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Phase 3 N=120 Randomized Quadruple-blind Treatment

Antiepileptic Efficacy Study of GWP42003-P in Children and Young Adults With Dravet Syndrome (GWPCARE1)

Epilepsy · Dravet Syndrome

Enrolled (actual)
120
Serious AEs
10.8%
Results posted
Jul 2018
Primary outcome: Primary: Percentage Change From Baseline In Convulsive Seizure Frequency During The Treatment Period — -38.94; -13.29 percent change — p=0.0123

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
GWP42003-P 20 mg/kg/day Dose (Drug); Placebo control (Drug)
Age
Pediatric, Adult · 2+ yrs
Sex
All
Sponsor
Jazz Pharmaceuticals
Primary completion
Nov 2015

Outcome Measures

OutcomeResultp-value
PRIMARY
Percentage Change From Baseline In Convulsive Seizure Frequency During The Treatment Period
-38.94; -13.29 0.0123 sig
SECONDARY
Number Of Participants With A ≥50% Reduction From Baseline In Convulsive Seizure Frequency During The Treatment Period
26; 16 0.0784
SECONDARY
Number of Participants With A ≥25%, ≥75% Or 100% Reduction From Baseline In Convulsive Seizure Frequency During The Treatment Period
38; 26; 14; 7; 3; 0
SECONDARY
Percentage Change From Baseline In Non-Convulsive Seizure Frequency During The Treatment Period
-40.16; -34.69
SECONDARY
Caregiver Global Impression Of Change In Seizure Duration (CGICSD)
17; 8; 32; 31; 0; 2
SECONDARY
Number Of Participants Using Rescue Medication
36; 41
SECONDARY
Number Of Participants With Inpatient Hospitalizations Due To Epilepsy
5; 1; 2; 1
SECONDARY
Change From Baseline In Sleep Disruption 0 To 10 Numerical Rating Scale (0 to 10 NRS) Score
-0.7; -0.3
SECONDARY
Change From Baseline In Epworth Sleepiness Scale (ESS) Score
0.82; -0.69
SECONDARY
Change From Baseline In Quality Of Life In Childhood Epilepsy (QOLCE) Score
5.6; 4.1
SECONDARY
Change From Baseline In Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) Score
-0.8; 3.0; -0.8; -0.8; -0.6; -0.6
SECONDARY
Caregiver Global Impression Of Change (CGIC)
9; 4; 10; 4; 18; 12

Summary

To investigate the potential antiepileptic effects of cannabidiol (GWP42003-P) in children and young adults with Dravet syndrome.

Eligibility Criteria

Key Inclusion Criteria

  • Participants were male or female aged between 2 and 18 years (inclusive).
  • Participants had a documented history of Dravet Syndrome that was not completely controlled by current antiepileptic drugs.
  • Participants took one or more antiepileptic drugs at a dose that had been stable for at least four weeks.
  • All medications or interventions for epilepsy (including ketogenic diet and vagus nerve stimulation) were stable for four weeks prior to screening and participants were willing to maintain a stable regimen throughout the study.

Key Exclusion Criteria

  • Participants had clinically significant unstable medical conditions other than epilepsy.
  • Participants had clinically relevant symptoms or a clinically significant illness in the four weeks prior to screening or randomization, other than epilepsy.
  • Participants were currently using or had in the past used recreational or medicinal cannabis or synthetic cannabinoid based medications (including Sativex®) within the three months prior to study entry and were unwilling to abstain for the duration for the study.
  • Participants had any known or suspected hypersensitivity to cannabinoids or any of the excipients of the investigational medicinal products.
  • Participants had been part of a previous clinical trial involving another investigational product in the previous six months.
  • There were plans for the participants to travel outside their country of residence during the study.
  • Participants previously randomized into this study. In particular, participants who participated in Part A of the study could not enter Part B.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02091375). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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