A Dose-ranging Pharmacokinetics and Safety Study of GWP42003-P in Children With Dravet Syndrome (GWPCARE1)
Condition: Epilepsy; Dravet Syndrome
MeSH terms: Epilepsies, Myoclonic; Syndrome
Intervention: GWP42003-P 5 mg/kg/day Dose; Placebo control; GWP42003-P 10 mg/kg/day Dose; GWP42003-P 20 mg/kg/day Dose
Intervention description: GWP42003-P was an oral solution containing 25 mg/milliliter (mL) cannabidiol (CBD) or 100 mg/mL 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). Participants were randomly assigned to receive either 5, 10 or 20 mg/kg/day.; 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).; GWP42003-P was an oral solution containing 25 mg/mL CBD or 100 mg/mL 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). Participants were randomly assigned to receive either 5, 10 or 20 mg/kg/day.; GWP42003-P was an oral solution containing 25 mg/mL CBD or 100 mg/mL 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). Participants were randomly assigned to receive either 5, 10 or 20 mg/kg/day.
Intervention MeSH terms: Cannabidiol
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 34.0
Sex: All
Minimum age: 4 Years
Maximum age: 10 Years
Healthy volunteers: No
Keywords: Cannabidiol; CBD; Epidiolex; GWP42003-P

Summary:

      To evaluate the safety and pharmacokinetics (PK) of multiple doses of GWP42003-P compared
      with placebo in children with Dravet syndrome.
    

Eligibility criteria:

        Key Inclusion Criteria:

          -  Participants were male or female aged between 4 and 10 years (inclusive).

          -  Participants had a documented history of Dravet Syndrome that was not completely
             controlled by AEDs.

          -  Participants took one or more AEDs at a dose which had been stable for at least 4
             weeks.

          -  Participants had experienced fewer than 4 convulsive seizures (tonic-clonic, tonic,
             clonic, atonic seizures) during the 28-day baseline period.

          -  All medications or interventions for epilepsy (including ketogenic diet and vagus
             nerve stimulation [VNS]) were stable for four weeks prior to screening and
             participants were willing to maintain a stable regimen throughout the study. The
             ketogenic diet and VNS treatments were not counted as an AED.

        Key Exclusion Criteria:

          -  Participants had clinically significant unstable medical conditions other than
             epilepsy.

          -  Participants had clinically relevant abnormalities in the 12-lead electrocardiogram
             measured at screening or randomization.

          -  Participants were currently using or had in the past used recreational or medicinal
             cannabis, or synthetic CBD based medications (including Sativex®) within the 3 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 IMP.

          -  Participants who had been part of a clinical trial involving another investigational
             product in the previous 6 months.

          -  There were plans for the participants to travel outside their country of residence
             during the study.

          -  Participants were previously randomized into this study. In particular, participants
             participating in Part A of the study cannot enter Part B.