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