To investigate the potential antiepileptic effects of cannabidiol (GWP42003-P) in children and adults with Dravet or Lennox-Gastaut syndromes.
This is a multi-center, open-label extension study for participants with Dravet syndrome or Lennox-Gastaut syndrome who have previously participated in double-blind, placebo-controlled clinical studies of GWP42003-P (Core Studies: GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], GWEP1424 \[NCT02224703\], and GWEP1423 \[NCT02224690\]). The first participant was enrolled into the open-label extension study after the Data Safety Monitoring Committee reviewed the safety data from Part A of study GWEP1332.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
681
Number of Participants With Any Treatment-emergent Adverse Event (TEAE) Occurring in ≥5% of Participants in Any Treatment Group
TEAEs, defined as AEs that started, or worsened in severity or seriousness, following the first dose of investigational medicinal product in this study, are reported. Any AEs that continued from the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) were only classified as treatment emergent if they worsened in this study.
Time frame: up to Week 260
Number of Participants With Clinically Significant Changes in the Indicated Vital Sign Values From the Pre-randomization Baseline of the Core Study at Any Time Post-dose
Clinical significance was determined by the investigator. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. bpm = beats per minute; DBP = Diastolic Blood Pressure; mmHg = millimeters of mercury; SBP = Systolic Blood Pressure. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Body Mass Index (BMI)
BMI is an estimate of body fat based on body weight divided by height squared. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Time frame: up to Week 260
Number of Participants With Clinically Significant Electrocardiogram (ECG) Values at the Pre-randomization Baseline of the Core Study and at Any Time Post-dose
Clinical significance was determined by the investigator. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. msec = milliseconds; QTcB = corrected QT interval with Bazett's correction. The time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to Week 260
Number of Participants With the Indicated Responses to Questions Regarding Suicidal Ideation and Behavior Using the Children's Columbia-Suicide Severity Rating Scale (C-SSRS) at Day 1 and at Any Time Post-dose
The C-SSRS questionnaire is a brief, standardized measure that uniquely assesses the essential information (behavior, ideation, lethality, and severity) and distinguishes between suicidal occurrences and non-suicidal self-injury.
Time frame: up to Week 260
Mean Cannabis Withdrawal Scale Score at End of Taper (EOT), the Post-Taper Safety Call, and at Safety Follow-up
The Cannabis Withdrawal Scale is a 19-item scale administered to participants (18 years and above), with each item (withdrawal symptom) measured on a 0 to 10 numerical rating scale (0 = Not at all; 5 = Moderately; 10 = Extremely). Total score, calculated as the sum of the 19 item sub-scores ranging from 0-190 are reported. Higher scores indicate more withdrawal symptoms and an increased negative impact to quality of life. The participant or participant's caregiver was asked to record the extent to which each withdrawal symptom was experienced in the last 24 hours and also to rate the negative impact on normal daily activities. L24S = Last 24 Hours Score; NIS = Negative Impact on Normal Daily Activity Score. The End of Treatment visit occurred after a maximum of 260 weeks of treatment. The End of Taper occurred up to 10 days after the End of Treatment visit. The Safety Call and Safety Follow-up occurred 2 and 4 weeks, respectively, after the End of Taper.
Time frame: up to Week 260
Mean Pediatric Cannabinoid Withdrawal Scale (PCWS) Score at the End of Treatment, the End of Taper, the Post-Taper Safety Call, and at Safety Follow-up
The PCWS was administered to participants aged 4 to 17 (inclusive) that was developed from the 19-item validated CWS (adults) to assess mood, behavioral, and physical symptoms associated with cannabis. The total score, calculated as the sum of 10 items (rated on a 4-point scale: 0 = none; 1 = a little bit; 2 = quite a bit; 3 = a lot) ranging from 0-30 are reported. Higher scores indicate more withdrawal symptoms and an increased negative impact to quality of life. The End of Treatment visit occurred after a maximum of 260 weeks of treatment. The participant or participant's caregiver was asked to record the extent to which each withdrawal symptom was experienced in the last 24 hours and also to rate the negative impact on normal daily activities. The End of Taper occurred up to 10 days after the End of Treatment visit. The Safety Call and Safety Follow-up occurred 2 and 4 weeks, respectively, after the End of Taper.
