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GWPCARE2 A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet Syndrome

This study is currently recruiting participants.
See Contacts and Locations
Verified October 2016 by GW Research Ltd
Sponsor:
Information provided by (Responsible Party):
GW Research Ltd
ClinicalTrials.gov Identifier:
NCT02224703
First received: August 21, 2014
Last updated: October 11, 2016
Last verified: October 2016
  Purpose
To investigate the potential antiepileptic effects of cannabidiol (GWP42003-P) in children and young adults with Dravet syndrome.

Condition Intervention Phase
Epilepsy Dravet Syndrome Drug: GWP42003-P Drug: Placebo Control Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-blind, Placebo-controlled Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet Syndrome.

Resource links provided by NLM:


Further study details as provided by GW Research Ltd:

Primary Outcome Measures:
  • Percentage change from baseline in convulsive seizure frequency during the treatment period. [ Time Frame: 0-14 weeks ]
    The primary endpoint is the percentage change from baseline in convulsive seizure frequency (average per 28 days) during the treatment period (Day 1 to the end of the evaluable period) in subjects taking GWP42003-P compared with placebo. Non-parametric analyses will be used for the primary endpoint should the assumptions for parametric analyses (e.g. Normality) not be valid.


Secondary Outcome Measures:
  • Number of subjects considered treatment responders, defined as those with a ≥25%, ≥50% or ≥75% reduction in convulsive seizures from baseline. [ Time Frame: 0-14 weeks ]
  • Number of subjects who are convulsive seizure free. [ Time Frame: 0-14 weeks ]
  • Percentage change from baseline in non-convulsive seizure frequency. [ Time Frame: 0-14 weeks ]
  • Change in types of seizures from baseline. [ Time Frame: 0-14 weeks ]
  • Caregiver Global Impression of Change (CGIC) at the end of treatment. [ Time Frame: End of week 14 of treatment ]
  • The incidence of adverse events as measure of subject safety. [ Time Frame: Day -28 to Day 137 ]
  • The number of age-appropriate subjects with a treatment-emergent flag using the Columbia-Suicide Severity Rating Scale (C-SSRS) (Children's) during the course of the study. [ Time Frame: Day -28 to Day 137 ]

Estimated Enrollment: 150
Study Start Date: March 2015
Estimated Study Completion Date: June 2017
Estimated Primary Completion Date: May 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: High Dose Level GWP42003-P
GWP42003-P oral solution (100 mg/mL cannabidiol in sesame oil with anhydrous ethanol with added sweetener [sucralose] and strawberry flavoring). The High Dose Level will be as recommended by the DSMC after assessment of safety and pharmacokinetic data from Part A of study GWEP1332.
Drug: GWP42003-P
Other Names:
  • Cannabidiol
  • CBD
Experimental: Low Dose Level GWP42003-P
GWP42003-P oral solution (100 mg/mL cannabidiol in sesame oil with anhydrous ethanol with added sweetener [sucralose] and strawberry flavoring). The Low Dose Level will be defined as 50% of the High Dose Level.
Drug: GWP42003-P
Other Names:
  • Cannabidiol
  • CBD
Placebo Comparator: Placebo Control
Excipients only. Patients will be split into two cohorts, half receiving High Dose Level dosing volumes and half receiving Low Dose Level dosing volumes.
Drug: Placebo Control
Other Name: Placebo

Detailed Description:

This study is a 1:1:1 randomized, double-blind, 14-week comparison of two Dose Levels of GWP42003-P versus placebo. A 28-day screening period prior to randomization (to establish baseline parameters) will precede the treatment period, which will consist of a two-week titration period followed by a 12-week maintenance period. The study will aim to determine the efficacy, safety and tolerability of GWP42003-P compared with placebo. The High Dose Level will be as recommended by the Data Safety Monitoring Committee (DSMC) after assessment of safety and pharmacokinetic data from Part A of study GWEP1332. The Low Dose Level will be defined as 50% of the High Dose Level. The first subject will not enroll into this study until the DSMC has reviewed the safety data from Part A of study GWEP1332.

Following study completion, all subjects will be invited to continue to receive GWP42003-P in an open label extension (OLE) study (under a separate protocol).

