Antiepileptic Efficacy Study of GWP42003-P in Children and Young Adults With Dravet Syndrome (GWPCARE1)
A Double Blind, Placebo Controlled Two-part Study to Investigate the Dose-ranging Safety and Pharmacokinetics, Followed by the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet Syndrome
2 other identifiers
interventional
120
4 countries
22
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 Mar 2015
Shorter than P25 for phase_3
22 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 17, 2014
CompletedFirst Posted
Study publicly available on registry
March 19, 2014
CompletedStudy Start
First participant enrolled
March 30, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 26, 2015
CompletedStudy Completion
Last participant's last visit for all outcomes
November 26, 2015
CompletedResults Posted
Study results publicly available
July 20, 2018
CompletedSeptember 28, 2022
September 1, 2022
8 months
March 17, 2014
June 25, 2018
September 22, 2022
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Percentage Change From Baseline In Convulsive Seizure Frequency During The Treatment Period
Convulsive seizures (atonic, clonic, tonic, or tonic-clonic) were recorded by the participant or caregiver using an interactive voice response system (IVRS) diary. Percentage change from baseline was calculated as: (\[frequency during the treatment period - frequency during baseline\]/frequency during baseline) \* 100. The frequency during each period was based on 28-day averages and calculated as: (number of seizures in the period/number of reported days in the IVRS period) \* 28. Baseline included all available data prior to Day 1 (28-day average). Negative percentages show an improvement from baseline.
Baseline to End of Treatment (EOT) (Day 99) or Early Termination (ET)
Secondary Outcomes (11)
Number Of Participants With A ≥50% Reduction From Baseline In Convulsive Seizure Frequency During The Treatment Period
Baseline to EOT (Day 99) or ET
Number of Participants With A ≥25%, ≥75% Or 100% Reduction From Baseline In Convulsive Seizure Frequency During The Treatment Period
Baseline to EOT (Day 99) or ET
Percentage Change From Baseline In Non-Convulsive Seizure Frequency During The Treatment Period
Baseline to EOT (Day 99) or ET
Caregiver Global Impression Of Change In Seizure Duration (CGICSD)
Baseline to EOT (Day 99) or ET
Number Of Participants Using Rescue Medication
Baseline to EOT (Day 99) or ET
- +6 more secondary outcomes
Study Arms (2)
GWP42003-P 20 mg/kg/day Dose
EXPERIMENTALParticipants received 20 mg/kg/day of GWP42003-P administered orally, half in the morning and half in the evening. Participants titrated GWP42003-P to 20 mg/kg/day over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day) period.
Placebo
PLACEBO COMPARATORParticipants received placebo (0 mg/mL CBD), volume-matched to the 20 mg/kg/day dose level, administered orally, half in the morning and half in the evening. To maintain the blinded aspect of the study, participants titrated the placebo dose over 11 days and remained at this dose for the 12-week maintenance period. If the participant did not immediately enter the OLE study, the maintenance period was followed by a 10-day taper (10% per day of the matched dose) period.
Interventions
GWP42003-P was an oral solution containing 100 mg/milliliter (mL) cannabidiol (CBD) dissolved in the excipients, sesame oil and anhydrous ethanol (79 mg/mL), with added sweetener (0.5 mg/mL sucralose) and strawberry flavoring (0.2 mg/mL).
Placebo oral solution contained the excipients, sesame oil and anhydrous ethanol (79 mg/mL), with added sweetener (0.5 mg/mL sucralose) and strawberry flavoring (0.2 mg/mL).
Eligibility Criteria
You may qualify if:
- Participants were male or female aged between 2 and 18 years (inclusive).
- Participants had a documented history of Dravet Syndrome that was not completely controlled by current antiepileptic drugs.
- Participants took one or more antiepileptic drugs at a dose that had been stable for at least four weeks.
- All medications or interventions for epilepsy (including ketogenic diet and vagus nerve stimulation) were stable for four weeks prior to screening and participants were willing to maintain a stable regimen throughout the study.
You may not qualify if:
- Participants had clinically significant unstable medical conditions other than epilepsy.
- Participants had clinically relevant symptoms or a clinically significant illness in the four weeks prior to screening or randomization, other than epilepsy.
- Participants were currently using or had in the past used recreational or medicinal cannabis or synthetic cannabinoid based medications (including Sativex®) within the three months prior to study entry and were unwilling to abstain for the duration for the study.
- Participants had any known or suspected hypersensitivity to cannabinoids or any of the excipients of the investigational medicinal products.
- Participants had been part of a previous clinical trial involving another investigational product in the previous six months.
- There were plans for the participants to travel outside their country of residence during the study.
- Participants previously randomized into this study. In particular, participants who participated in Part A of the study could not enter Part B.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (22)
Unknown Facility
Miami, Florida, 33155, United States
Unknown Facility
Orlando, Florida, 32819, United States
Unknown Facility
Atlanta, Georgia, 30328, United States
Unknown Facility
Chicago, Illinois, 60611, United States
Unknown Facility
Iowa City, Iowa, 52242, United States
Unknown Facility
Boston, Massachusetts, 02114, United States
Unknown Facility
Rochester, Minnesota, 55905, United States
Unknown Facility
New York, New York, 10016, United States
Unknown Facility
Winston-Salem, North Carolina, 27408, United States
Unknown Facility
Columbus, Ohio, 43205, United States
Unknown Facility
Philadelphia, Pennsylvania, 19104, United States
Unknown Facility
Houston, Texas, 77030, United States
Unknown Facility
Salt Lake City, Utah, 84113, United States
Unknown Facility
Marseille, 13385, France
Unknown Facility
Paris, 75015, France
Unknown Facility
Strasbourg, 67098, France
Unknown Facility
Toulouse, 70034, France
Unknown Facility
Gdansk, 80-952, Poland
Unknown Facility
Krakow, 30-349, Poland
Unknown Facility
Glasgow, G51 4TF, United Kingdom
Unknown Facility
Liverpool, L12 2AP, United Kingdom
Unknown Facility
London, WC1N 3JH, United Kingdom
Related Publications (2)
Devinsky O, Cross JH, Laux L, Marsh E, Miller I, Nabbout R, Scheffer IE, Thiele EA, Wright S; Cannabidiol in Dravet Syndrome Study Group. Trial of Cannabidiol for Drug-Resistant Seizures in the Dravet Syndrome. N Engl J Med. 2017 May 25;376(21):2011-2020. doi: 10.1056/NEJMoa1611618.
PMID: 28538134BACKGROUNDMadan 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: 34265088DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Enquires
- 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
March 17, 2014
First Posted
March 19, 2014
Study Start
March 30, 2015
Primary Completion
November 26, 2015
Study Completion
November 26, 2015
Last Updated
September 28, 2022
Results First Posted
July 20, 2018
Record last verified: 2022-09