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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02224703
Registration number
NCT02224703
Ethics application status
Date submitted
21/08/2014
Date registered
25/08/2014
Date last updated
28/09/2022
Titles & IDs
Public title
GWPCARE2 A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet Syndrome
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Scientific 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.
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Secondary ID [1]
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2014-002939-34
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Secondary ID [2]
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GWEP1424
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Epilepsy
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Dravet Syndrome
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Condition category
Condition code
Neurological
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Epilepsy
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - GWP42003-P
Treatment: Drugs - Placebo Control
Experimental: 10 mg/kg/day GWP42003-P - GWP42003-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.
Experimental: 20 mg/kg/day GWP42003-P - GWP42003-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 Comparator: Placebo Control - Excipients 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.
Treatment: Drugs: GWP42003-P
Treatment: Drugs: Placebo Control
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change In Convulsive Seizures During The Treatment Period Compared To Baseline
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Assessment method [1]
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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.
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Timepoint [1]
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Baseline to Day 99 or Early Termination (ET)
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Secondary outcome [1]
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Change In Total Seizures During The Treatment Period Compared To Baseline
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Assessment method [1]
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Total seizures were defined as the combination of convulsive and non-convulsive seizures. Convulsive seizures were defined as tonic-clonic, tonic, clonic, or atonic seizures. Non-convulsive seizures were defined as myoclonic, countable partial, other partial, or absence seizures. Participants or their caregivers recorded the number and type of convulsive seizures and non-convulsive seizures each day from screening until completion of dosing using an IVRS diary. Change compared to baseline was calculated as per the primary outcome measure. Data reported as the ratio of geometric least squares mean in total seizures and expressed as a percentage reduction.
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Timepoint [1]
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Baseline to Day 99 or ET
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Secondary outcome [2]
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Participants With A =50% Reduction From Baseline In Convulsive Seizure Frequency During The Treatment Period
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Assessment method [2]
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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 IVRS diary. Baseline included all available data prior to Day 1 (28-day average). Percentage change from baseline was calculated as: ([frequency during the treatment period - frequency during baseline]/frequency during baseline) x 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) x 28. Baseline included all available data prior to Day 1 (28-day average).
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Timepoint [2]
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Baseline to Day 99 or ET
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Secondary outcome [3]
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Caregiver Global Impression Of Change (CGIC) At The Last Visit
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Assessment method [3]
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On Day 1 (prior to receiving study drug), the caregiver was asked to write a brief description of the participant's overall condition as a memory aid for the CGIC questionnaire at subsequent visits. The CGIC questionnaire comprised the following question, to be rated on a 7-point scale: Since your child started treatment, please assess the status of your child's overall condition (comparing their condition now to their condition before treatment) using the scale below. The markers were: "Very Much Improved"; "Much Improved"; "Slightly Improved"; "No Change"; "Slightly Worse"; "Much Worse"; "Very Much Worse". The CGIC response/score, recorded at each visit, was summarized, on both a categorical and continuous scale, by treatment group. The scores at the last scheduled visit (not including the end of taper or safety follow-up visits) at which participant's last evaluation was performed were analyzed using ordinal logistic regression.
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Timepoint [3]
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Baseline to Last Visit
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Eligibility
Key inclusion criteria
Key
- 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.
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Minimum age
2
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Maximum age
18
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- 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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
13/04/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
9/04/2018
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Sample size
Target
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Accrual to date
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Final
199
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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- Heidelberg
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Recruitment hospital [2]
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- Randwick
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Recruitment postcode(s) [1]
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3084 - Heidelberg
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Recruitment postcode(s) [2]
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NSW 2031 - Randwick
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Recruitment outside Australia
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United States of America
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Alabama
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Ramat Gan
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Heeze
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Valencia
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Funding & Sponsors
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Commercial sector/Industry
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Name
Jazz Pharmaceuticals
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Ethics approval
Ethics application status
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Summary
Brief summary
To investigate the potential antiepileptic effects of cannabidiol (GWP42003-P) in children
and young adults with Dravet syndrome.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02224703
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Contacts
Principal investigator
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02224703
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