A Phase 3 interventional study of GWP42003-P 20 mg/kg/day Dose and Placebo in Epilepsy and Lennox-Gastaut Syndrome, sponsored by Jazz Pharmaceuticals. Completed at 24 sites in 3 countries. Open to participants aged 2 Years to 55 Years. Per ClinicalTrials.gov, last updated 2022-09-28.
Sponsored by Jazz Pharmaceuticals · Phase 3, Interventional, and Treatment
To evaluate the efficacy of GWP42003-P as adjunctive treatment in reducing the number of drop seizures when compared with placebo, in participants with Lennox-Gastaut Syndrome (LGS).
This study was a 1:1 randomized, double-blind, 14-week comparison of 20 milligram [mg] per kilogram [kg] per day [mg/kg/day] of GWP42003-P versus placebo. The treatment period consisted of a 2-week titration period followed by a 12-week maintenance period. The study determined the efficacy, safety and tolerability of GWP42003-P compared with placebo. The dose was recommended by the Data Safety Monitoring Committee (DSMC) after assessment of safety and pharmacokinetic data from Part A of study GWEP1332. The first participants enrolled into this study after the DSMC reviewed the safety data from Part A of study GWEP1332. Following study completion, all participants were invited to continue to receive GWP42003-P in an open label extension (OLE) study (under a separate protocol).
1,842 studies on the registry are indexed under Epilepsy; 423 are open to participants now.
This study's enrollment of 171 is above the median of 50 across 1,228 interventional studies indexed under Epilepsy.
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Key Inclusion Criteria:
Key Exclusion Criteria:
Participants received GWP42003-P 20 mg/kg/day 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.
Drug: GWP42003-P 20 mg/kg/day Dose
Participants received placebo (0 mg/mL CBD), volume matched to the 20 mg/kg/day dose, 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) period.
Drug: Placebo
Drug page: Cannabidiol
GWP42003-P was presented as an oral solution containing 100 mg/milliliter (mL) cannabidiol (CBD) in the excipients sesame oil and anhydrous ethanol (79 mg/mL) with added sweetener (0.5 mg/mL sucralose) and strawberry flavoring (0.2 mg/mL).
Also known as: Cannabidiol, Epidiolex
Placebo was presented as an oral solution containing 0 mg/mL CBD in the excipients sesame oil and anhydrous ethanol (79 mg/mL) with added sweetener (0.5 mg/mL sucralose) and strawberry flavoring (0.2 mg/mL).
Percentage Change From Baseline In Drop Seizure Frequency During The Treatment Period
Drop seizures were recorded by the participant or caregiver using an interactive voice response system (IVRS) diary. Drop seizures were defined as the subset of tonic-clonic, tonic or atonic seizures that were reported as drop seizures in the IVRS. 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.
Time frame: Baseline to End of Treatment (EOT) (Day 99) or Early Termination (ET)
Number Of Participants With a ≥50% Reduction From Baseline in Drop Seizure Frequency During The Treatment Period
Drop seizures were recorded by the participant or caregiver using an IVRS diary. Drop seizures included the subset of tonic-clonic, tonic or atonic seizures that were reported as drop seizures in IVRS. Percentage change from baseline was calculated as per the primary outcome measure.
Time frame: Baseline to EOT (Day 99) or ET
Percentage Change From Baseline In Total Seizure Frequency During The Treatment Period
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 using an IVRS diary. Percentage change from baseline was calculated as per the primary outcome measure. Negative percentages show an improvement from baseline.
Time frame: Baseline to EOT (Day 99) or ET
Subject/Caregiver Global Impression Of Change Assessment (S/CGIC)
The S/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 S/CGIC questionnaire at subsequent visits. If both a CGIC and SGIC were completed then the CGIC was used; if only a CGIC was completed then the CGIC was used; if only a SGIC was completed then the SGIC was used. 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.
Time frame: Baseline to Last Visit (Day 99) or ET
| Milestone | GWP42003-P 20 mg/kg/Day Dose | Placebo |
|---|---|---|
| Started | 86 | 85 |
| Safety analysis set | 86 | 85 |
| Intent to treat (itt) analysis set | 86 | 85 |
| Completed | 72 | 84 |
| Not completed | 14 | 1 |
| Withdrew: Adverse event | 8 | 1 |
| Withdrew: Met withdrawal criteria | 4 | 0 |
| Withdrew: Use of g-tube | 1 | 0 |
| Withdrew: Did not meet eligibility criteria | 1 | 0 |
Drop seizures were recorded by the participant or caregiver using an interactive voice response system (IVRS) diary. Drop seizures were defined as the subset of tonic-clonic, tonic or atonic seizures that were reported as drop seizures in the IVRS. 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.
