An Open Label Extension Study of Cannabidiol (GWP42003-P) in Children and Adults With Dravet or Lennox-Gastaut Syndromes

NCT02224573 | Completed Phase 3 Results DMC

• 2 other identifiers: GWEP14152014-001834-27
• Study Type: interventional
• Target: 681
• Locations: 0 countries
• Sites: N/A

Brief Summary

To investigate the potential antiepileptic effects of cannabidiol (GWP42003-P) in children and adults with Dravet or Lennox-Gastaut syndromes.

Conditions

Epilepsy; Dravet Syndrome; Lennox-Gastaut Syndrome

Keywords

Cannabidiol; CBD; GWP42003-P; GWPCARE5

Outcome Measures

Primary Outcomes (28)

• 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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  Baseline; At last 12 weeks

Secondary Outcomes (32)

• 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

  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

  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

  Baseline; up to Week 260

• Number of Participants With Inpatient Hospitalizations Due to Epilepsy Since the Previous Visit

  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

  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

Study Arms (1)

GWP42003-P

EXPERIMENTAL

Drug: GWP42003-P

Interventions

GWP42003-P DRUG

Also known as: Cannabidiol, CBD, Epidiolex/Epidyolex®

GWP42003-P

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participant has completed the treatment phase of their Core Studies: GWEP1332A \[NCT02091206\], GWEP1332B \[NCT02091375\], GWEP1424 \[NCT02224703\], GWEP1414 \[NCT02224560\], GWEP1424 \[NCT02224703\], and GWEP1423 \[NCT02224690\].

You may not qualify if:

• Participant is currently using or has in the past used recreational or medicinal cannabis, or synthetic cannabinoid-based medications (including Sativex®) within the 3 months prior to study entry other than the investigational medicinal product (IMP) received during the Core Study and are unwilling to abstain for the duration for the study.
• Any history of suicidal behavior or any suicidal ideation of type 4 or 5 on the Columbia-Suicide Severity Rating Scale (C-SSRS) at Visit 1.
• Participant has been part of a clinical trial involving an IMP during the inter-study period.
• Female participant is of child bearing potential or male participant's partner is of child bearing potential, unless willing to ensure that they or their partner use highly effective contraception, for example, hormonal contraceptives, intrauterine devices/hormone-releasing systems, bilateral tubal occlusion, vasectomized partner or sexual abstinence, during the study and for 3 months thereafter (however, a male condom should not be used in conjunction with a female condom).
• Participant has significantly impaired hepatic function at the 'End of Treatment' visit of their Core Study or at Visit 1 if re-assessed: i) Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>5 × upper limit of normal (ULN); ii) ALT or AST \>3 × ULN and (total bilirubin \[TBL\] \>2 × ULN or international normalized ratio \[INR\] \>1.5); iii) ALT or AST \>3 × ULN with the presence of fatigue, nausea, vomiting, right upper quadrant pain or tenderness, fever, rash, and/or eosinophilia (\>5%). This criterion must be confirmed prior to entering the study.

Sponsors & Collaborators

• GW Research Ltd: lead

Related Publications (2)

• Scheffer IE, Halford JJ, Miller I, Nabbout R, Sanchez-Carpintero R, Shiloh-Malawsky Y, Wong M, Zolnowska M, Checketts D, Dunayevich E, Devinsky O. Add-on cannabidiol in patients with Dravet syndrome: Results of a long-term open-label extension trial. Epilepsia. 2021 Oct;62(10):2505-2517. doi: 10.1111/epi.17036. Epub 2021 Aug 18.

  PMID: 34406656 DERIVED

• Patel AD, Mazurkiewicz-Beldzinska M, Chin RF, Gil-Nagel A, Gunning B, Halford JJ, Mitchell W, Scott Perry M, Thiele EA, Weinstock A, Dunayevich E, Checketts D, Devinsky O. Long-term safety and efficacy of add-on cannabidiol in patients with Lennox-Gastaut syndrome: Results of a long-term open-label extension trial. Epilepsia. 2021 Sep;62(9):2228-2239. doi: 10.1111/epi.17000. Epub 2021 Jul 20.

  PMID: 34287833 DERIVED

MeSH Terms

Conditions

Epilepsy; Epilepsies, Myoclonic; Lennox Gastaut Syndrome

Interventions

Cannabidiol

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epilepsy, Generalized; Epileptic Syndromes; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals

Results Point of Contact

• Title: Medical Enquiries
• Organization: Greenwich Biosciences

medinfo@greenwichbiosciences.com

1-833-424-6724

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 21, 2014
• First Posted: August 25, 2014
• Study Start: June 11, 2015
• Primary Completion: September 24, 2020
• Study Completion: September 24, 2020
• Last Updated: February 3, 2022
• Results First Posted: February 3, 2022

Record last verified: 2022-01