Time frame: up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Red Blood Cells (RBCs) Values
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Hemoglobin Levels
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Hematocrit Values
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Erythrocyte Mean Corpuscular Volume
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Erythrocyte Mean Corpuscular Hemoglobin
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes, and Neutrophils
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in the Percentage of Basophils, Eosinophils, Lymphocytes, Monocytes, and Neutrophils in White Blood Cells (WBCs)
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Sodium, Potassium, Blood Urea Nitrogen, Glucose, Calcium, and High-Density Lipoprotein (HDL) Cholesterol
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Creatinine (Jaffe), Creatinine (Enzymatic), and Bilirubin
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Creatinine Clearance (Schwartz) and Creatinine Clearance (Cockcroft-Gault)
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. mL/min/1.73 m\^2 = millilitres per minute per 1.73 meters squared. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Alkaline Phosphatase, Aspartate Aminotransferase, Alanine Aminotransferase, and Gamma Glutamyl Transferase
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Albumin and Protein
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Prolactin
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Prothrombin Time
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Prothrombin International Normalized Ratio
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Insulin-like Growth Factor-1 (IGF-1) Levels
IGF-1 levels in serum were analyzed as part of the clinical laboratory testing in participants. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: up to Week 260
Mean Subject/Caregiver Global Impression of Change (S/CGIC) Score
The S/CGIC allows participants and caregivers to rate the participant's overall condition on a scale of 1 to 7 (1 = Very Much Improved, and 7 = Very Much Worse). The combined caregiver and participant summary used either the caregiver or participant version if only one version was completed, or the caregiver version when both the caregiver and participant versions were completed. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Percent Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Total Seizure Frequency
Total seizures included the sum of all seizures (tonic-clonic, tonic, atonic, clonic, myoclonic, countable partial, other partial, and absence seizures) recorded by the participant or caregiver. Change from Baseline was calculated as the percentage change from Baseline for each 12-week period. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study. "Last 12 weeks" is equal to the last 12 weeks' data for each participant irrespective of time in study.
Time frame: Baseline; Week 49 to 60, Week 97 to 108, Week 145 to 156, Week 205 to 216, Week 253 to 264, and Last 12 Weeks
Percent Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Drop Seizure Frequency in Participants With Lennox-Gastaut Syndrome
Drop seizures are defined as the subset of tonic-clonic, tonic, or atonic seizures. Change from Baseline was calculated as the percentage change from Baseline for each 12-week period. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study. The outcome was reported for Lennox-Gastaut Syndrome participants only. "Last 12 weeks" is equal to the last 12 weeks' data for each participant irrespective of time in study.
Time frame: Baseline; Week 49 to 60, Week 97 to 108, Week 145 to 156, Week 205 to 216, and Last 12 Weeks
Percent Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Convulsive Seizure Frequency in Participants With Dravet Syndrome
Convulsive seizures are defined as tonic-clonic, tonic, clonic, or atonic seizures. Change from Baseline was calculated as the percentage change from Baseline for each 12-week period. Pre-randomization Baseline of the Core Study (GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], and GWEP1423 \[NCT02224690\]) is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. Data for change from Baseline at pre-randomization of the Core Study are presented; however, the time frame represents the duration for which participants were enrolled in GWEP1415. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study. The outcome was reported for Dravet Syndrome participants only. "Last 12 weeks" is equal to the last 12 weeks' data for each participant irrespective of time in study.
