| Primary | Percentage Change From Baseline In Convulsive Seizure Frequency During The Treatment Period | Convulsive seizures (atonic, clonic, tonic, or tonic-clonic) were recorded by the participant or caregiver using an interactive voice response system (IVRS) diary. Percentage change from baseline was calculated as: ([frequency during the treatment period - frequency during baseline]/frequency during baseline) * 100. The frequency during each period was based on 28-day averages and calculated as: (number of seizures in the period/number of reported days in the IVRS period) * 28. Baseline included all available data prior to Day 1 (28-day average). Negative percentages show an improvement from baseline. | ITT analysis set: included all participants in the Part B safety analysis set who had post-baseline efficacy data. Participants were analyzed according to the treatment group to which they were randomized. The ITT analysis set was the primary analysis set for all efficacy outcome measures. | Posted | | Median | Inter-Quartile Range | percent change | | Baseline to End of Treatment (EOT) (Day 99) or Early Termination (ET) | | | | ID | Title | Description |
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| OG000 | GWP42003-P 20 mg/kg/Day Dose | Participants received 20 mg/kg/day of GWP42003-P administered orally, half in the morning and half in the evening. Participants titrated GWP42003-P to 20 mg/kg/day over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day) period. | | OG001 | Placebo | Participants received placebo (0 mg/mL CBD), volume-matched to the 20 mg/kg/day dose level, administered orally, half in the morning and half in the evening. To maintain the blinded aspect of the study, participants titrated the placebo dose over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day of the matched dose) period. |
| | | Title | Denominators | Categories |
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| | | Title | Measurements |
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| - OG000-38.94(-69.53 to -4.83)
- OG001-13.29(-52.53 to 20.20)
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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 |
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| | Wilcoxon rank-sum test | | 0.0123 | | Median Difference (Final Values) | -22.79 | | | 2-Sided | 95 | -41.06 | -5.43 | | | Calculated using the Hodges-Lehmann approach. | | Superiority | | |
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| Secondary | Number Of Participants With A ≥50% Reduction From Baseline In Convulsive Seizure Frequency During The Treatment Period | Convulsive seizures (atonic, clonic, tonic, or tonic-clonic) were recorded by the participant or caregiver using an IVRS diary. Percentage change from baseline was calculated as per the primary outcome measure. | ITT analysis set: included all participants in the Part B safety analysis set who had post-baseline efficacy data. Participants were analyzed according to the treatment group to which they were randomized. The ITT analysis set was the primary analysis set for all efficacy outcome measures. | Posted | | Count of Participants | | Participants | | Baseline to EOT (Day 99) or ET | | | | ID | Title | Description |
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| OG000 | GWP42003-P 20 mg/kg/Day Dose | Participants received 20 mg/kg/day of GWP42003-P administered orally, half in the morning and half in the evening. Participants titrated GWP42003-P to 20 mg/kg/day over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day) period. | | OG001 | Placebo | Participants received placebo (0 mg/mL CBD), volume-matched to the 20 mg/kg/day dose level, administered orally, half in the morning and half in the evening. To maintain the blinded aspect of the study, participants titrated the placebo dose over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day of the matched dose) period. |
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| Secondary | Number of Participants With A ≥25%, ≥75% Or 100% Reduction From Baseline In Convulsive Seizure Frequency During The Treatment Period | Convulsive seizures (atonic, clonic, tonic, or tonic-clonic) were recorded by the participant or caregiver using an IVRS diary. Percentage change from baseline was calculated as per the primary outcome measure. | ITT analysis set: included all participants in the Part B safety analysis set who had post-baseline efficacy data. Participants were analyzed according to the treatment group to which they were randomized. The ITT analysis set was the primary analysis set for all efficacy outcome measures. | Posted | | Count of Participants | | Participants | | Baseline to EOT (Day 99) or ET | | | | ID | Title | Description |
