Antiepileptic Efficacy Study of GWP42003-P in Children and Young Adults With Dravet Syndrome (GWPCARE1)
Condition: Epilepsy; Dravet Syndrome
MeSH terms: Epilepsies, Myoclonic; Syndrome
Intervention: GWP42003-P 20 mg/kg/day Dose; Placebo control
Intervention description: GWP42003-P was an oral solution containing 100 mg/milliliter (mL) cannabidiol (CBD) dissolved 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).; Placebo oral solution contained 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).
Intervention MeSH terms: Cannabidiol
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 120.0
Sex: All
Minimum age: 2 Years
Maximum age: 18 Years
Healthy volunteers: No
Keywords: Cannabidiol; CBD; Epidiolex; GWP42003-P

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.