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

September 22, 2022 updated by: Jazz Pharmaceuticals

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

224

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Heidelberg, Australia
        • Austin Health
      • Herston, Australia
        • Royal Brisbane and Women's Hospital
      • Parkville, Australia
        • The Royal Melbourne Hospital
      • Randwick, Australia
        • Sydney Children's Hospital
  • Netherlands
      • Rotterdam, Netherlands
        • Erasmus MC/Sophia Children's Hospital
      • Utrecht, Netherlands
        • UMC Utrecht/ Wilhelmina, Kinderziekenhuis
  • Poland
      • Bydgoszcz, Poland
        • Vitamed Gałaj i Cichomski spółka jawna
      • Kraków, Poland
        • Centrum Medyczne Plejady
      • Lublin, Poland
        • Wojewódzki Szpital Specjalistyczny im S. K. Wyszyńskiego SPZOZ
      • Warsaw, Poland
        • Instytut "Pomnik - Centrum Zdrowia Dziecka"
      • Warsaw, Poland
        • Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego w Warszawie
      • Wrocław, Poland
        • Centrum Neuropsychiatrii "Neuromed"
  • Spain
      • Barcelona, Spain
        • Centro Médico Teknon
      • Barcelona, Spain
        • Clinical Research Unit
      • Barcelona, Spain
        • Unitat d'Epilèpsia
      • Madrid, Spain
        • Hospital Infantil Universitario Nino Jesus
      • Pamplona, Spain
        • Clinica Universidad de Navarra
  • United Kingdom
      • Cardiff, United Kingdom
        • Cardiff and Vale University Local Health Board
      • Cardiff, United Kingdom
        • Children and Young Adults' Research Unit
      • London, United Kingdom
        • St George's University Hospitals NHS Foundation Trust
      • London, United Kingdom
        • NIHR Clinical Research Facility
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • UAB Epilepsy Center
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Arkansas Children's Hospital
    • California
      • Los Angeles, California, United States, 90095
        • UCLA-Pediatric Neurology
      • Oakland, California, United States, 94609
        • UCSF Benioff Children's Hospital Oakland
    • Colorado
      • Aurora, Colorado, United States, 80045
        • University of Colorado Denver
    • Florida
      • Miami, Florida, United States, 33155
        • Pediatric Neurology
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H. Lurie Children's Hospital of Chicago
    • Maryland
      • Bethesda, Maryland, United States, 20817
        • Mid Atlantic Epilepsy & Sleep Centre
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic
      • Saint Paul, Minnesota, United States, 55102
        • Minnesota Epilepsy Group, P.A
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University School of Medicine
    • New York
      • New York, New York, United States, 10016
        • NYU Comprehensive Epilepsy Center
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • University of North Carolina at Chapel Hill
      • Winston-Salem, North Carolina, United States, 27157
        • Wake Forest Baptist Medical Center
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health & Science University
    • Pennsylvania
      • Manchester, Pennsylvania, United States, 17345
        • WellSpan Paediatric Neurology
    • Tennessee
      • Memphis, Tennessee, United States, 38103
        • Le Bonheur Children's Hospital
    • Texas
      • Dallas, Texas, United States, 75104
        • Texas Scottish Rite Hospital for Children
      • Fort Worth, Texas, United States, 76104
        • Cook Children's Health Care System
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • Paediatric Neurology
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • University of Virginia
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 65 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Key Inclusion Criteria:

  • Participant has a well-documented clinical history of epilepsy.
  • Participant has a clinical diagnosis of Tuberous Sclerosis Complex (TSC) according to the criteria agreed by the 2012 International TSC Consensus Conference.
  • All medications or interventions for epilepsy (including ketogenic diet and any neurostimulation devices for epilepsy) must have been stable for 1 month prior to screening and the participant is willing to maintain a stable regimen throughout the trial.

Key Exclusion Criteria:

