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    Summary
    EudraCT Number:2015-002154-12
    Sponsor's Protocol Code Number:GWEP1521
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2016-07-15
    Trial 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 ConcernedSpain - AEMPS
    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
    Estudio aleatorizado doble ciego controlado con placebo para investigar la eficacia y la seguridad del cannabidiol (GWP42003-P, CBD) como terapia complementaria en pacientes con complejo de esclerosis tuberosa que sufren crisis epilépticas inadecuadamente controladas.
    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.
    Estudio de cannabidiol (GWP42003-P, CBD) en pacientes con complejo de esclerosis tuberosa que sufren crisis epilépticas.
    A.4.1Sponsor's protocol code numberGWEP1521
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02544763
    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 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 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
    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)
    Complejo de esclerosis tuberosa (CET o TSC, por su sigla en inglés)
    E.1.1.1Medical condition in easily understood language
    Genetic disease of benign tumour growth with multiple symptoms including seizures
    Enfermedad genética con cremiento tumoral benigno de múltiples síntomas incluidas las crisis epilépticas.
    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 19.0
    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.
    Fase ciega:
    Evaluar la eficacia de GWP42003-P como terapia complementaria para reducir la frecuencia de las crisis convulsivas comparado con el placebo en pacientes con TSC.
    Fase de extensión abierta:
    Evaluar mediante el perfil de acontecimientos adversos (AAs) la seguridad y la tolerabilidad a largo plazo de GWP42003-P como terapia complementaria en niños y adultos con TSC que sufren crisis epilépticas inadecuadamente controladas.
    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.
    • To determine the pharmacokinetics (PK) following single and multiple doses of GWP42003-P.

    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.
    Fase ciega:
    • Evaluar el efecto de GWP42003-P en comparación con el placebo en las medidas antiepilépticas.
    • Evaluar la seguridad y tolerabilidad de GWP42003-P en comparación con el placebo.
    • Determinar la farmacocinética (PK) tras dosis únicas y múltiples de GWP42003-P.

    Estudio abierto de extensión:
    • Evaluar los efectos a largo plazo de GWP42003-P, como terapia complementaria, sobre medidas antiepilépticas.
    • Evaluar la seguridad y la tolerabilidad a largo plazo de 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 history of epilepsy, with compatible
    electroencephalogram (EEG) and clinical history.
    • Clinical diagnosis of TSC according to criteria agreed by the
    2012 International Tuberous Sclerosis Complex Consensus
    Conference.
    • 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).
    • El paciente debe ser hombre o mujer de entre uno a 65 años de edad, inclusive.
    • El paciente y/o sus padres/representante legal deben estar dispuestos y ser capaces de dar su consentimiento/asentimiento informado para participar en el estudio.
    • El paciente debe tener un historial de epilepsia bien documentado, con electroencefalograma (EEG) e historial clínico compatibles.
    • Diagnóstico clínico de TSC según los criterios acordados por la Conferencia Internacional para el Consenso del Complejo de Esclerosis Tuberosa de 2012.
    • Todos los medicamentos o las intervenciones para la epilepsia (incluyendo la dieta cetogénica y cualquier dispositivo de neuroestimulación para la epilepsia) deben haber sido estables durante un mes antes de la selección y el paciente está dispuesto a mantener un régimen estable durante todo el estudio.
    Al final del periodo basal los pacientes también deben cumplir el siguiente criterio:
    • El paciente debe haber cumplimentado como mínimo el 90% de las llamadas al IVRS durante los primeros 28 días del periodo basal (un mínimo de 25 llamadas realizadas).
    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 is taking felbamate, and they have been taking it for
    less than one year prior to screening.
    • Patient is taking an oral (mTOR) inhibitor.
    • Active suicidal plan/intent in the past six months, or a history
    of suicide attempt in the last two years, or more than one
    lifetime suicide attempt.
    • 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 within the 12 weeks prior to the
    screening visit.
    • El paciente tiene historial de seudocrisis.
    • El paciente ha recibido anestesia general en las cuatro semanas antes de la visita de selección o aleatorización.
    • El paciente ha sido quirúrgicamente intervenido debido a la epilepsia en los seis meses antes de la visita de selección.
    • El paciente está siendo considerado para una intervención quirúrgica asociada con la epilepsia o para cualquier procedimiento en el que se le administre anestesia general durante la fase ciega del estudio.
    • Un paciente que esté tomando felbamato y que lo haya estado tomando durante menos de un año antes de la visita de selección.
    • Un paciente que esté tomando un inhibidor oral (mTOR).
    • El paciente ha tenido algún plan/intención suicida en los últimos seis meses o tiene historial de comportamiento suicida en los últimos dos años o más de un intento suicida durante su vida.
    • El paciente utiliza en la actualidad o ha utilizado en el pasado, cannabis recreativo, cannabis medicinal o medicamentos a base de cannabinoides, en los tres meses antes del estudio y no está dispuestos a abstenerse de utilizarlos durante el estudio.
    • El paciente tiene crecimiento tumoral que, según la opinión del investigador, podría afectar la variable primaria.
    • Una paciente con potencial para procrear o un paciente cuya pareja tenga potencial para procrear, a menos que estén dispuestos a asegurar que tanto él como su pareja utilizarán anticonceptivos altamente eficaces (p. ej., anticonceptivos hormonales, dispositivos intrauterinos, sistemas intrauterinos liberadores de hormonas, oclusión tubárica bilateral, pareja vasectomizada, abstinencia sexual) durante el estudio y tres meses después de finalizar el estudio.
    • Una paciente embarazada (prueba de embarazo positiva), que esté dando pecho o planificando un embarazo durante el transcurso del estudio y tres meses después del estudio.
    • Pacientes que han recibido un MI en los doce meses anteriores a la visita de selección.
    E.5 End points
    E.5.1Primary end point(s)
    Blinded Phase:
    The primary endpoint is the percentage change from baseline in
    number of TSC-associated seizures (average per 28 days) 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.
    Fase ciega:
    La variable principal es el porcentaje de cambio respecto al basal en el número de crisis (promedio de 28 días) durante el periodo de tratamiento (mantenimiento y ajuste de dosis) en pacientes que tomen GWP42003-P comparado con el placebo.

