Clinical Trial: A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

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

Official Title: A Double-blind, Randomized, Placebo-controlled Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P, CBD) as Add-on Therapy in Patients With Tuberous Sclerosis

Brief Summary: This trial consists of 2 parts: a double-blinded phase and an open-label extension phase. The blinded phase only will be described in this record. Participants will receive 1 of 2 doses of GWP42003-P or matching placebo. The primary clinical hypothesis is that there will be a difference between GWP42003-P and placebo in their effect on seizure frequency.

Detailed Summary:
Sponsor: GW Research Ltd

Current Primary Outcome: Change in seizure frequency [ Time Frame: Baseline and average over the 16-week treatment period (or up to the point of withdrawal) ]

Original Primary Outcome: Change in focal seizure frequency [ Time Frame: Baseline and average over the 16-week treatment period (or up to the point of withdrawal) ]

The percentage change from baseline in number of focal seizures (average per 28 days) during the treatment period (maintenance and titration) is presented.


Current Secondary Outcome:

  • Number of treatment responders [ Time Frame: 16 weeks ]
  • Number of participants with worsening, no change, or improvements in seizure frequency [ Time Frame: 16 weeks ]
  • Change in composite focal seizure score [ Time Frame: Baseline and average over the 16-week treatment period ]
  • Change in number of seizure-free days [ Time Frame: Baseline and average over the 16-week treatment period ]
  • Change in number of seizures by subtype [ Time Frame: Baseline and average over the 16-week treatment period ]
  • Change in number of infantile/epileptic spasms. [ Time Frame: Baseline and average over the 16-week treatment period ]
  • Change in use of rescue medication [ Time Frame: Baseline and average over the 16-week treatment period ]
  • Change in number of episodes of status epilepticus [ Time Frame: Baseline and average over the 16-week treatment period ]
  • Change in duration of seizures by subtype [ Time Frame: 16 weeks ]
  • Change in overall condition as assessed by the participant/caregiver [ Time Frame: 16 weeks ]
  • Change in overall condition as assessed by the physician [ Time Frame: 16 weeks ]
  • Change in Vineland-II score [ Time Frame: Baseline and average over the 16-week treatment period ]
  • Change in Wechsler score by subtest [ Time Frame: Baseline and average over the 16-week treatment period ]
  • Change in Behavior Checklist score [ Time Frame: Baseline and average over the 16-week treatment period ]
  • Change in Social Communication Questionnaire score [ Time Frame: Baseline and average over the 16-week treatment period ]
  • Change in Quality of Life score [ Time Frame: Baseline and average over the 16-week treatment period ]
  • Change in serum IGF-1 levels [ Time Frame: Baseline and 16 weeks ]
  • Number of participants with changes in Tanner stage [ Time Frame: 16 weeks ]
  • Incidence of adverse events [ Time Frame: Screening to End of Trial (up to Week 21) ]
  • Incidence of suicidality [ Time Frame: Screening to End of Dosing (up to Week 17) ]


Original Secondary Outcome:

  • Number of treatment responders [ Time Frame: 16 weeks ]
    The number of participants considered treatment responders, defined as those with a ≥ 25%, ≥ 50%, ≥ 75% or 100% reduction in focal seizure frequency, are presented.
  • Number of participants with worsening, no change, or improvements in focal seizure frequency [ Time Frame: 16 weeks ]
    The number of participants experiencing a > 25% worsening, −25 to +25% no change, 25-50% improvement, 50-75% improvement or > 75% improvement in focal seizure frequency is presented.
  • Change in composite focal seizure score [ Time Frame: Baseline and average over the 16-week treatment period ]
    The change from baseline in composite focal seizure score (frequency × severity) is presented.
  • Change in number of focal seizure-free days [ Time Frame: Baseline and average over the 16-week treatment period ]
    The change from baseline in number of focal seizure-free days is presented.
  • Change in number of seizures by subtype [ Time Frame: Baseline and average over the 16-week treatment period ]
    The change from baseline in number of seizures of each subtype is presented.
  • Change in number of infantile/epileptic spasms. [ Time Frame: Baseline and average over the 16-week treatment period ]
    The change from baseline in number of infantile/epileptic spasms is presented.
  • Change in use of rescue medication [ Time Frame: Baseline and average over the 16-week treatment period ]
    The change from baseline in use of rescue medication is presented.
  • Change in number of episodes of status epilepticus [ Time Frame: Baseline and average over the 16-week treatment period ]
    The change from baseline in the number of episodes of status epilepticus (convulsive and non-convulsive) is presented.
  • Change in duration of seizures by subtype [ Time Frame: 16 weeks ]

    The change in the duration of seizures of each subtype was assessed using the Subject- or Caregiver Global Impression of Change in Seizure Duration (SGIC-SD/CGIC-SD), which comprise the following:

    • SGIC-SD: "Since you started treatment, please assess the average duration of your seizures (comparing your condition now to your condition before treatment) using the scale below."
    • CGIC-SD: "Since the patient started treatment, please assess the average duration of the patient's seizures (comparing their condition now to their condition before treatment) using the scale below."

    The markers are "Average duration of seizures has decreased"; "Average duration of seizures has stayed the same"; "Average duration of seizures has increased".

    The number of participants/caregivers who selected each marker at the end of treatment is presented.

  • Change in overall condition as assessed by the participant/caregiver [ Time Frame: 16 weeks ]

    The change in overall condition was assessed using the Subject- or Caregiver Global Impression of Change (SGIC/CGIC), which comprise the following:

    • SGIC: "Since you started treatment, please assess the status of your overall condition (comparing your condition now to your condition before treatment) using the scale below."
    • CGIC: "Since your child started treatment, please assess the status of your child's overall condition (comparing their condition now to their condition before treatment) using the scale below."

    The markers are "Very Much Improved"; "Much Improved"; "Slightly Improved"; "No Change"; "Slightly Worse"; "Much Worse"; "Very Much Worse".

    The number of participants/caregivers who selected each marker at the end of treatment is presented.

  • Change in overall condition as assessed by the physician [ Time Frame: 16 weeks ]

    The change in overall condition was assessed using the Physician Global Impression of Change (PGIC), which comprises the following:

    "Since the patient started treatment, please assess the status of the patient's overall condition (comparing their condition now to their condition before treatment) using the scale below."

    The markers are "Very Much Improved"

    Information By: GW Research Ltd

    Dates:
    Date Received: September 7, 2015
    Date Started: April 2016
    Date Completion: July 2017
    Last Updated: October 11, 2016
    Last Verified: October 2016