  Eligibility

Ages Eligible for Study:   2 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Subject must be male or female aged between 2 and 18 years (inclusive).
  • Subject must have a documented history of Dravet syndrome which is not completely controlled by current antiepileptic drugs.
  • Subject must be taking one or more antiepileptic drugs at a dose which has been stable for at least four weeks.
  • All medications or interventions for epilepsy (including ketogenic diet and vagus nerve stimulation) must have been stable for four weeks prior to screening and subject is willing to maintain a stable regimen throughout the study.

Key Exclusion Criteria:

  • Subject has clinically significant unstable medical conditions other than epilepsy.
  • Subject has had clinically relevant symptoms or a clinically significant illness in the four weeks prior to screening or randomization, other than epilepsy.
  • Subject is currently using or has in the past used recreational or medicinal cannabis, or synthetic cannabinoid based medications (including Sativex®) within the three months prior to study entry and is unwilling to abstain for the duration for the study.
  • Subject has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the investigational medicinal products.
  • There are plans for the subject to travel outside their country of residence during the study.
  • Any history of suicidal behavior or any suicidal ideation of type four or five on the Columbia-Suicide Severity Rating Scale (Children's) at screening.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02224703

Contacts
Contact: GW Research Medical Information +44 (0) 1223 266800 mailto:medinfo@gwpharm.com

Locations
United States, California
Sutter Neuroscience Institute Recruiting
Sacramento, California, United States, 95816
Contact: GW Medical Information    +44(0)1223 266800    medinfo@gwpharm.com   
Principal Investigator: Michael Chez, MD         
United States, Florida
Miami Children's Hospital Recruiting
Miami, Florida, United States, 33155
Contact: GW Medical Information    +44 (0) 1223 266800    medinfo@gwpharm.com   
Principal Investigator: Ian Miller, MD         
United States, Iowa
McFarland Clinic Recruiting
Ames, Iowa, United States, 50010
Contact: GW Medical Information    +44(0)1223 266800    medinfo@gwpharm.com   
Principal Investigator: David Moore, MD         
United States, Kentucky
University of Kentucky Medical Center Recruiting
Lexington, Kentucky, United States, 40536
Contact: GW Medical Information    +44(0) 1223 266800    medinfo@gwpharm.com   
Principal Investigator: Siddharth Kapoor, MD         
United States, Minnesota
Minnesota Epilepsy Group Recruiting
St Paul, Minnesota, United States, 55102
Contact: GW Medical Information    +44(0)1223 266800    medinfo@gwpharm.com   
Principal Investigator: Dimitros Arkilo, MD         
United States, Missouri
St Lukes Hospital Recruiting
Chesterfield, Missouri, United States, 63017
Contact: GW Medical Information    +44(0)1223 266800    medinfo@gwpharm.com   
Principal Investigator: William Rosenfeld, MD         
United States, New Hampshire
Dartmouth Hitchcock Medical Center Recruiting
Lebanon, New Hampshire, United States, 03756
Contact: GW Medical Information    +44(0)1223 266800    medinfo@gwpharm.com   
Principal Investigator: Richard Morse, MD         
United States, New York
Women and Children's Hospital of Buffalo Recruiting
Buffalo, New York, United States, 14222
Contact: GW Medical Information    +44(0) 1223 266800    medinfo@gwpharm.com   
Principal Investigator: Arie Weinstock, MD         
United States, Virginia
Valley Health Winchester Medical Center Recruiting
Winchester, Virginia, United States, 22601
Contact: GW Medical Information    +44(0)1223 266800    medinfo@gwpharm.com   
Principal Investigator: Paul Lyons, MD         
Netherlands
SEIN Recruiting
Zwolle, Netherlands
Contact: GW Medical Information    +44 (0) 1223 266800    medinfo@gwpharm.com   
Sponsors and Collaborators
GW Research Ltd
  More Information

Responsible Party: GW Research Ltd
ClinicalTrials.gov Identifier: NCT02224703     History of Changes
Other Study ID Numbers: GWEP1424
2014-002939-34 ( EudraCT Number )
Study First Received: August 21, 2014
Last Updated: October 11, 2016

Keywords provided by GW Research Ltd:
Cannabidiol
CBD
GWP42003-P

Additional relevant MeSH terms:
Syndrome
Epilepsies, Myoclonic
Disease
Pathologic Processes
Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases

ClinicalTrials.gov processed this record on June 23, 2017