    Fase de extensión abierta:
    Se evaluará la seguridad de GWP42003-P evaluando la incidencia, el tipo y la severidad de los AAs.
    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.
    Visita inicial (visita 2 a la visita 3) y los últimos 28 días del período evaluable.
    E.5.2Secondary end point(s)
    The following endpoints will be compared between treatment groups over the 16-week, double-blind treatment period (all changes relative to baseline):
    -Change in number of total seizures (percentage change from baseline and responder analyses).
    -The plasma concentration/time curve of CBD and its major metabolites
    Safety and Tolerability:
    -Clinical laboratory parameters.
    -ECG.
    -Physical examination parameters (including height and weight).
    -Vital signs.
    -C-SSRS (19+ years) or C-SSRS Children’s (6–18 years) score, where applicable.
    -Number of inpatient hospitalizations due to epilepsy.
    -Abuse liability.
    -Effects on menstruation cycles (in females).

    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.
    Los siguientes objetivos serán comparados entre los grupos de tratamiento de más de 16 semanas, periodo de tratamiento doble ciego (todos los cambios respecto al valor basal):
    -Cambio en el número de crisis epilepticas totales (Variación porcentual respecto al valor basal y análisis de respuesta).
    -La Curva de concentración plasmática / tiempo del CDB y sus principales metabolitos.
    Seguridad y tolerabilidad:
    • Parámetros clínicos de laboratorio.
    • ECG
    • Parámetros del examen físico (incluyendo la altura y el peso).
    • Signos vitales.
    • C-SSRS (19 años y más mayores) o C-SSRS, cuando sea aplicable.
    • Número de hospitalizaciones debidas a la epilepsia.
    • Propensión al abuso del uso del fármaco.
    • Efectos sobre los ciclos de menstruación (en las pacientes).

    Fase de extensión abierta
    Incluirá los mismos objetivos secundarios que en el estudio principal, con la adición de variación porcentual de las crisis epilepticas asociadas a TSC y la eliminación del análisis farmacocinético.
    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.
    Fase ciega (crisis epilepticas) - visita inicial (visita 2 a la visita 3) y los últimos 28 días del período evaluable.
    Fase ciega (otro eficacia) - Visitas 3, 4, 5, 6, 7, 8, 9 y 10.
    Fase ciega (seguridad) - Visitas 3, 4, 5, 6, 7, 8, 9 y 10.
    Fase de extension abierta - TODA visita comienza en 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 concerned4
    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
    France
    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 years4
    E.8.9.1In the Member State concerned months2
    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 months6
    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: 84
    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: 40
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 28
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 108
    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 96
    F.4.2.2In the whole clinical trial 192
    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)
    Patients who complete the blinded phase of this trial will be given the opportunity to enter into the Open Label Extension (OLE) phase, where they will be guaranteed to receive GWP42003-P. The OLE phase will continue until market authorisation is granted for GWP42003-P in the treatment of Tuberous Sclerosis Complex (TSC).
    A aquellos pacientes que completen la fase ciega de este ensayo se les dará la oportunidad de entrar en la fase de extensión abierta (OLE por sus siglas en ingles), donde se les garantiza recibir GWP42003-P. La fase OLE continuará hasta que se conceda la autorización de comercialización para GWP42003-P en el tratamiento del complejo de esclerosis tuberosa (TSC).
    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 Decision2016-09-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-09-12
    P. End of Trial
    P.End of Trial StatusOngoing
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