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    Summary
    EudraCT Number:2014-001834-27
    Sponsor's Protocol Code Number:GWEP1415
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-07-20
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedFrance - ANSM
    A.2EudraCT number2014-001834-27
    A.3Full title of the trial
    An open label extension study to investigate the safety of cannabidiol (GWP42003-P; CBD) in children and adults with inadequately controlled Dravet or Lennox-Gastaut Syndromes
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An open label study of the safety of cannabidiol (GWP42003-P) in children and adults with Dravet or Lennox-Gastaut Syndromes
    A.4.1Sponsor's protocol code numberGWEP1415
    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
    B.5.6E-mailinfo@gwpharm.com
    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.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Dravet Syndrome (DS) or Lennox-Gastaut Syndrome (LGS)
    E.1.1.1Medical condition in easily understood language
    Epilepsy
    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 18.0
    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 evaluate the long term safety and tolerability of GWP42003-P, as adjunctive treatment, in children and adults with inadequately controlled Dravet Syndrome or Lennox-Gastaut Syndrome.
    E.2.2Secondary objectives of the trial
    All Patients:
    To evaluate the effect of GWP42003-P, as adjunctive treatment, on:
    • Quality of life.
    • Adaptive behavior.
    • Need for hospitalizations due to epilepsy.
    • Usage of rescue medication.
    • Maintenance of seizure frequency reduction and freedom from seizures during the open label extension (OLE) study.
    • Frequency of subtypes of seizures.
    • Change in duration of subtypes of seizures.
    • Number of episodes of status epilepticus.
    • Cognitive function, Growth and development, Menstruation cycles (in females).
    • Signals indicating drug abuse liability of GWP42003-P.
    DS Patients Only:
    • Total convulsive and non-convulsive seizure frequency.
    • Number of patients convulsive seizure free.
    • Responder rate (defined in terms of percentage reduction in total convulsive seizure frequency).
    LGS Patients Only:
    • Drop and Non- Drop seizure frequency.
    • Number of patients drop seizure free.
    • Responder rate (defined in terms of percentage reduction in drop seizure frequency).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Patient has completed all scheduled visits in the treatment phase of their Core Study.
    • Patient and/or parent(s)/legal representative must be willing and able to give informed consent/assent 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 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.
    E.4Principal exclusion criteria
    • 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, not including IMP received during the Core study.
    • 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.
    • Any history of suicidal behavior or any suicidal ideation of type four or five on the C-SSRS at Visit 1.
    • Patient has been part of a clinical trial involving an IMP during the inter-study period.
    • Patient has previously been enrolled and dosed in this study.
    • 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, lactating or planning pregnancy during the course of the study and for three months thereafter.
    • 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.
    • 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.
    • Patient has significantly impaired hepatic function at the ‘End of Treatment’ visit of their Core Study or at Visit 1 if re-assessed (alanine aminotransferase [ALT] >5 × upper limit of normal [ULN] and total bilirubin [TBL] >2 × ULN) OR ALT or aspartate aminotransferase (AST) >3 × ULN and (TBL >2 × ULN or international normalized ratio [INR] >1.5).
    E.5 End points
    E.5.1Primary end point(s)
    The safety of GWP42003-P will be assessed by the adverse event (AE) profile and by evaluating changes in the following, relative to the pre-randomization baseline of the Core Study:
    • Vital signs.
    • Physical examination (including height and body weight).
    • 12-lead electrocardiogram (ECG).
    • Columbia-Suicide Severity Rating Scale (C-SSRS) score.
    • Cannabis Withdrawal Scale (CWS) or Pediatric Cannabinoid Withdrawal Scale (PCWS) score, as appropriate.
    • Clinical Laboratory parameters.
    The CWS will be administered to patients aged 18 and older while the PCWS will be administered to patients aged 4–17 (inclusive).
    The Children’s C-SSRS will be used for patients aged 6–18 (inclusive) while the C-SSRS will be used for patients aged 19 and older.
    E.5.1.1Timepoint(s) of evaluation of this end point
    From Visit 1 to End of Treatment Visit:
    Adverse event (AE) profile, Vital signs, Physical examination (including height and body weight), 12-lead electrocardiogram (ECG), laboratory parameters.
    -At Visit 1, from Visit 5 to End of Treatment Visit: Columbia-Suicide Severity Rating Scale (C-SSRS) score.
    -At End of Taper visit: Cannabis Withdrawal Scale (CWS) and Pediatric Cannabinoid Withdrawal (PCWS) scores.
    E.5.2Secondary end point(s)
    All Patients:
    • Change in quality of life as measured with Quality of Life in Childhood Epilepsy (QOLCE) if 18 years of age or younger, or Quality of Life in Epilepsy (QOLIE) if 19 years of age or older, relative to the pre-randomization baseline of the Core Study, if assessed during the Core Study.
