Clinical Trial: An Open Label Extension Study of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet or Lennox-Gastaut Syndromes

Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional

Official Title: An Open Label Extension Study to Investigate the Safety of Cannabidiol (GWP42003-P; CBD) in Children and Young Adults With Inadequately Controlled Dravet or Lennox-Gastaut Syndro

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

Detailed Summary: This is a multi-center, open label extension study for patients with Dravet syndrome or Lennox-Gastaut syndrome who have previously participated in double-blind, placebo-controlled clinical studies of GWP42003-P (Core Studies). The first subject will not enroll into the open label extension study until the Data Safety Monitoring Committee has reviewed the safety data from Part A of study GWEP1332.
Sponsor: GW Research Ltd

Current Primary Outcome: The incidence of adverse events and other assessments as measure of subject safety. [ Time Frame: Subjects will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular subject, or a maximum of 3 years. ]

The number of subjects who experienced an adverse event during the study is presented. The time frame for adverse event reporting was from enrolment to the follow-up visit.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Mean change in quality of life, relative to the pre-randomization baseline of the Core Study, if assessed during the Core Study. [ Time Frame: Subjects will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular subject, or a maximum of 3 years. ]
  • Changes in the Caregiver Global Impression of Change (CGIC) or Subject Global Impression of Change score, relative to the pre-randomization baseline of the Core Study, if assessed during the Core Study. [ Time Frame: Subjects will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular subject, or a maximum of 3 years. ]
  • Mean percentage change in the frequencies of sub-types of seizures, relative to the pre-randomization baseline of the Core Study. [ Time Frame: Subjects will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular subject, or a maximum of 3 years. ]
  • Mean percentage change in total convulsive seizure frequency, relative to the pre-randomization baseline of the Core Study. [ Time Frame: Subjects will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular subject, or a maximum of 3 years. ]
  • Mean percentage change in total non-convulsive seizure frequency, relative to the pre-randomization baseline of the Core Study. [ Time Frame: Subjects will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular subject, or a maximum of 3 years. ]
  • Number of subjects considered treatment responders, defined as those with a ≥25%, ≥50%, ≥75%, or 100% reduction in convulsive seizures, relative to the pre-randomization baseline of the Core Study. [ Time Frame: Subjects will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular subject, or a maximum of 3 years. ]
  • Number of subjects experiencing a >25% worsening, −25 to +25% no change, 25-50% improvement, 50-75% improvement or >75% improvement in convulsive seizures, relative to the pre-randomization baseline of the Core Study. [ Time Frame: Subjects will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular subject, or a maximum of 3 years. ]
  • Mean percentage change in the number of drop seizures, relative to the pre-randomization baseline of the Core Study. [ Time Frame: Subjects will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular subject, or a maximum of 3 years. ]
  • Mean percentage change in the number of non-drop seizures, relative to the pre-randomization baseline of the Core Study. [ Time Frame: Subjects will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular subject, or a maximum of 3 years. ]
  • Number of subjects considered treatment responders, defined as those with a ≥25%, ≥50%, ≥75%, or 100% reduction in drop seizures, relative to the pre-randomization baseline of the Core Study. [ Time Frame: Subjects will be followed until market authorization is granted for GWP42003-P, in DS or LGS, or a compassionate program becomes available in the country of a particular subject, or a maximum of 3 years. ]


Original Secondary Outcome: Same as current

Information By: GW Research Ltd

Dates:
Date Received: August 21, 2014
Date Started: June 2015
Date Completion: June 2016
Last Updated: January 27, 2016
Last Verified: January 2016