Time frame: Baseline; Week 49 to 60, Week 97 to 108, Week 145 to 156, Week 205 to 216, Week 253 to 264, and Last 12 Weeks
Number of Participants With Convulsive and Non-convulsive Seizures Greater Than 30 Minutes in Duration (Status Epilepticus)
Status epilepticus is defined as any seizure lasting for 30 minutes or longer. The number of convulsive seizures greater than 30 minutes in duration and the number of non-convulsive seizures greater than 30 minutes in duration were collected weekly. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study. mins = minutes. "Last 12 weeks" is equal to the last 12 weeks' data for each participant irrespective of time in study.
Time frame: Baseline; At last 12 weeks
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Quality of Life in Childhood Epilepsy (QOLCE) Scores
The QOLCE is a parent-reported questionnaire that evaluates health-related QOL in children aged 2-18 years old. It contains 76 items with 16 subscales covering 7 domains (physical activities, social activities, cognition, emotional well-being, behavior, general health, and general QOL). All items in the questionnaire are rated on a 5-point or 6-point categorical scale. Based on the responses to the items in each domain, scores for 16 subscales are derived. Score range from 0 to 100. Higher score indicate highest level of functioning. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study is defined as the Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment QOL in Epilepsy Scores (QOLIE-31-P) in Participants With Lennox-Gastaut Syndrome
The QOLIE-31-P is a survey of health-related QOL for adults with epilepsy. It is comprised of 38 questions about health daily activities and evaluates how much distress the participant feels about problems and worries related to epilepsy. The questionnaire consists of 7 subscales (energy, mood, daily activities, cognition, medication effects, seizure worry, and overall QOL). Scores range from 0 to 100. Higher scores indicate better QOL. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study. The outcome was reported for Lennox-Gastaut Syndrome participants only.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Vineland Adaptive Behavior Scale, Second Edition (Vineland-II) Scores
The Vineland-II is an individually administered instrument for assessing adaptive behaviors. It consists of 4 adaptive behavior domains (1 = low; 5 = high) and a maladaptive behavior domain (1 = clinically significant; 3 = average), and an overall Adaptive Behavior Composite Score. Standard scores (population mean = 100, SD = 15, range = 20 to 160) are produced for the domain and composite scores, and scaled scores (called v-scale scores, with a population mean = 15, SD = 3, range = 1 to 24) are produced for the subscale scores. Higher scores indicate better functioning. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Pre-randomization Baseline of the Core Study is defined as Baseline of the double-blind, placebo-controlled, clinical studies with GWP42003-P. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Number of Participants With Inpatient Hospitalizations Due to Epilepsy Since the Previous Visit
Inpatient hospitalizations due to epilepsy were recorded by the investigator. The timing of the "End of Treatment" varied per participant based on when the participant discontinued IMP.
Time frame: Week 48, 104, 156, 208, and End of Treatment (up to Week 260 based on when the participant discontinued treatment)
Number of Treatment Responders With Greater Than or Equal to 50% Reduction in Total Seizures
Total seizures included the sum of all seizures (tonic-clonic, tonic, atonic, clonic, myoclonic, countable partial, other partial, and absence seizures) recorded by the participant or caregiver.
Time frame: Baseline; Week 49 to 60, Week 97 to 108, Week 145 to 156, Week 205 to 216, Week 253 to 264, and Last 12 Weeks
Number of Participants With Changes in the Average Duration of Seizure Subtypes as Assessed by the Participant/Caregiver Global Impression of Change in Seizure Duration (S/CGICSD)
The SGICSD and CGICSD are comprised of the following questions rated on a 3-point scale for each seizure type. The CGICSD score is used to assess the average duration of the participant's seizures (comparing their condition now to their condition before treatment) and the SGICSD score is used to assess the average duration of seizures (comparing condition now to condition before treatment). Scores range from 1 to 3 (1 = Decrease in average duration; 3 = Increase in average duration).