    • Change in Subject/Caregiver Global Impression of Change (S/CGIC), relative to the pre-randomization baseline of the Core Study.
    • Change in adaptive behavior as measured with the Vineland Adaptive Behavior Scales, Second Edition (Vineland-II), relative to the pre-randomization baseline of the Core Study, if assessed during the Core Study.
    • Change in the number of inpatient epilepsy-related hospitalizations (number of hospitalizations due to epilepsy in each 28-day period), relative to the pre-randomization baseline of the Core Study.
    • Change in the use of rescue medication (number of days used in each 28-day period), relative to the pre-randomization baseline of the Core Study.
    • Maintenance of seizure frequency reduction and freedom from seizures during the OLE study.
    • Percentage change in the frequencies of subtypes of seizures, relative to the pre-randomization baseline of the Core Study.
    • Changes in duration of seizure subtypes as assessed by the Subject/Caregiver Global Impression of Change in Seizure Duration (S/CGICSD), relative to the pre-randomization baseline of the Core Study.
    • Change in the number of episodes of status epilepticus, relative to the pre-randomization baseline of the Core Study.
    • Change in cognitive function as measured with a cognitive assessment battery, relative to the pre-randomization baseline of the Core Study, if assessed during the Core Study.
    • Change in growth and development for patients less than 18 years of age by measurement of height, weight, insulin-like growth factor-1 (IGF-1) levels and Tanner Staging (for patients aged 12–17 [inclusive]), relative to the pre-randomization baseline of the Core Study.
    • Effects on menstruation cycles (in females).
    • Drug abuse liability, as measured by AEs of abuse potential, drug accountability and Study Medication Use and Behavior Survey in patients aged 12 and older.
    DS Patients Only:
    • Percentage change in total convulsive seizure frequency, relative to the pre-randomization baseline of the Core Study.
    • Percentage change in total non-convulsive seizure frequency, relative to the pre-randomization baseline of the Core Study.
    • Number of patients considered treatment responders, defined as those with a ≥25%, ≥50%, ≥75%, or 100% reduction in convulsive seizures, relative to the pre-randomization baseline of the Core Study.
    • Number of patients experiencing a >25% worsening, −25 to +25% no change, 25–50% improvement, 50–75% improvement or >75% improvement in convulsive seizures, relative to the pre-randomization baseline of the Core Study.
    LGS Patients Only:
    • Percentage change in the number of drop seizures, relative to the pre-randomization baseline of the Core Study.
    • Percentage change in the number of non-drop seizures, relative to the pre-randomization baseline of the Core Study.
    • Number of patients considered treatment responders, defined as those with a ≥25%, ≥50%, ≥75%, or 100% reduction in drop seizures, relative to the pre-randomization baseline of the Core Study.
    • Number of patients experiencing a >25% worsening, −25 to +25% no change, 25–50% improvement, 50–75% improvement or >75% improvement in drop seizures, relative to the pre-randomization baseline of the Core Study.
    E.5.2.1Timepoint(s) of evaluation of this end point
    All Patients:
    -At Visit 1, from Visit 5 to End of Treatment Visit: QOL, S/CGIC, S/CGICSD, Vineland.
    -At Visits 1 and End of Treatment: Cognitive function, IGF-1 levels, Tanner Staging, menstruations cycles.
    - From Visit 1 to End of Treatment Visit:
    Number of epilepsy-related hospitalizations, rescue medication use, maintenance of seizure frequency reduction, seizures freedom, seizures sub-types, number of status epilepticus.
    Dravet Syndrome only: Number of convulsive and non-convulsive seizure, treatment responders and patients experiencing a worsening, no change or improvement in convulsive seizures.
    Lennox-Gastaut Syndrome only: Number of drop and non-drop seizures, treatment responders, patients experiencing a worsening, no change or improvement in drop seizures.
    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 No
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    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.7.1.3.1Other 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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA29
    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
    France
    Israel
    Netherlands
    Poland
    Spain
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    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
    LVLS
    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 months19
    E.8.9.1In the Member State concerned days21
    E.8.9.2In all countries concerned by the trial years2
    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: 420
    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.1.1.5.1Number of subjects for this age range: 210
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 210
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 110
    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.3.3.6.1Details 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 state34
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 140
    F.4.2.2In the whole clinical trial 530
    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)
    The end of treatment will take place once market authorization is granted or after a maximum of 1 year's treatment, whichever comes first. There is no guarantee that the patient can continue to take GWP42003-P. The patient study doctor will talk to parents/patients about possible options.
    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 Decision2015-06-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-09-03
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-09-24
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