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

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ClinicalTrials.gov Identifier: NCT02544763
Recruitment Status : Completed
First Posted : September 9, 2015
Results First Posted : September 23, 2020
Last Update Posted : October 6, 2020
Sponsor:
Information provided by (Responsible Party):
GW Research Ltd

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.

Condition or disease Intervention/treatment Phase
Tuberous Sclerosis Complex Seizures Drug: GWP42003-P Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 224 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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 Complex Who Experience Inadequately-controlled Seizures
Actual Study Start Date : April 6, 2016
Actual Primary Completion Date : January 22, 2019
Actual Study Completion Date : February 26, 2019


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.




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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)."

  3. 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.

  4. 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).



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Ages Eligible for Study:   1 Year to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02544763


Locations
Show Show 44 study locations
Sponsors and Collaborators
GW Research Ltd
  Study Documents (Full-Text)

Documents provided by GW Research Ltd:
Study Protocol  [PDF] April 23, 2019
Statistical Analysis Plan  [PDF] November 28, 2019

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: GW Research Ltd
ClinicalTrials.gov Identifier: NCT02544763    
Other Study ID Numbers: GWEP1521 Blinded Phase
2015-002154-12 ( EudraCT Number )
First Posted: September 9, 2015    Key Record Dates
Results First Posted: September 23, 2020
Last Update Posted: October 6, 2020
Last Verified: October 2020
Additional relevant MeSH terms:
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Epidiolex
Tuberous Sclerosis
Seizures
Sclerosis
Pathologic Processes
Neurologic Manifestations
Nervous System Diseases
Hamartoma
Neoplasms
Neoplasms, Multiple Primary
Neoplastic Syndromes, Hereditary
Malformations of Cortical Development, Group I
Malformations of Cortical Development
Nervous System Malformations
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Anticonvulsants