Time frame: Up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Delis-Kaplan Executive Function System (D-KEFS) Visual Scanning Completion Time Scaled Score
D-KEFS Visual Scanning Completion Time Scale was used to measure visual attention and processing speed. The cognitive assessment battery items are age-specific, used to measure a variety of functions for both children and adults. The items were administered by an experienced psychometrician to a sub-group of sites that had the expertise to conduct the test. The raw score i.e., total time in seconds for completing the visual scanning trial was measured in the range of 0-150; higher scores indicated worse functioning. The raw score was normed with a referenced score to convert into a scaled score (i.e., score on a scale) ranging from 1-19; higher scores indicated better functioning. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as score on a scale. Negative values indicate worsening and positive values indicate improvement in cognitive function.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in D-KEFS Number Sequencing Completion Time Scaled Score
D-KEFS Number Sequencing Completion Time Scale was used to measure processing speed. The raw score i.e., total time in seconds for completing the number sequencing trial was measured in the range of 0-150; higher scores indicated worse functioning. The raw score was normed with a referenced score to convert into a scaled score (i.e., score on a scale) ranging from 1-19; higher scores indicated better processing speed. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as score on a scale. Negative values indicate worsening and positive values indicate improvement in processing speed. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in D-KEFS Letter Sequencing Completion Time Scaled Score
D-KEFS Letter Sequencing Completion Time Scaled was used to measure processing speed. The raw score i.e., total time in seconds for completing the letter sequencing trial was measured in the range of 0-150; higher scores indicated worse functioning. The raw score was normed with a referenced score to calculate a scaled score (i.e., score on a scale) ranging from 1-19; higher scores indicated better processing speed. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as score on a scale. Negative values indicate worsening and positive values indicate improvement in processing speed. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in D-KEFS Number-letter Switching Completion Time Scaled Score
D-KEFS Number-letter Switching Completion Time Scale was used to measure cognitive flexibility, task-set switching, and general executive functioning. The raw score i.e., total time in seconds for completing the number-letter switching trial was measured in the range of 0-240; higher scores indicated worse functioning. The raw score was normed with a referenced score to calculate a scaled score (i.e., score on a scale) ranging from 1-19; higher scores indicated better functioning. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as score on a scale. Negative values indicate worsening and positive values indicate improvement in cognitive function. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in D-KEFS Motor Speed Completion Time Scaled Score
D-KEFS Motor Speed Completion Time Scale was used to measure motor speed. The raw score i.e., total time in seconds for completing the motor speed trial was measured in the range of 0-150; higher scores indicated worse functioning. The raw score was normed with a referenced score to calculate a scaled score (i.e., score on a scale) ranging from 1-19; higher scores indicated better motor speed. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as score on a scale. Negative values indicate worsening and positive values indicate improvement in motor speed. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Purdue Pegboard Fine Motor Speed (Both Hands Z Score)
Purdue Pegboard test was used to measure fine motor dexterity of both hands. The raw score was calculated as the total number of pegs in 30 seconds; higher scores indicated higher level of motor dexterity. The raw score was converted to a Z-score ranging from 0-19; higher scores indicate a higher level of motor dexterity. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as Z-score (i.e., score on a scale). Negative values indicate worsening and positive values indicate improvement in motor dexterity. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Purdue Pegboard Fine Motor Speed (Dominant Hand Z Score)
Purdue Pegboard test was used to measure fine motor dexterity of the dominant hand. The raw score was calculated as the total number of pegs in 30 seconds; higher scores indicated higher level of motor dexterity. The raw score was converted to a Z-score ranging from 0-19; higher scores indicate a higher level of motor dexterity. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as Z-score (i.e., score on a scale). Negative values indicate worsening and positive values indicate improvement in motor dexterity. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Purdue Pegboard Fine Motor Speed (Non Dominant Hand Z Score)
Purdue Pegboard test was used to measure fine motor dexterity of the non dominant hand. The raw score was calculated as the total number of pegs in 30 seconds; higher scores indicated higher level of motor dexterity. The raw score was converted to a Z-score ranging from 0-19; higher scores indicate a higher level of motor dexterity. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as Z-score (i.e., score on a scale). Negative values indicate worsening and positive values indicate improvement in motor dexterity. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Wechsler Adult Intelligence Scale (WAIS) Coding Scaled Score
WAIS Coding Scale was used to measure processing speed. The raw score i.e., sum of correct substitutions was measured in the range of 0-16. The raw score was normed with a referenced score to calculate a scaled score (i.e., score on a scale) ranging from 1-19; higher scores indicated better processing speed. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as score on a scale. Negative values indicate worsening and positive values indicate improvement in processing speed. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WAIS Digit Span Backward Scaled Score
WAIS Digit Span Backward Scale was used to measure working memory. The raw score i.e., sum of correct trials was measured in the range of 0-16. The raw score was normed with a referenced score to calculate a scaled score (i.e., score on a scale) ranging from 1-19; higher scores indicated better working memory. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as score on a scale. Negative values indicate worsening and positive values indicate improvement in working memory. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WAIS Digit Span Forward Scaled Score
WAIS Digit Span Forward Scale was used to measure auditory memory. The raw score i.e., sum of correct trials was measured in the range of 0-16. The raw score was normed with a referenced score to calculate a scaled score (i.e., score on a scale) ranging from 1-19; higher scores indicated better auditory memory. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as score on a scale. Negative values indicate worsening and positive values indicate improvement in auditory memory. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WAIS Longest Digit Span Backward Scaled Score
WAIS Longest Digit Span Backward Scale was used to measure working memory. The raw score i.e., sum of correct trials was measured in the range of 2-8. The raw score was normed with a referenced score to calculate a scaled score (i.e., score on a scale) ranging from 1-19; higher scores indicated better working memory. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as score on a scale. Negative values indicate worsening and positive values indicate improvement in working memory. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WAIS Longest Digit Span Forward Scaled Score
WAIS Longest Digit Span Forward Scale was used to measure auditory memory. The raw score i.e., sum of correct trials was measured in the range of 2-8. The raw score was normed with a referenced score to calculate a scaled score (i.e., score on a scale) ranging from 1-19; higher scores indicated better auditory memory. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as score on a scale. Negative values indicate worsening and positive values indicate improvement in auditory memory. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Wechsler Abbreviated Scale of Intelligence (WASI)-II Vocabulary T Score
WAIS-II Vocabulary T Score was used to estimate general intellectual ability based on fund of information and verbal intelligence. The raw score i.e., the sum of correct responses was converted to a T-score ranging from 20-80 using the WASI assessment manual; T-scores are standard scores with a mean of 50 and a standard deviation of 10. Higher scores indicate a higher level of intelligence. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as T-score (i.e., score on a scale). Negative values indicate worsening and positive values indicate improvement in intelligence. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WASI-II Matrix Reasoning T Score
WASI-II Matrix Reasoning T Score was used to estimate general intellectual ability based abstract reasoning. The raw score i.e., the sum of correct responses was converted to a T-score ranging from 20-80 using the WASI assessment manual; T-scores are standard scores with a mean of 50 and a standard deviation of 10. Higher scores indicate a higher level of abstract reasoning. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as T-score (i.e., score on a scale). Negative values indicate worsening and positive values indicate improvement in abstract reasoning. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Wechsler Intelligence Scale for Children (WISC) Coding Scaled Score
WISC Coding Scale was used to measure processing speed in children aged 6-16 years 11 months, with scores ranging from 0 to 119. Higher scores indicate better processing speed. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as score on a scale. Negative values indicate worsening and positive values indicate improvement in processing speed. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WISC Digit Span Backward Scaled Score
WISC Digit Span Backward Scale was used to measure working memory. The raw score i.e., sum of correct trials was measured on a scale of 0-18; higher scores indicated better working memory. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as score on a scale. Negative values indicate worsening and positive values indicate improvement in working memory. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WISC Digit Span Forward Scaled Score
