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    EudraCT Number:2014-002939-34
    Sponsor's Protocol Code Number:GWEP1424
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-01-25
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2014-002939-34
    A.3Full title of the trial
    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.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of the safety and efficacy of cannabidiol (GWP42003-P) in children and young adults with Dravet syndrome
    A.4.1Sponsor's protocol code numberGWEP1424
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorGW Research Ltd
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportGW Research Ltd
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGW Research Ltd
    B.5.2Functional name of contact pointGW Research Ltd Switchboard
    B.5.3 Address:
    B.5.3.1Street AddressSovereign House, Vision Park, Chivers Way
    B.5.3.2Town/ cityHiston, Cambridge
    B.5.3.3Post codeCB24 9BZ
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+441223266800
    B.5.5Fax number+441223235667
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameCannabidiol (CBD)
    D.3.2Product code GWP42003-P
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNN/A
    D.3.9.1CAS number 13956-29-1
    D.3.9.2Current sponsor codeGWP42003-P
    D.3.9.3Other descriptive nameCANNABIDIOL
    D.3.9.4EV Substance CodeSUB26600
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product Yes
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product Yes
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOral solution
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Dravet syndrome
    E.1.1.1Medical condition in easily understood language
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10073677
    E.1.2Term Severe myoclonic epilepsy of infancy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the efficacy of GWP42003-P as an adjunctive antiepileptic treatment compared with placebo, with respect to the percentage change from baseline during the treatment period of the study in convulsive seizure frequency. The dose response effect between two GWP42003-P Dose Levels (10 mg/kg/day and 20 mg/kg/day) and placebo will also be explored. Convulsive seizures are defined as tonic-clonic, tonic, clonic or atonic and non-convulsive seizures as myoclonic, partial or absence.
    E.2.2Secondary objectives of the trial
    • To assess changes from baseline in non-convulsive seizure frequency, duration, usage of rescue medication, number of
    inpatient hospitalizations due to epilepsy, sleep disruption, daytime sleepiness, quality of life, growth and development, and conduct behavioral and cognitive assessments in patients taking GWP42003-P as an adjunctive treatment, when compared with placebo.
    • To determine the pharmacokinetics (PKs) of cannabidiol (CBD) and its major metabolites following single and multiple doses of GWP42003-P.
    • To determine effects of GWP42003-P on plasma concentrations of concomitant antiepileptic drugs (AEDs), where available.
    • To assess the safety of both GWP42003-P doses when compared with placebo.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Patient and/or parent(s)/legal representative must be willing and able to give informed assent/consent for participation in the study.
    • Patient and their caregiver must be willing and able (in the investigator’s opinion) to comply with all study requirements.
    • Patient must be male or female aged between two and 18 years (inclusive).
    • Patient must have a documented history of DS which is not completely controlled by current AEDs.
    • Patient must be experiencing four or more convulsive seizures (i.e., tonic-clonic, tonic, clonic, atonic seizures) during the first 28 days of the baseline period.
    • Patient must be taking one or more AEDs at a dose which has/have been stable for at least four weeks.
    • All medications or interventions for epilepsy (including ketogenic diet and vagus nerve stimulation [VNS]) must have been stable for four weeks prior to screening and patient and caregiver are willing to maintain a stable regimen throughout the study. The ketogenic diet and VNS treatments are not counted as an AED.
    • Patient and/or parent(s)/legal representative is willing to allow his or her primary care practitioner and consultant to be notified of participation in the study.
    • Patient has completed their Interactive Voice Response System (IVRS) telephone diary on at least 25 days of the baseline period; patients who are non-compliant will be deemed ineligible to continue.
    E.4Principal exclusion criteria
    • Patient has clinically significant unstable medical conditions other than epilepsy.
    • Patient has had clinically relevant symptoms or a clinically significant illness in the four weeks prior to screening or randomization, other than epilepsy.
    • Patient has clinically significant abnormal laboratory values, in the investigator’s opinion, at screening or randomization.
    • Patient has clinically relevant abnormalities in the ECG measured at screening or randomization.
    • Patient has any concurrent cardiovascular conditions which will, in the investigator’s opinion, interfere with the ability to assess their ECGs.
    • Patient has a history or presence of alcohol or substance abuse within the last two years prior to the study or daily consumption of five or more alcohol-containing beverages.
    • Patient 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.
    • Patient is unwilling to abstain from using recreational or medicinal cannabis, or synthetic cannabinoid based medications (including Sativex®) during the study.
    • Patient has a history of symptoms (e.g., dizziness, light-headedness, blurred vision, palpitations, weakness, syncope) related to a drop in blood pressure due to postural changes.
    • Patient has ingested alcohol in the 24-hour period prior to the first study visit and/or is unwilling to abstain from drinking alcohol throughout the treatment period.
    • Patient has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the IMPs (e.g., sesame oil).
    • Female patient is of child bearing potential or male patient’s partner is of child bearing potential; unless willing to ensure that they or their partner use highly effective contraception for the duration of the study and for three months thereafter. Highly effective methods of contraception are defined as those, alone or in combination, that result in a low failure rate (i.e., less than 1% per year) when used consistently and correctly. Such methods include hormonal contraceptives, intrauterine devices/hormone-releasing systems, bilateral tubal occlusion, vasectomized partner or sexual abstinence.
    • Female patient who is pregnant (positive pregnancy test), lactating or planning pregnancy during the course of the study and for three months thereafter.
    • Patient has been part of a clinical trial involving another IMP in the previous six months.
    • Patient is taking felbamate and they have been taking it for less than one year prior to screening.
    • Any other significant disease or disorder which, in the opinion of the investigator, may either put the patient at risk because of participation in the study, may influence the result of the study, or affect the patient’s ability to participate in the study.
    • Patient has significantly impaired hepatic function at screening (Visit 1) or randomization (Visit 2), defined as any of the
    - Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >5 x upper limit of normal (ULN).
    - ALT or AST >3 x ULN and (total bilirubin [TBL] >2 x ULN or international normalized ratio [INR] >1.5).
    - ALT or AST >3 × ULN with the presence of fatigue, nausea, vomiting, right upper quadrant pain or tenderness, fever, rash, and/or eosinophilia (>5%).
    • Following a physical examination the patient has any abnormalities that, in the opinion of the investigator, would prevent the patient from safe participation in the study.
    • Patient is unwilling to abstain from donation of blood during the study.
    • There are plans for the patient to travel outside their country of residence during the study.
    • Patient has previously been randomized into this study.
    • Any history of suicidal behavior or any suicidal ideation of type four or five on the C-SSRS at screening.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the mean percentage change from baseline in total convulsive seizure frequency during the treatment period (Day 1 to the end of the evaluable period) in patients taking GWP42003-P compared with placebo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Assessed from Visit 2 to Visit 8 (Day 1 to Day 99).
    E.5.2Secondary end point(s)
    The following endpoints will be compared between treatment groups over the 14-week, double-blind treatment period:
    • Number of patients experiencing a >25% worsening, −25 to +25% no change, 25–50% improvement, 50–75% improvement or >75% improvement in convulsive seizures from baseline.
    • Number of patients considered treatment responders, defined as those with a ≥25%, ≥50% or ≥75% reduction in convulsive seizures from baseline (overall and four-weekly).
    • Number of patients who are convulsive seizure free.
    • Percentage changes from baseline in total non-convulsive seizure frequency.
    • Change in types of seizures.
    • Changes from baseline in number of episodes of status epilepticus.
    • Changes from baseline in duration of seizure subtypes as assessed by the Caregiver Global Impression of Change in Seizure Duration (CGICSD).
    • Changes from baseline in usage of rescue medication.
    • Changes from baseline in number of inpatient hospitalizations due to epilepsy.
    • Changes from baseline in Sleep Disruption 0–10 Numerical Rating Scale (0–10 NRS) score.
    • Changes from baseline in Epworth Daytime Sleepiness Scale (EDSS) score.
    • Changes from baseline in the Quality of Life in Childhood Epilepsy (QOLCE) score.
    • Changes from baseline in the Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) score.
    • Change from baseline in cognitive function as measured with a cognitive assessment battery.
    • Change from baseline in growth and development by measurement of height, weight, insulin-like growth factor-1 (IGF-1) levels and Tanner Staging (for patients aged 10–17 [inclusive], or earlier if clinically indicated by onset of menarche or other signs of precocious puberty).
    • Changes from baseline in the Caregiver Global Impression of Change (CGIC) score.
    • The plasma concentration/time curve of CBD and its major metabolites will be described following single and multiple doses of GWP42003-P, with the aim being to define:
    - Peak plasma concentration (Cmax).
    - Time to peak concentration (tmax).
    - Area under the plasma concentration curve from time zero to infinity (AUC(0–∞)) or to the last measurable concentration (AUC(0–tz)).
    - Terminal half-life (t½).
    • Plasma concentrations of concomitant AEDs before and after treatment with GWP42003-P, where available.
    The safety profile of GWP42003-P compared with placebo will also be the assessed at each Dose Level by measuring:
    • Adverse events (AEs).
    • Vital signs.
    • Physical examination parameters.
    • 12-lead Electrocardiogram (ECG).
    • Clinical laboratory parameters.
    • Columbia-Suicide Severity Rating Scale (C-SSRS) score.
    • Cannabis Withdrawal Scale (CWS) or Pediatric Cannabinoid Withdrawal Scale (PCWS) score, as appropriate.
    • Abuse liability.
    • Effects on menstruation cycles (in females).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Assessed from Visit 2 to Visit 8 (Day 1 to Day 99).
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E. trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA16
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 186
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F. of subjects for this age range: 112
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 62
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 12
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F. of subjects incapable of giving consent
    Some patients entering this study will be unable to give consent personally due to Mental Disability or Cognitive Impairment. In these cases consent/assent will be sought from patient and/or parent(s)/legal representative.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state40
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 89
    F.4.2.2In the whole clinical trial 186
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Following study completion, all patients will be invited to continue to receive GWP42003-P in an open label extension (OLE) study (under a separate protocol).
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2016-04-19
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-04-09
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