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| OG000 | GWP42003-P 20 mg/kg/Day Dose | Participants received 20 mg/kg/day of GWP42003-P administered orally, half in the morning and half in the evening. Participants titrated GWP42003-P to 20 mg/kg/day over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day) period. | | OG001 | Placebo | Participants received placebo (0 mg/mL CBD), volume-matched to the 20 mg/kg/day dose level, administered orally, half in the morning and half in the evening. To maintain the blinded aspect of the study, participants titrated the placebo dose over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day of the matched dose) period. |
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| Secondary | Percentage Change From Baseline In Non-Convulsive Seizure Frequency During The Treatment Period | Non-convulsive seizures (myoclonic, partial, or absence) were recorded by the participant or caregiver using an IVRS diary. Percentage change from baseline was calculated as per the primary outcome measure. Only participants with non-convulsive seizures during the baseline period were included. Negative percentages show an improvement from baseline. | ITT analysis set: included all participants in the Part B safety analysis set who had post-baseline efficacy data. Participants were analyzed according to the treatment group to which they were randomized. The ITT analysis set was the primary analysis set for all efficacy outcome measures. | Posted | | Median | Inter-Quartile Range | percent change | | Baseline to EOT (Day 99) or ET | | | | ID | Title | Description |
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| OG000 | GWP42003-P 20 mg/kg/Day Dose | Participants received 20 mg/kg/day of GWP42003-P administered orally, half in the morning and half in the evening. Participants titrated GWP42003-P to 20 mg/kg/day over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day) period. | | OG001 | Placebo | Participants received placebo (0 mg/mL CBD), volume-matched to the 20 mg/kg/day dose level, administered orally, half in the morning and half in the evening. To maintain the blinded aspect of the study, participants titrated the placebo dose over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day of the matched dose) period. |
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| Secondary | Caregiver Global Impression Of Change In Seizure Duration (CGICSD) | Seizure duration was assessed qualitatively using the CGICSD. Caregivers were asked "Since the patient started treatment, please assess the average duration of the patient's seizures (comparing their condition now to their condition before treatment)"; responses included decrease, no change, or increase in average duration. For each seizure type, only participants with at least 1 seizure for the corresponding seizure type, reported at any time during the study, were included. | ITT analysis set: included all participants in the Part B safety analysis set who had post-baseline efficacy data. Participants were analyzed according to the treatment group to which they were randomized. The ITT analysis set was the primary analysis set for all efficacy outcome measures. | Posted | | Count of Participants | | Participants | | Baseline to EOT (Day 99) or ET | | | | ID | Title | Description |
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| OG000 | GWP42003-P 20 mg/kg/Day Dose | Participants received 20 mg/kg/day of GWP42003-P administered orally, half in the morning and half in the evening. Participants titrated GWP42003-P to 20 mg/kg/day over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day) period. | | OG001 | Placebo | Participants received placebo (0 mg/mL CBD), volume-matched to the 20 mg/kg/day dose level, administered orally, half in the morning and half in the evening. To maintain the blinded aspect of the study, participants titrated the placebo dose over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day of the matched dose) period. |
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| Secondary | Number Of Participants Using Rescue Medication | The use of rescue medication was recorded by the participant or caregiver using a paper diary. | Safety Analysis Set: included all participants randomized to treatment who received at least 1 dose of IMP. Participants were analyzed according to the actual treatment they received. | Posted | | Count of Participants | | Participants | | Baseline to EOT (Day 99) or ET | | | | ID | Title | Description |
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| OG000 | GWP42003-P 20 mg/kg/Day Dose | Participants received 20 mg/kg/day of GWP42003-P administered orally, half in the morning and half in the evening. Participants titrated GWP42003-P to 20 mg/kg/day over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day) period. | | OG001 | Placebo | Participants received placebo (0 mg/mL CBD), volume-matched to the 20 mg/kg/day dose level, administered orally, half in the morning and half in the evening. To maintain the blinded aspect of the study, participants titrated the placebo dose over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day of the matched dose) period. |