WISC Digit Span Forward Scale was used to measure auditory memory. The raw score i.e., sum of correct trials was measured on a scale of 0-18; higher scores indicated better auditory memory. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as score on a scale. Negative values indicate worsening and positive values indicate improvement in auditory memory. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WISC Longest Digit Span Backward Scaled Score
WISC Longest Digit Span Backward Scale was used to measure working memory. The raw score i.e., sum of correct trials was measured on a scale of 2-8; higher scores indicated better working memory. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as score on a scale. Negative values indicate worsening and positive values indicate improvement in working memory. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WISC Longest Digit Span Forward Scaled Score
WISC Longest Digit Span Forward Scale was used to measure auditory memory. The raw score i.e., sum of correct trials was measured on a scale of 2-10; higher scores indicated better auditory memory. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as score on a scale. Negative values indicate worsening and positive values indicate improvement in auditory memory. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Wechsler Preschool & Primary Scale of Intelligence (WPPSI)-IV Bug Search T Score
WPPSI-IV Bug Search T Score was used to measure processing speed and complex attention. The raw score i.e., the sum of correct responses was measured on a scale of 1-19 and converted to a T-score ranging from 20-80; T-scores are standard scores with a mean of 50 and a standard deviation of 10. Higher scores indicate a higher level of attention. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as T-score (i.e., score on a scale). Negative values indicate worsening and positive values indicate improvement in attention. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WPPSI-IV Receptive Vocabulary T Score
WPPSI-IV Receptive Vocabulary T Score was used to measure fund of information. The raw score i.e., the sum of correct responses was measured on a scale of 1-19 and converted to a T-score ranging from 20-80; T-scores are standard scores with a mean of 50 and a standard deviation of 10. Higher scores indicate a higher level of intelligence. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as as T-score (i.e., score on a scale). Negative values indicate worsening and positive values indicate improvement in intelligence. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in WPPSI-IV Matrix Reasoning T Score
WPPSI-IV Matrix Reasoning T Score was used to measure abstract reasoning. The raw score i.e., the sum of correct responses was measured on a scale of 1-19 and converted to a T-score ranging from 20-80; T-scores are standard scores with a mean of 50 and a standard deviation of 10. Higher scores indicate a higher level of abstract reasoning. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as T-score (i.e., score on a scale). Negative values indicate worsening and positive values indicate improvement in abstract reasoning. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Expressive One-Word Picture Vocabulary Test (EOWPVT) Scaled Score
Expressive One-Word Picture Vocabulary Test-4th edition was used to test vocabulary. The raw score i.e., the sum of correct responses was measured on a scale of 1-19 and converted to a T-score ranging from 0-83; T-scores are standard scores with a mean of 100 and a standard deviation of 15. Higher scores indicate a higher level of language performance. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as as T-score (i.e., score on a scale). Negative values indicate worsening and positive values indicate improvement in language performance. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in a Developmental NEuroPSYchological Assessment (NEPSY)-2 Word Generation Scaled Score
Developmental NEuroPSYchological Assessment (NEPSY)-2 Word Generation Scale was used to measure verbal fluency. The raw score i.e., the sum of correct responses was converted to a scaled score (i.e., score on a scale) ranging from 1-19; higher scores indicate a higher level of verbal fluency. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as score on a scale. Negative values indicate worsening and positive values indicate improvement in verbal fluency. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260
Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Visual Perception Standard Scaled Score
Visual Perception Standard scale was used to measure visual motor functioning. The raw score i.e., the sum of the correct responses was measured on a 6-standard score range. Score range of 70-79 indicated "low," 80-89 indicated "below average," 90-109 indicated "average," 110-119 indicated "above average," 120-129 indicated "high," and \>129 indicated "very high" visual motor functioning. Change from the pre-randomization Baseline of the core study to the End of Treatment are reported as score on a scale. Negative values indicate worsening and positive values indicate improvement in visual motor functioning. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data at pre-randomization of the Core Study.
Time frame: Baseline; up to Week 260