Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).


    The EU Clinical Trials Register currently displays   43800   clinical trials with a EudraCT protocol, of which   7272   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2015-002154-12
    Sponsor's Protocol Code Number:GWEP1521
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-01-17
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2015-002154-12
    A.3Full title of the trial
    A double-blind, randomized, placebo-controlled study to investigate the efficacy and safety of cannabidiol (GWP42003-P, CBD) as add-on therapy in patients with tuberous sclerosis complex who experience inadequately-controlled seizures
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of cannabidiol (GWP42003-P, CBD) in patients with tuberous sclerosis complex who experience seizures.
    A.4.1Sponsor's protocol code numberGWEP1521
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02544763
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/136/2017
    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 number00441223266800
    B.5.5Fax number00441223235667
    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 Yes
    D.2.1.1.1Trade name CBD-Oral Solution, is known as Epidiolex, and is the approved name in the USA.
    D.2.1.1.2Name of the Marketing Authorisation holderGreenwich Biosciences, Inc.
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/17/1959
    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
    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
    Tuberous Sclerosis Complex (TSC)
    E.1.1.1Medical condition in easily understood language
    Genetic disease of benign tumour growth with multiple symptoms including seizures
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10045138
    E.1.2Term Tuberous sclerosis
    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
    Blinded Phase:
    To evaluate the efficacy of GWP42003-P as add-on therapy in reducing the frequency of seizures when compared with placebo
    in patients with TSC.
    Open-label Extension:
    To evaluate via the adverse events (AE) profile the long term safety and tolerability of GWP42003-P as add-on therapy in children and adults with TSC who experience inadequately-controlled seizures.
    E.2.2Secondary objectives of the trial
    Blinded Phase:
    • To evaluate the effect of GWP42003-P compared with placebo on antiepileptic measures.
    • To evaluate the safety and tolerability of GWP42003-P compared with placebo.

    Open-label Extension:
    • To evaluate the long term effects of GWP42003-P, as add-on therapy, on antiepileptic measures.
    • To evaluate the long term safety and tolerability of GWP42003-P.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Patient is male or female aged between one and 65 years
    inclusive.
    • Patient and/or parent(s)/legal representative is willing and able
    to give informed consent/assent for participation in the study.
    • Well-documented clinical history of epilepsy which is not completely controlled by their current AEDs.
    • Clinical diagnosis of TSC according to criteria agreed by the 2012 International Tuberous Sclerosis Complex Consensus Conference.
    • Taking one or more AEDs at a dose which has been stable for at least four
    weeks prior to screening.
    • All medications or interventions for epilepsy (including ketogenic diet and any neurostimulation devices for epilepsy) must have been stable for one month prior to screening and the
    patient is willing to maintain a stable regimen throughout the study.
    At the end of the baseline period patients must also meet the
    following criterion:
    • Completed at least 90% of calls to IVRS during the first
    28 days of the baseline period (a minimum of 25 completed calls).
    E.4Principal exclusion criteria
    • Patient has a history of pseudo-seizures.
    • Patient has undergone general anesthetic in the four weeks prior to screening or randomization.
    • Patient has undergone surgery for epilepsy in the six months prior to screening.
    • Patient is being considered for epilepsy surgery or any procedure involving general anesthesia during the blinded phase of the study.
    • Patient has been taking felbamate for less than one year prior to screening.
    • Patient is taking an oral (mTOR) inhibitor.
    • Patient is currently using or has in the past used recreational or medicinal cannabis, or cannabinoid-based medications, within the three months prior to screening and is unwilling to abstain for the duration for the study.
    • Patient has tumor growth which, in the opinion of the Investigator, could affect the primary endpoint.
    • Patient is female and of child bearing potential, or is male whose partner is of child bearing potential, unless willing to
    ensure that they or their partner use a highly effective method of birth control (e.g., hormonal contraceptives, intrauterine devices/hormone-releasing systems, bilateral tubal occlusion, vasectomized partner, sexual abstinence) during the study and for three months thereafter.
    • 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 received an IMP less than 12 weeks prior to the screening visit.
    E.5 End points
    E.5.1Primary end point(s)
    Blinded Phase:
    The primary endpoint is the change in number of TSC-associated seizures* during the treatment period (maintenance and titration) in patients taking GWP42003-P compared with placebo.

    Open-label Extension:
    The safety of GWP42003-P will be evaluated by assessing the incidence, type and severity of AEs.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline (Visit 2 to Visit 3) and the last 28 days of the evaluable period.
    E.5.2Secondary end point(s)
    Blinded Phase:
    The following endpoints will be compared between treatment groups over the 16-week, double-blind
    treatment period (all changes relative to baseline):
    Key:
    • Number of patients considered treatment responders defined as those with a
    ≥ 50% reduction in TSC-associated seizure* frequency.
    • Change in Caregiver Global Impression of Change (CGIC) or Subject Global Impression of Change
    (SGIC) score.
    • Change in total seizures.

    Other:
    • Antiepileptic Efficacy Measures
    • Quality of life
    • Safety and Tolerability

    Open-label Extension
    Will include secondary endpoints as in the core study, with the addition of percentage change in the TSC associated seizures and with removal of PK analysis.


    E.5.2.1Timepoint(s) of evaluation of this end point
    Blinded phase (seizures) - Baseline (Visit 2 to Visit 3) and the last 28 days of the evaluable period.
    Blinded phase (other efficacy) - Visits 3, 4, 5, 6, 7, 8 , 9 and 10.
    Blinded phase (safety) - Visits 3, 4, 5, 6, 7, 8, 9 and 10.
    OLE - ALL visit beginning at B1.
    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 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 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 Yes
    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 trial4
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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
    Australia
    Netherlands
    Poland
    Spain
    United Kingdom
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months5
    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: 94
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 16
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 45
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 33
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 116
    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
    Paediatric patients
    Patients displaying varying degrees of TAND (TSC Associated Neuropsychiatric Disorders)
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 210
    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)
    Normal standard of care will resume. In countries where available, subjects will have access to named patient supply programs, or equivalent.
    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 Decision2017-01-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-09-01
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2021-06-11
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA