Clinical Trial: GWPCARE2 A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet Syndrome

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Randomized, Double-blind, Placebo-controlled Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet Syndrome.

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

Detailed Summary:

This study is a 1:1:1 randomized, double-blind, 14-week comparison of two Dose Levels of GWP42003-P versus placebo. A 28-day screening period prior to randomization (to establish baseline parameters) will precede the treatment period, which will consist of a two-week titration period followed by a 12-week maintenance period. The study will aim to determine the efficacy, safety and tolerability of GWP42003-P compared with placebo. The High Dose Level will be as recommended by the Data Safety Monitoring Committee (DSMC) after assessment of safety and pharmacokinetic data from Part A of study GWEP1332. The Low Dose Level will be defined as 50% of the High Dose Level. The first subject will not enroll into this study until the DSMC has reviewed the safety data from Part A of study GWEP1332.

Following study completion, all subjects will be invited to continue to receive GWP42003-P in an open label extension (OLE) study (under a separate protocol).


Sponsor: GW Research Ltd

Current Primary Outcome: Percentage change from baseline in convulsive seizure frequency during the treatment period. [ Time Frame: 0-14 weeks ]

The primary endpoint is the percentage change from baseline in convulsive seizure frequency (average per 28 days) during the treatment period (Day 1 to the end of the evaluable period) in subjects taking GWP42003-P compared with placebo. Non-parametric analyses will be used for the primary endpoint should the assumptions for parametric analyses (e.g. Normality) not be valid.


Original Primary Outcome: Mean percentage change from baseline in convulsive seizure frequency during the maintenance period. [ Time Frame: 2-14 weeks ]

The primary endpoint is the mean percentage change from baseline in convulsive seizure frequency during the maintenance period (Day 15 to the end of the evaluable period) in subjects taking GWP42003-P compared with placebo.


Current Secondary Outcome:

  • Number of subjects considered treatment responders, defined as those with a ≥25%, ≥50% or ≥75% reduction in convulsive seizures from baseline. [ Time Frame: 0-14 weeks ]
  • Number of subjects who are convulsive seizure free. [ Time Frame: 0-14 weeks ]
  • Percentage change from baseline in non-convulsive seizure frequency. [ Time Frame: 0-14 weeks ]
  • Change in types of seizures from baseline. [ Time Frame: 0-14 weeks ]
  • Caregiver Global Impression of Change (CGIC) at the end of treatment. [ Time Frame: End of week 14 of treatment ]
  • The incidence of adverse events as measure of subject safety. [ Time Frame: Day -28 to Day 137 ]
  • The number of age-appropriate subjects with a treatment-emergent flag using the Columbia-Suicide Severity Rating Scale (C-SSRS) (Children's) during the course of the study. [ Time Frame: Day -28 to Day 137 ]


Original Secondary Outcome:

  • Number of subjects experiencing a >25% worsening, −25 to +25% no change, 25-50% improvement, 50-75% improvement or >75% improvement in convulsive seizures from baseline. [ Time Frame: 2-14 weeks ]
  • Number of subjects who are convulsive seizure free. [ Time Frame: 2-14 weeks ]
  • Mean percentage change from baseline in non-convulsive seizure frequency. [ Time Frame: 2-14 weeks ]
  • Change in types of seizures from baseline. [ Time Frame: 2-14 weeks ]
  • Caregiver Global Impression of Change (CGIC) at the end of treatment. [ Time Frame: End of week 14 of treatment ]
  • The incidence of adverse events as measure of subject safety. [ Time Frame: Day -28 to Day 137 ]
  • The number of age-appropriate subjects with a treatment-emergent flag using the Columbia-Suicide Severity Rating Scale (C-SSRS) (Children's) during the course of the study. [ Time Frame: Day -28 to Day 137 ]


Information By: GW Research Ltd

Dates:
Date Received: August 21, 2014
Date Started: March 2015
Date Completion: June 2017
Last Updated: October 11, 2016
Last Verified: October 2016