GWPCARE2 A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet Syndrome
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.
2 other identifiers
interventional
199
6 countries
38
Brief Summary
To investigate the potential antiepileptic effects of cannabidiol (GWP42003-P) in children and young adults with Dravet syndrome.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Apr 2015
Typical duration for phase_3
38 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 21, 2014
CompletedFirst Posted
Study publicly available on registry
August 25, 2014
CompletedStudy Start
First participant enrolled
April 13, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 9, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
April 9, 2018
CompletedResults Posted
Study results publicly available
August 1, 2019
CompletedSeptember 28, 2022
September 1, 2022
3 years
August 21, 2014
July 11, 2019
September 22, 2022
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change In Convulsive Seizures During The Treatment Period Compared To Baseline
Convulsive seizures were defined as tonic-clonic, tonic, clonic, or atonic. Participants or their caregivers recorded the number and type of convulsive seizures each day from screening until completion of dosing using an interactive voice response system (IVRS) diary. The primary endpoint was analyzed using negative binomial regression on the sum of the convulsive seizure counts during the treatment period, based on the ITT analysis set. Baseline included all available data prior to Day 1. Data reported as the ratio of geometric least squares mean in convulsive seizures and expressed as a percentage reduction.
Baseline to Day 99 or Early Termination (ET)
Secondary Outcomes (3)
Change In Total Seizures During The Treatment Period Compared To Baseline
Baseline to Day 99 or ET
Participants With A ≥50% Reduction From Baseline In Convulsive Seizure Frequency During The Treatment Period
Baseline to Day 99 or ET
Caregiver Global Impression Of Change (CGIC) At The Last Visit
Baseline to Last Visit
Study Arms (3)
10 mg/kg/day GWP42003-P
EXPERIMENTALGWP42003-P oral solution (100 mg/milliliter \[mL\] cannabidiol in sesame oil with anhydrous ethanol with added sweetener \[sucralose\] and strawberry flavoring). The 10 mg/kg/day dose was defined as 50% of the 20 mg/kg/day dose.
20 mg/kg/day GWP42003-P
EXPERIMENTALGWP42003-P oral solution (100 mg/mL cannabidiol in sesame oil with anhydrous ethanol with added sweetener \[sucralose\] and strawberry flavoring). The 20 mg/kg/day dose was recommended by the DSMC after assessment of safety and pharmacokinetic data from Part A of study GWEP1332 (NCT02091206).
Placebo Control
PLACEBO COMPARATORExcipients only. Participants were pooled from 2 placebo cohorts, half receiving 10 mg/kg/day dose-volume equivalent and half receiving 20 mg/kg/day dose-volume equivalent.
Interventions
Eligibility Criteria
You may qualify if:
- Participant must have been male or female, aged between 2 and 18 years (inclusive).
- Participant must have had a documented history of Dravet syndrome that was not completely controlled by current antiepileptic drugs.
- Participant must have been taking 1 or more antiepileptic drugs at a dose that had been stable for at least 4 weeks.
- All medications or interventions for epilepsy (including ketogenic diet and vagus nerve stimulation) must have been stable for 4 weeks prior to screening and participant was willing to maintain a stable regimen throughout the study.
You may not qualify if:
- Participant had clinically significant unstable medical conditions other than epilepsy.
- Participant had clinically relevant symptoms or a clinically significant illness in the 4 weeks prior to screening or randomization, other than epilepsy.
- Participant was currently using or had in the past used recreational cannabis, medicinal cannabis, or synthetic cannabinoid-based medications (including Sativex®) within the 3 months prior to study entry and was unwilling to abstain for the duration for the study.
- Participant had any known or suspected hypersensitivity to cannabinoids or any of the excipients of the investigational medicinal products.
- There were plans for the participant 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (38)
Unknown Facility
Birmingham, Alabama, 35233, United States
Unknown Facility
Little Rock, Arkansas, 72202, United States
Unknown Facility
Los Angeles, California, 90027, United States
Unknown Facility
Sacramento, California, 95816, United States
Unknown Facility
Hartford, Connecticut, 06106, United States
Unknown Facility
Miami, Florida, 33155, United States
Unknown Facility
Savannah, Georgia, 31404, United States
Unknown Facility
Chicago, Illinois, 60611, United States
Unknown Facility
Lexington, Kentucky, 40536-0284, United States
Unknown Facility
Louisville, Kentucky, 40202, United States
Unknown Facility
Saint Paul, Minnesota, 55102, United States
Unknown Facility
St Louis, Missouri, 63141, United States
Unknown Facility
Omaha, Nebraska, 68106, United States
Unknown Facility
Lebanon, New Hampshire, 03756, United States
Unknown Facility
Buffalo, New York, 14203, United States
Unknown Facility
Chapel Hill, North Carolina, 27599, United States
Unknown Facility
Portland, Oregon, 97239, United States
Unknown Facility
Philadelphia, Pennsylvania, 19104-4399, United States
Unknown Facility
Charleston, South Carolina, 29425, United States
Unknown Facility
Austin, Texas, 78723, United States
Unknown Facility
Fort Worth, Texas, 76104, United States
Unknown Facility
Richmond, Virginia, 23298-0211, United States
Unknown Facility
Seattle, Washington, 98105, United States
Unknown Facility
Heidelberg, 3084, Australia
Unknown Facility
Randwick, NSW 2031, Australia
Unknown Facility
Ramat Gan, 52621, Israel
Unknown Facility
Heeze, 5591 VE, Netherlands
Unknown Facility
Zwolle, 8025 BV, Netherlands
Unknown Facility
Krakow, 30-363, Poland
Unknown Facility
Lodz, 93-271, Poland
Unknown Facility
Warsaw, 04-730, Poland
Unknown Facility
Barcelona, 08022, Spain
Unknown Facility
Madrid, 28009, Spain
Unknown Facility
Madrid, 28034, Spain
Unknown Facility
Madrid, 28222, Spain
Unknown Facility
Pamplona, 31008, Spain
Unknown Facility
Seville, 41013, Spain
Unknown Facility
Valencia, 46026, Spain
Related Publications (2)
Madan Cohen J, Checketts D, Dunayevich E, Gunning B, Hyslop A, Madhavan D, Villanueva V, Zolnowska M, Zuberi SM. Time to onset of cannabidiol treatment effects in Dravet syndrome: Analysis from two randomized controlled trials. Epilepsia. 2021 Sep;62(9):2218-2227. doi: 10.1111/epi.16974. Epub 2021 Jul 15.
PMID: 34265088DERIVEDMiller I, Scheffer IE, Gunning B, Sanchez-Carpintero R, Gil-Nagel A, Perry MS, Saneto RP, Checketts D, Dunayevich E, Knappertz V; GWPCARE2 Study Group. Dose-Ranging Effect of Adjunctive Oral Cannabidiol vs Placebo on Convulsive Seizure Frequency in Dravet Syndrome: A Randomized Clinical Trial. JAMA Neurol. 2020 May 1;77(5):613-621. doi: 10.1001/jamaneurol.2020.0073.
PMID: 32119035DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Enquiries
- Organization
- GW Research Ltd
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- GT60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 21, 2014
First Posted
August 25, 2014
Study Start
April 13, 2015
Primary Completion
April 9, 2018
Study Completion
April 9, 2018
Last Updated
September 28, 2022
Results First Posted
August 1, 2019
Record last verified: 2022-09