Efficacy and Safety of GWP42003-P for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults
Condition: Epilepsy; Lennox Gastaut Syndrome
MeSH terms: Seizures; Lennox Gastaut Syndrome; Syndrome
Study type: Interventional
Phase: Phase 3

Summary: 
      The primary objective of this study was 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).
    

Detailed description: 
      This study was a 1:1:1 randomized, double-blind, 14-week comparison of 2 dose levels (10
      milligram [mg] per kilogram [kg] per day [mg/kg/day] and 20 mg/kg/day) of GWP42003-P versus
      placebo. Participants who satisfied all inclusion and none of the exclusion criteria began a
      28-day baseline observation period. The treatment period consisted of a 2-week titration
      period followed by a 12-week maintenance period. The 20 mg/kg/day dose was recommended by the
      Data Safety Monitoring Committee (DSMC) after assessment of safety and pharmacokinetic data
      from Part A of study GWEP1332 (NCT02091206). The 10 mg/kg/day dose was defined as 50% of the
      20 mg/kg/day dose. The first participant was not enrolled into this study until the DSMC had
      reviewed the safety data from Part A of study GWEP1332.

      Participants who satisfied all eligibility criteria were randomized at Day 1 to receive 10
      mg/kg/day GWP42003-P, 20 mg/kg/day GWP42003-P, or placebo at a 1:1:1 ratio. Participants in
      the placebo group were split into 2 equivalent cohorts; one half received 10 mg/kg/day dosing
      volumes and one half received 20 mg/kg/day dosing volumes. The 2 placebo cohorts were pooled
      for the analyses of efficacy. Participants titrated GWP42003-P to the 10 mg/kg/day or 20
      mg/kg/day (or equivalent volume of placebo) dose over 7 and 11 days, respectively, and
      remained at this dose for the 12-week maintenance period. Following the end of treatment (Day
      99), participants were invited to continue to receive GWP42003-P in an open-label extension
      (OLE) study under a separate protocol (GWEP1415; NCT02224573). All participants who did not
      immediately enter the OLE study tapered investigational medicinal product (IMP) (10% per day
      over 10 days). However, the taper period could be interrupted if the participant wished to
      enter the OLE study within a 7-day timeframe. Participants who down-titrated IMP returned for
      an end of taper period visit (Day 109). Participants who did not enter the OLE study or who
      withdrew prematurely had a safety follow-up visit 28 days later (Day 137).
    

Intervention name(s): GWP42003-P; Placebo control
Intervention type(s): Drug; Drug
Intervention description(s): 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). Participants were randomly assigned to receive either 10 or 20 mg/kg/day.; 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).
Other intervention name(s): Cannabidiol; Epidiolex; Placebo
Intervention MeSH terms: Cannabidiol
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Cannabidiol; CBD; Epidiolex; GWP42003-P