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| Secondary | Number Of Participants With Inpatient Hospitalizations Due To Epilepsy | Inpatient hospitalizations due to epilepsy were recorded by the participant or caregiver and through the serious adverse events (SAE) reporting process. | ITT analysis set: included all participants in the Part B safety analysis set who had post-baseline efficacy data. Participants were analyzed according to the treatment group to which they were randomized. The ITT analysis set was the primary analysis set for all efficacy outcome measures. | Posted | | Number | | participants | | Baseline to Safety Follow-up (Day 137) | | | | ID | Title | Description |
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| OG000 | GWP42003-P 20 mg/kg/Day Dose | Participants received 20 mg/kg/day of GWP42003-P administered orally, half in the morning and half in the evening. Participants titrated GWP42003-P to 20 mg/kg/day over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day) period. | | OG001 | Placebo | Participants received placebo (0 mg/mL CBD), volume-matched to the 20 mg/kg/day dose level, administered orally, half in the morning and half in the evening. To maintain the blinded aspect of the study, participants titrated the placebo dose over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day of the matched dose) period. |
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| Secondary | Change From Baseline In Sleep Disruption 0 To 10 Numerical Rating Scale (0 to 10 NRS) Score | The sleep disruption 0 to 10 NRS questionnaire was completed by the participant's caregiver. The caregiver was asked 'On a scale of '0 to 10', please indicate the number that best describes your child's sleep disruption in the last week.' The markers ranged from 0 = 'slept extremely well' to 10 = 'unable to sleep at all'. The change from baseline in the sleep disruption 0 to 10 numerical rating scale score was analyzed using an analysis of covariance (ANCOVA) model with baseline and age group (2 to 5 years, 6 to 12 years and 13 to 18 years) as covariates and treatment group as a fixed factor. A negative change from baseline represents an improvement in sleep. Last visit for endpoints assessed at clinic visits was defined as the last scheduled visit (not including the end of taper or safety follow-up visits) at which participant's last evaluation was performed. | ITT analysis set: included all participants in the Part B safety analysis set who had post-baseline efficacy data. Participants were analyzed according to the treatment group to which they were randomized. The ITT analysis set was the primary analysis set for all efficacy outcome measures. | Posted | | Least Squares Mean | 95% Confidence Interval | units on a scale | | Baseline to Last Visit (Day 99) or ET | | | | ID | Title | Description |
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| OG000 | GWP42003-P 20 mg/kg/Day Dose | Participants received 20 mg/kg/day of GWP42003-P administered orally, half in the morning and half in the evening. Participants titrated GWP42003-P to 20 mg/kg/day over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day) period. |
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| Secondary | Change From Baseline In Epworth Sleepiness Scale (ESS) Score | The ESS questionnaire was completed by the participant's caregiver. The change from baseline in the ESS score was analyzed using an ANCOVA model with baseline and age group (2 to 5 years, 6- to 2 years and 13 to 18 years) as covariates and treatment group as a fixed factor. The total score was the sum of the 8 item-scores and ranged from 0 to 24. A higher total score represents greater levels of daytime sleepiness. | ITT analysis set: included all participants in the Part B safety analysis set who had post-baseline efficacy data. Participants were analyzed according to the treatment group to which they were randomized. The ITT analysis set was the primary analysis set for all efficacy outcome measures. | Posted | | Least Squares Mean | 95% Confidence Interval | units on a scale | | Baseline to Last Visit (Day 99) or ET | | | | ID | Title | Description |
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| OG000 | GWP42003-P 20 mg/kg/Day Dose | Participants received 20 mg/kg/day of GWP42003-P administered orally, half in the morning and half in the evening. Participants titrated GWP42003-P to 20 mg/kg/day over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day) period. | | OG001 | Placebo | Participants received placebo (0 mg/mL CBD), volume-matched to the 20 mg/kg/day dose level, administered orally, half in the morning and half in the evening. To maintain the blinded aspect of the study, participants titrated the placebo dose over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day of the matched dose) period. |
