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

This study is currently recruiting participants.
Verified October 2016 by GW Research Ltd
Sponsor:
ClinicalTrials.gov Identifier:
NCT02544763
First Posted: September 9, 2015
Last Update Posted: October 12, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
GW Research Ltd
  Purpose
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 Intervention Phase
Tuberous Sclerosis Complex Seizures Drug: GWP42003-P Drug: Placebo Phase 3

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by GW Research Ltd:

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

Secondary Outcome Measures:
  • 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) ]

Other Outcome Measures:
  • Plasma concentrations of cannabidiol and its major metabolites [ Time Frame: 0, 2, and 4 hours post-dose. An additional sample was taken 6 hours post-dose in participants aged 18 years and older. ]

Estimated Enrollment: 192
Study Start Date: April 2016
Estimated Study Completion Date: July 2017
Estimated Primary Completion Date: July 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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

Key Inclusion Criteria:

  • Participant has a well-documented history of epilepsy, with compatible electroencephalogram (EEG) and clinical history.
  • 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 is taking felbamate, and they have been taking it 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 had active suicidal plan/intent in the past 6 months, or has a history of suicide attempt in the last 2 years, or more than 1 lifetime suicide attempt.
  • Participant has C-SSRS grade 4 or 5 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.
  Contacts and Locations
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


Contacts
Contact: GW Research Medical Information +44 (0) 1223 266 800 medinfo@gwpharm.com

Locations
United States, Massachusetts
Recruiting
Boston, Massachusetts, United States
Sponsors and Collaborators
GW Research Ltd
  More Information

Responsible Party: GW Research Ltd
ClinicalTrials.gov Identifier: NCT02544763     History of Changes
Other Study ID Numbers: GWEP1521 Blinded Phase
2015-002154-12 ( EudraCT Number )
First Submitted: September 7, 2015
First Posted: September 9, 2015
Last Update Posted: October 12, 2016
Last Verified: October 2016

Additional relevant MeSH terms:
Sclerosis
Seizures
Tuberous Sclerosis
Pathologic Processes
Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurologic Manifestations
Signs and Symptoms
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
Pharmaceutical Solutions