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An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

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ClinicalTrials.gov Identifier: NCT02544750
Recruitment Status : Enrolling by invitation
First Posted : September 9, 2015
Last Update Posted : October 1, 2018
Sponsor:
Information provided by (Responsible Party):
GW Research Ltd

Tracking Information
First Submitted Date  ICMJE September 7, 2015
First Posted Date  ICMJE September 9, 2015
Last Update Posted Date October 1, 2018
Study Start Date  ICMJE August 2016
Estimated Primary Completion Date February 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 13, 2016)
Incidence of adverse events [ Time Frame: From OLE screening until market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
Original Primary Outcome Measures  ICMJE
 (submitted: September 7, 2015)
Incidence of adverse events [ Time Frame: From OLE screening until market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
The number of participants who experienced an adverse event during the trial is presented.
Change History Complete list of historical versions of study NCT02544750 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 13, 2016)
  • Change in seizure frequency [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Number of treatment responders [ Time Frame: Once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Number of participants with worsening, no change, or improvements in seizure frequency [ Time Frame: Once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Change in composite focal seizure score [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Change in number of seizure-free days [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Change in number of seizures by subtype [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Change in number of infantile/epileptic spasms. [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Change in use of rescue medication [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Change in number of episodes of status epilepticus [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Change in duration of seizures by subtype [ Time Frame: Once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Change in overall condition as assessed by the participant/caregiver [ Time Frame: Once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Change in overall condition as assessed by the physician [ Time Frame: Once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Change in Vineland-II score [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Change in Wechsler score by subtest [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Change in Behavior Checklist score [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Change in Social Communication Questionnaire score [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Change in Quality of Life score [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Change in serum IGF-1 levels [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Number of participants with changes in Tanner stage [ Time Frame: Once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
  • Incidence of suicidality [ Time Frame: From OLE screening until market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
Original Secondary Outcome Measures  ICMJE
 (submitted: September 7, 2015)
  • Change in focal seizure frequency [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    The percentage change from the pre-randomization baseline of the blinded phase in number of focal seizures (average per 28 days) is presented.
  • Number of treatment responders [ Time Frame: Once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    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: Once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    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 once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    The change from the pre-randomization baseline of the blinded phase in composite focal seizure score (frequency × severity) is presented.
  • Change in number of focal seizure-free days [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    The change from the pre-randomization baseline of the blinded phase in number of focal seizure-free days is presented.
  • Change in number of seizures by subtype [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    The change from the pre-randomization baseline of the blinded phase in number of seizures of each subtype is presented.
  • Change in number of infantile/epileptic spasms. [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    The change from the pre-randomization baseline of the blinded phase in number of infantile/epileptic spasms is presented.
  • Change in use of rescue medication [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    The change from the pre-randomization baseline of the blinded phase in use of rescue medication is presented.
  • Change in number of episodes of status epilepticus [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    The change from the pre-randomization baseline of the blinded phase in the number of episodes of status epilepticus (convulsive and non-convulsive) is presented.
  • Change in duration of seizures by subtype [ Time Frame: Once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    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: Once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    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: Once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    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"; "Much Improved"; "Slightly Improved"; "No Change"; "Slightly Worse"; "Much Worse"; "Very Much Worse". The number of physicians who selected each marker at the end of treatment is presented.
  • Change in Vineland-II score [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    The change from the pre-randomization baseline of the blinded phase in the Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) score is presented.
  • Change in Wechsler score by subtest [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    The change from the pre-randomization baseline of the blinded phase in the following are presented:
    • Wechsler Preschool and Primary Scale of Intelligence - Fourth Edition (WPPSI-IV), Vocabulary and Matrix Reasoning score (for participants aged 2-6 years).
    • Wechsler Abbreviated Scale of Intelligence - Second Edition (WASI-II), Vocabulary and Matrix Reasoning score (for participants aged 6 years and older).
    • Wechsler Intelligence Scale for Children - Fourth Edition (WISC-IV) or Wechsler Adult Intelligence Scale - Fourth Edition (WAIS-IV), Digit Span and Coding score (for participants aged 6 years and older).
  • Change in Behavior Checklist score [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    The change from the pre-randomization baseline of the blinded phase in the following are presented:
    • Achenbach Child Behavior Checklist (CBCL) score.
    • Achenbach Adult Behavior Checklist (ABCL) score.
  • Change in Social Communication Questionnaire score [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    The change from the pre-randomization baseline of the blinded phase in Social Communication Questionnaire (SCQ) score is presented.
  • Change in Quality of Life score [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    The change from the pre-randomization baseline of the blinded phase in the following are presented:
    • Quality of Life in Childhood Epilepsy (QOLCE) score (for participants aged 2-18 years).
    • Patient-Weighted Quality of Life in Epilepsy (QOLIE-31-P) score (for participants aged 19 years and older).
  • Change in serum IGF-1 levels [ Time Frame: Baseline and once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    The change from the pre-randomization baseline of the blinded phase in serum concentrations of insulin-like growth factor-1 (IGF-1) is presented.
  • Number of participants with changes in Tanner stage [ Time Frame: Once market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    The number of participants under 18 years of age with changes in Tanner stage is presented.
  • Incidence of suicidality [ Time Frame: From OLE screening until market authorization is granted for GWP42003-P in TSC (anticipated to be 2 years after the OLE trial start date) ]
    Suicidality was assessed using the Columbia-Suicide Severity Rating Scale (C-SSRS; for participants 19 and older) or Children's C-SSRS (for participants aged 6-18). The number of participants with a treatment-emergent flag is presented.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)
Official Title  ICMJE 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 Complex Who Experience Inadequately-controlled Seizures
Brief Summary This trial consists of 2 parts: a double-blinded phase and an open-label extension phase. The open-label extension phase only will be described in this record. All participants will receive the same dose of GWP42003-P. However, investigators may subsequently decrease or increase the participant's dose until the optimal dose is found.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Tuberous Sclerosis Complex
  • Seizures
Intervention  ICMJE Drug: GWP42003-P
Yellow oily solution containing cannabidiol dissolved in the excipients sesame oil and anhydrous ethanol with added sweetener (sucralose) and strawberry flavoring.
Other Names:
  • Cannabidiol
  • CBD
Study Arms  ICMJE Experimental: GWP42003-P
100 mg/mL GWP42003-P oral solution taken twice daily (morning and evening). Participants will be dosed up to a maximum of 50 mg/kg/day. Dose may be lower if Investigator judges benefit and/or tolerability issues.
Intervention: Drug: GWP42003-P
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Enrolling by invitation
Estimated Enrollment  ICMJE
 (submitted: October 20, 2017)
210
Original Estimated Enrollment  ICMJE
 (submitted: September 7, 2015)
144
Estimated Study Completion Date  ICMJE February 2022
Estimated Primary Completion Date February 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Completion of the GWEP1521 Blinded Phase
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Year to 65 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02544750
Other Study ID Numbers  ICMJE GWEP1521 Open-Label Extension
2015-002154-12 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party GW Research Ltd
Study Sponsor  ICMJE GW Research Ltd
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account GW Research Ltd
Verification Date September 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP