the arithmetic mean of the transformed scores was computed as the sum of the items transformed scores divided by the number of items answered. Baseline values are closest observation prior to and including randomization visit.
| The FAS included all randomized participants regardless of treatment received. Only number of participants included in analysis at each time point are reported. | Posted | | Mean | Standard Deviation | score on a scale | | For ages ≥2 to <5 years and ≥5 to <8 years: Baseline (observation prior to and including the randomization visit) and Months 6, and 12; For ages ≥8 to <13 years and ≥13 to ≤18 years: Baseline and Months 6, 12, and 18 | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
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| Secondary | Percentage of Days of Absence From School or Work Due to Sickle Cell Disease | For participants attending school/work at randomization, absence from school/work due to SCD was recorded weekly by the participant in the eDevice with the help of the caregiver if needed. The percentage of days absent from school/work due to SCD in the defined treatment period was calculated as follows: Percentage of absent days = (total number of days reported)/(total number of questionnaires answered ×7). | The FAS included all randomized participants regardless of treatment received. Only participants going to school or work at randomization are reported. | Posted | | Mean | Standard Deviation | percentage of days | | From randomization (Day 0) up to EOS visit or date of premature study discontinuation, up to approximately 20 months | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
| | OG001 | Placebo | Pediatric participants received placebo matching with ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The number of placebo tablets was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of placebo matching with ticagrelor 15 mg bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of placebo matching with ticagrelor 30 mg bd.
- Participants with a body weight >48 kg received 3 tablets of placebo matching with ticagrelor 45 mg bd.
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| Secondary | Average Intensity of Worst Pain Daily During Vaso-Occlusive Crisis Events in Participants <5 Years of Age | The Face, Legs, Activity, Cry, Consolability (FLACC) scale is caregiver-reported and used to assess pain daily during the VOC event for those participants <5 years of age as determined at randomization. Each of the 5 behaviours observed are assigned a score of 0, 1 or 2. The total FLACC score ranges between 0 and 10, with 0 representing "no pain" and 10 representing "very much pain". Lower score indicate better outcome. Worst pain ratings were collected once daily throughout the duration of the VOC event using an eDevice. | The FAS included all randomized participants regardless of treatment received. Only participants <5 years of age who had experienced at least one individual VOC event and analyzed for pain assessment are reported. | Posted | | Mean | Standard Deviation | score on a scale | | From randomization (Day 0) up to EOS visit or date of premature study discontinuation, up to approximately 20 months | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
| | OG001 |
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| Secondary | Average Intensity of Worst Pain Daily During Vaso-Occlusive Crisis Events in Participants ≥5 Years of Age | The Faces Pain Scale-revised (FPS-R) was administered to assess pain daily during the VOC event by those participants aged ≥5 years as determined at randomization. The FPS-R consists of 6 faces and scoring ranges between 0 and 10 (with an increase in numeric value by 2), where 0 is "no pain" and 10 is "very much pain". Lower score indicate better outcome. Worst pain ratings were collected once daily throughout the duration of the VOC event using an eDevice. | The FAS included all randomized participants regardless of treatment received. Only participants ≥5 years of age who had experienced at least one individual VOC event and analyzed for pain assessment are reported. | Posted | | Mean | Standard Deviation | score on a scale | | From randomization (Day 0) up to EOS visit or date of premature study discontinuation, up to approximately 20 months | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
| | OG001 | Placebo |
|
| Secondary | Type of Analgesics Used by Participants During Vaso-Occlusive Crisis Events | Analgesics use (opioid and non-opioid) during VOC events. | The FAS included all randomized participants regardless of treatment received. Only participants who had at least one VOC and took an analgesic are reported. | Posted | | Number | | participants | | From randomization (Day 0) up to EOS visit or date of premature study discontinuation, up to approximately 20 months | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
| | OG001 | Placebo | Pediatric participants received placebo matching with ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The number of placebo tablets was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of placebo matching with ticagrelor 15 mg bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of placebo matching with ticagrelor 30 mg bd.
- Participants with a body weight >48 kg received 3 tablets of placebo matching with ticagrelor 45 mg bd.
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| Secondary | Palatability of the Study Treatment Assessed by Study Medication Palatability Assessment (SMPA) in Participants ≤4 Years of Age | Response to palatability was assessed through the SMPA question "Was any behaviour observed when the study medication was given to this participant that would be indicative of a negative response to the palatability of the study medication?". This was presented as a binary outcome (that is, where "No" is no negative response and "Yes" is negative response). No negative response was considered as a positive outcome. | The Safety Analysis Set included all participants who received at least 1 single dose of randomized study treatment, ticagrelor or placebo, and for whom any post-dose data were available. Only participants ≤4 years of age who completed the assessment for study treatment palatability are reported. | Posted | | Count of Participants | | Participants | | Baseline (randomization visit) and Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
| | OG001 | Placebo |
|
| Secondary | Swallowability of the Study Treatment Assessed by Study Medication Palatability Assessment in Participants ≤4 Years of Age | An observer's assessment of the participant's behaviour using the SMPA was performed for all participants taking the study treatment who are 2 to 4 years of age. Willingness to swallow was assessed and categorized as follows:
- Swallowed without a problem
- Some resistance but did swallow
- Spit out some/all of the medication
- Vomited up the medication. The category "swallowed without a problem" was considered as positive outcome.
| The Safety Analysis Set included all participants who received at least 1 single dose of randomized study treatment, ticagrelor or placebo, and for whom any post-dose data were available. Only participants ≤4 years of age who completed the assessment for study treatment swallowability are reported. | Posted | | Count of Participants | | Participants | | Baseline (randomization visit) and Month 6 | | | | ID | Title | Description |
|---|
| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
| | OG001 | Placebo |
|
| Secondary | Palatability of the Study Treatment Assessed by Facial Hedonic Scale (FHS) in Participants ≥5 Years of Age | The FHS method was used for all participants taking the study treatment who are ≥5 years of age. The FHS consists of 5 faces with descriptions ranging from "Dislike very much" to "Like very much". The face with description "Like very much" was considered as positive outcome. The way in which the study treatment was taken, that is, whether the tablet is whole or dispersed, was captured. | The Safety Analysis Set included all participants who received at least 1 single dose of randomized study treatment, ticagrelor or placebo, and for whom any post-dose data were available. Only participants ≥5 years of age who completed the assessment for study treatment palatability are reported. | Posted | | Count of Participants | | Participants | | Baseline (randomization visit) and Month 6 | | | | ID | Title | Description |
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| OG000 | Ticagrelor 15/30/45 mg bd | Pediatric participants received ticagrelor 15 mg, or 30 mg, or 45 mg tablets orally bd for at least 12 months but no longer than 24 months. The dose of ticagrelor was determined based on body weight at randomization:
- Participants with a body weight ≥12 to ≤24 kg received 1 tablet of ticagrelor 15 mg (1x15 mg) bd.
- Participants with a body weight >24 to ≤48 kg received 2 tablets of ticagrelor 15 mg (2x15 mg) bd.
- Participants with a body weight >48 kg received 3 tablets of ticagrelor 15 mg (3x15 mg) bd.
| | OG001 | Placebo | |
|