| percentage change | GWP42003-P 20 mg/kg/Day Dose | Placebo |
|---|---|---|
| Percentage Change From Baseline In Drop Seizure Frequency During The Treatment Period | -43.90 (-69.62 to 1.93) | -21.80 (-45.72 to 1.74) |
Drop seizures were recorded by the participant or caregiver using an IVRS diary. Drop seizures included the subset of tonic-clonic, tonic or atonic seizures that were reported as drop seizures in IVRS. Percentage change from baseline was calculated as per the primary outcome measure.
| Participants | GWP42003-P 20 mg/kg/Day Dose | Placebo |
|---|---|---|
| Number Of Participants With a ≥50% Reduction From Baseline in Drop Seizure Frequency During The Treatment Period | 38 | 20 |
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 using an IVRS diary. Percentage change from baseline was calculated as per the primary outcome measure. Negative percentages show an improvement from baseline.
| percentage change | GWP42003-P 20 mg/kg/Day Dose | Placebo |
|---|---|---|
| Percentage Change From Baseline In Total Seizure Frequency During The Treatment Period | -41.24 (-62.85 to -13.00) | -13.70 (-45.00 to 7.27) |
The S/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 S/CGIC questionnaire at subsequent visits. If both a CGIC and SGIC were completed then the CGIC was used; if only a CGIC was completed then the CGIC was used; if only a SGIC was completed then the SGIC was used. 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.
| Participants | GWP42003-P 20 mg/kg/Day Dose | Placebo |
|---|---|---|
| Very Much Improved | 15 | 5 |
| Much Improved | 14 | 9 |
| Slightly Improved | 20 | 15 |
| No Change | 27 | 43 |
| Slightly Worse | 7 | 9 |
| Much Worse | 1 | 2 |
| Very Much Worse | 0 | 2 |
Collected over Day 1 through Day 137 (Safety Follow-up). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| GWP42003-P 20 mg/kg/Day Dose | — | 20/86 (23.3%) | 53/86 (61.6%) |
| Placebo | — | 4/85 (4.7%) | 43/85 (50.6%) |
| Event | GWP42003-P 20 mg/kg/Day Dose | Placebo |
|---|---|---|
| Alanine aminotransferase increasedInvestigations | 4/86 | 0/85 |
| Aspartate aminotransferase increasedInvestigations | 4/86 | 0/85 |
| PneumoniaInfections and infestations | 3/86 | 0/85 |
| Gamma-glutamyltransferase increasedInvestigations | 3/86 | 0/85 |
| Acute respiratory failureRespiratory, thoracic and mediastinal disorders | 3/86 | 0/85 |
| DehydrationMetabolism and nutrition disorders | 0/86 | 2/85 |
| Viral infectionInfections and infestations | 2/86 | 0/85 |
| ConvulsionNervous system disorders | 2/86 | 0/85 |
| HypoxiaRespiratory, thoracic and mediastinal disorders | 2/86 | 0/85 |
| PyrexiaGeneral disorders | 0/86 | 1/85 |
| Event | GWP42003-P 20 mg/kg/Day Dose | Placebo |
|---|---|---|
| DiarrhoeaGastrointestinal disorders | 16/86 | 7/85 |
| VomitingGastrointestinal disorders | 8/86 | 14/85 |
| SomnolenceNervous system disorders | 12/86 | 8/85 |
| PyrexiaGeneral disorders | 11/86 | 7/85 |
| Decreased appetiteMetabolism and nutrition disorders | 11/86 | 2/85 |
| SedationNervous system disorders | 7/86 | 1/85 |
| Nasal congestionRespiratory, thoracic and mediastinal disorders | 7/86 | 2/85 |
| Upper respiratory tract infectionInfections and infestations | 2/86 | 6/85 |
| RashSkin and subcutaneous tissue disorders | 6/86 | 1/85 |
| ConvulsionNervous system disorders | 3/86 | 5/85 |
| Age, Continuous(years) | GWP42003-P 20 mg/kg/Day Dose | Placebo | Total |
|---|---|---|---|
| Mean | 15.478 ± 8.685 | 15.284 ± 9.7945 | 15.381 ± 9.2264 |
| Sex: Female, Male(Participants) | GWP42003-P 20 mg/kg/Day Dose | Placebo | Total |
|---|---|---|---|
| Female | 41 | 42 | 83 |
| Male | 45 | 43 | 88 |
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Studies of the drug tested here: Cannabidiol