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| Secondary | Change From Baseline In Quality Of Life In Childhood Epilepsy (QOLCE) Score | The QOLCE questionnaire was completed by the parent or caregiver of participants aged 4 years and above. The change from baseline in the overall quality of life score was analyzed using an ANCOVA model with baseline and age group (2 to 5 years, 6 to 12 years and 13 to 18 years) as covariates and treatment group as a fixed factor. Zero represents the lowest or poorest category and 100 represents the highest level of functioning. The overall quality of life score was calculated by taking the mean of the subscale scores. | ITT analysis set: included all participants in the Part B safety analysis set who had post-baseline efficacy data. Participants were analyzed according to the treatment group to which they were randomized. The ITT analysis set was the primary analysis set for all efficacy outcome measures. | Posted | | Least Squares Mean | 95% Confidence Interval | units on a scale | | Baseline to EOT (Day 99) or ET | | | | ID | Title | Description |
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| OG000 | GWP42003-P 20 mg/kg/Day Dose | Participants received 20 mg/kg/day of GWP42003-P administered orally, half in the morning and half in the evening. Participants titrated GWP42003-P to 20 mg/kg/day over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day) period. | | OG001 | Placebo | Participants received placebo (0 mg/mL CBD), volume-matched to the 20 mg/kg/day dose level, administered orally, half in the morning and half in the evening. To maintain the blinded aspect of the study, participants titrated the placebo dose over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day of the matched dose) period. |
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| Secondary | Change From Baseline In Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) Score | The Vineland-II scores (standard scores and adaptive levels for each adaptive behavior domain, the adaptive behavior composite, and the maladaptive behavior index score and level) were assessed by the participant's caregiver. Scores were analyzed using an ANCOVA model with baseline and age group (2 to 5 years, 6 to 12 years, and 13 to 18 years) as covariates and treatment group as a fixed factor. Higher scores represent greater levels of functioning except for the maladaptive behavior index, for which a negative change from baseline represents an improvement in condition. | ITT analysis set: included all participants in the Part B safety analysis set who had post-baseline efficacy data. Participants were analyzed according to the treatment group to which they were randomized. The ITT analysis set was the primary analysis set for all efficacy outcome measures. | Posted | | Least Squares Mean | 95% Confidence Interval | units on a scale | | Baseline to Last Visit (Day 99) or ET | | | | ID | Title | Description |
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| OG000 | GWP42003-P 20 mg/kg/Day Dose | Participants received 20 mg/kg/day of GWP42003-P administered orally, half in the morning and half in the evening. Participants titrated GWP42003-P to 20 mg/kg/day over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day) period. | | OG001 | Placebo |
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| Secondary | Caregiver Global Impression Of Change (CGIC) | The CGIC was used to assess the participant's overall condition on a 7-point scale using the markers "very much improved, much improved, slightly improved, no change, slightly worse, much worse, or very much worse" (1 = very much improved; 7 = very much worse). On Day 1 (prior to starting IMP), the caregiver was asked to write a brief description of the participant's overall condition as a memory aid for the CGIC questionnaire at subsequent visits. | ITT analysis set: included all participants in the Part B safety analysis set who had post-baseline efficacy data. Participants were analyzed according to the treatment group to which they were randomized. The ITT analysis set was the primary analysis set for all efficacy outcome measures. | Posted | | Count of Participants | | Participants | | Baseline to Last Visit (Day 99) or ET | | | | ID | Title | Description |
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| OG000 | GWP42003-P 20 mg/kg/Day Dose | Participants received 20 mg/kg/day of GWP42003-P administered orally, half in the morning and half in the evening. Participants titrated GWP42003-P to 20 mg/kg/day over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day) period. | | OG001 | Placebo | Participants received placebo (0 mg/mL CBD), volume-matched to the 20 mg/kg/day dose level, administered orally, half in the morning and half in the evening. To maintain the blinded aspect of the study, participants titrated the placebo dose over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day of the matched dose) period. |
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