  • Participant has a history of pseudo-seizures.
  • Participant has clinically significant unstable medical conditions other than epilepsy.
  • Participant has an illness in the 4 weeks prior to screening or randomization, other than epilepsy, which in the opinion of the investigator could affect seizure frequency.
  • Participant has undergone general anesthetic in the 4 weeks prior to screening or randomization.
  • Participant has undergone surgery for epilepsy in the 6 months prior to screening.
  • Participant is being considered for epilepsy surgery or any procedure involving general anesthesia.
  • Participant has been taking felbamate for less than 1 year prior to screening.
  • Participant is taking an oral mTOR inhibitor.
  • Participant has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the Investigational Medicinal Product (IMP), such as sesame oil.
  • Participant has any history of suicidal behavior or any suicidal ideation of type 4 or 5 on the C-SSRS in the last month or at screening.
  • Participant is currently using or has in the past used recreational or medicinal cannabis, or cannabinoid-based medications, within the 3 months prior to screening and is unwilling to abstain for the duration for the study.
  • Participant has tumor growth which, in the opinion of the Investigator, could affect the primary endpoint.
  • Participant has significantly impaired hepatic function at the screening or randomization visit
  • Participant has received an IMP within the 12 weeks prior to the screening visit.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 25 mg/kg/day GWP42003-P
100 mg/mL GWP42003-P oral solution taken twice daily (morning and evening).
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
Experimental: 50 mg/kg/day GWP42003-P
100 mg/mL GWP42003-P oral solution taken twice daily (morning and evening).
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
Placebo Comparator: Placebo
Placebo oral solution matching 100 mg/mL GWP42003-P.
Drug: Placebo
Yellow oily solution containing the excipients sesame oil and anhydrous ethanol with added sweetener (sucralose) and strawberry flavoring.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percent Change From Baseline in the Number of Tuberous Sclerosis Complex (TSC)-Associated Seizures During the Treatment Period (Maintenance and Titration)
Time Frame: Baseline; up to Week 16
TSC-associated seizures included: focal motor seizures without impairment of consciousness or awareness (Type 1 focal motor); focal seizures with impairment of consciousness or awareness (Type 2 focal); focal seizures evolving to bilateral generalized convulsive seizures (Type 3 focal); and tonic-clonic, tonic, clonic, or atonic seizures that are countable. Percent change from Baseline was calculated as the (post-Baseline value minus the Baseline value) divided by the Baseline value x 100.
Baseline; up to Week 16

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants Considered Treatment Responders During the Treatment Period (Maintenance and Titration)
Time Frame: Baseline; up to Week 16
Treatment responders are defined as those participants with a ≥ 50% reduction in TSC-associated seizure frequency. TSC-associated seizures included: focal motor seizures without impairment of consciousness or awareness (Type 1 focal motor); focal seizures with impairment of consciousness or awareness (Type 2 focal); focal seizures evolving to bilateral generalized convulsive seizures (Type 3 focal); and tonic-clonic, tonic, clonic, or atonic seizures that are countable. Participants who withdrew from the trial during the treatment period are considered non-responders.
Baseline; up to Week 16
Change From Baseline in the Caregiver Global Impression of Change (CGIC) or Participant Global Impression of Change (PGIC) Score at the Participant's Last Visit
Time Frame: Baseline; up to Week 16
The combined caregiver and participant summary uses either the caregiver or participant version if only one version was completed, or the caregiver version if both caregiver and participant versions were completed. The CGIC comprised the following question, to be rated on a 7-point scale (1, Very Much Improved; 2, Much Improved; 3, Slightly Improved; 4, No Change; 5, Slightly Worse; 6, Much Worse; 7, Very Much Worse): "Since your child started treatment, please assess the status of your child's overall condition (comparing their condition now to their condition before treatment)." The SGIC comprised the following question, to be rated on a 7-point scale (1, Very Much Improved; 2, Much Improved; 3, Slightly Improved; 4, No Change; 5, Slightly Worse; 6, Much Worse; 7, Very Much Worse): "Since you started treatment, please assess the status of your overall condition (comparing your condition now to your condition before treatment)."
Baseline; up to Week 16
Percent Change From Baseline in Total Seizures During the Treatment Period (Maintenance and Titration)
Time Frame: Baseline; up to Week 16
Total seizures included all seizure types combined. Percent change from Baseline was calculated as the (post-Baseline value minus the Baseline value) divided by the Baseline value x 100.
Baseline; up to Week 16
Number of Participants With Any Severe Treatment-emergent Adverse Event (TEAE)
Time Frame: up to approximately Week 22
A TEAE was defined as an AE with a start date on or after the first dose of IMP during the Blinded Phase up to and including the date of first dose of the Open-label Extension (OLE) Phase (OLE Day 1).
up to approximately Week 22

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Jazz Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 6, 2016

Primary Completion (Actual)

January 22, 2019

Study Completion (Actual)

February 26, 2019

Study Registration Dates

First Submitted

September 7, 2015

First Submitted That Met QC Criteria

September 7, 2015

First Posted (Estimate)

September 9, 2015

Study Record Updates

Last Update Posted (Actual)

September 28, 2022

Last Update Submitted That Met QC Criteria

September 22, 2022

Last Verified

September 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GWEP1521 Blinded Phase
  • 2015-002154-12 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Seizures

Clinical Trials on GWP42003-P

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Senegal

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe