Antiepileptic Efficacy Study of GWP42003-P in Children and Young Adults With Dravet Syndrome (GWPCARE1)
Condition: Epilepsy; Dravet Syndrome
MeSH terms: Epilepsies, Myoclonic; Syndrome
Study type: Interventional
Phase: Phase 3

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

Detailed description: 
      GWEP1332 Part B recruited an entirely new group of participants than GWEP1332 Part A.
      Participants who failed the entry criteria for Part A were eligible to take part in Part B.

      Part B was a 1:1 randomized, double-blind, placebo-controlled, 14-week comparison of
      GWP42003-P versus placebo. The aim of Part B was to assess the antiepileptic efficacy of
      GWP42003-P as an adjunctive antiepileptic treatment compared with placebo, with respect to
      the percentage change from baseline during the treatment period of the study in convulsive
      seizure frequency in children and young adults.

      Following the establishment of initial eligibility and baseline measurements, participants
      entered Part B and began a 28-day baseline observation period.

      Eligible participants were then randomized to receive either GWP42003-P or placebo on a 1:1
      basis and titrated up to the target dose that was identified in Part A (up to 20 milligrams
      [mg] per kilogram [kg] per day), which was confirmed following completion of Part A by an
      independent Data Safety Monitoring Committee who reviewed unblinded safety and
      pharmacokinetic data from Part A.

      Participants received investigational medicinal product for 14 weeks, consisting of a
      titration period followed by a 12-week maintenance period.

      Efficacy and safety were monitored at various clinic visits and via telephone. After 14 weeks
      of treatment, all participants were offered the option of entering an open label extension
      (OLE) study. Entry was within seven days of the final treatment visit. Participants who did
      not immediately enter the OLE study commenced a down-titration taper period lasting up to 10
      days. The taper period was interrupted if the participant wished to enter the open label
      extension study within the seven-day timeframe.

      For participants who opted not to enter the OLE study, a follow-up telephone call was made 28
      days after the end of dosing and weekly safety telephone calls were made during the 28-day
      follow-up period.
    

Intervention name(s): GWP42003-P 20 mg/kg/day Dose; Placebo control
Intervention type(s): Drug; Drug
Intervention description(s): 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).
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