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    Summary
    EudraCT Number:2014-002940-42
    Sponsor's Protocol Code Number:GWEP1414
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-12-16
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2014-002940-42
    A.3Full title of the trial
    A randomized, double-blind, placebo-controlled study to investigate the efficacy and safety of cannabidiol (GWP42003-P; CBD) as adjunctive treatment for seizures associated with Lennox-Gastaut syndrome in children and adults.
    Estudio aleatorizado doble ciego controlado con placebo para investigar la eficacia y la seguridad del cannabidiol (GWP42003-P; CBD) como tratamiento complementario para convulsiones asociadas en el Síndrome de Lennox-Gastaut en niños y adultos.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P; CBD) as Adjunctive Treatment for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults
    Estudio para investigar la eficacia y seguridad del cannabidiol (GWP42003-P; CBD) como tratamiento complementario para convulsiones asociadas en el Síndrome de Lennox-Gastaut en niños y adultos.
    A.4.1Sponsor's protocol code numberGWEP1414
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02224560
    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 AddressPorton Down Science Park
    B.5.3.2Town/ citySalisbury
    B.5.3.3Post codeSP4 0JQ
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+441980557000
    B.5.5Fax number+441980557111
    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
    Lennox-Gastaut syndrome (LGS)
    Síndrome de Lennox-Gastaut (LGS)
    E.1.1.1Medical condition in easily understood language
    Epilepsy
    Epilepsia
    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 17.1
    E.1.2Level PT
    E.1.2Classification code 10048816
    E.1.2Term Lennox-Gastaut syndrome
    E.1.2System Organ Class 10029205 - Nervous system 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 efficacy of GWP42003-P as adjunctive treatment in reducing the number of drop seizures compared with placebo, in patients with LGS.
    Drop seizure is defined as an attack or spell (atonic, tonic, tonic-clonic or myoclonic [if severe enough to cause a fall]) involving the entire body, trunk or head that led or could have led to a fall, injury, slumping in a chair or hitting the patient?s head on a surface.
    Evaluar la eficacia de GWP42003 P como tratamiento complementario para reducir el número de convulsiones con episodio de caída, comparado con el placebo, en pacientes con SLG.
    Una convulsión con episodio de caída se define como un ataque o un episodio (atónico, tónico, tonicoclónico o mioclónico) que implica todo el cuerpo, el tronco o la cabeza, que dio lugar o podría haber dado lugar a una caída, lesión, desplomarse en una silla o que el paciente se golpee la cabeza en una superficie.
    E.2.2Secondary objectives of the trial
    To assess the following in LGS patients taking GWP42003-P as adjunctive treatment, when compared with placebo:
    Key:
    ? Number of patients drop seizure-free.
    ? Responder rate, in terms of reduction in drop seizures.
    ? Reduction in the number of non-drop seizures.
    ? Frequency of sub-types of seizures.
    ? Safety and tolerability of GWP42003-P through monitoring of:
    ? Adverse events (AEs).
    ? Suicidal ideation.
    ? Abuse liability.
    ? Cannabis withdrawal effects.
    ? Clinical laboratory tests.
    ? Vital signs.
    Other:
    ? Number of episodes of status epilepticus.
    ? Need for hospitalization due to epilepsy.
    ? Change in duration of subtypes of seizures.
    ? Sleep disruption and daytime sleepiness.
    ? Quality of life.
    ? Adaptive behavior.
    ? Cognitive function.
    Evaluar los efectos del GWP42003-P, como tratamiento complementario, en pacientes con SLG, comparado con el placebo, sobre:
    Principales:
    ?El número de pacientes sin convulsiones con episodio de caída.
    ?La tasa de respondedores, definida como la reducción en convulsiones con episodio de caída.
    ?La reducción en el número de convulsiones sin episodio de caída.
    ?La frecuencia de otros tipos de convulsiones.
    ?La seguridad y tolerabilidad a GWP42003-P mediante la monitorización de:
    ?Acontecimientos adversos (AAs).
    ?Ideación suicida.
    ?Propensión al abuso del uso del fármaco.
    ?Efectos de la abstinencia al cannabis.
    ?Pruebas clínicas de laboratorio.
    ?Signos vitales.
    Otros:
    ?Número de episodios de estado epiléptico.
    ?Necesidad de hospitalización debida a la epilepsia.
    ?Cambio en la duración de otros tipos de convulsiones
    ?Alteración del sueño y somnolencia diurna.
    ?Calidad de vida.
    ?Comportamiento adaptivo.
    ?Función cognitiva
    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 55 years (inclusive).
    ? Patient must have a documented history of LGS. This includes written documentation of having met electroencephalogram (EEG) diagnostic criteria during the patient?s history and evidence of at least one type of generalized seizure, including drop seizures (atonic, tonic, tonic-clonic, clonic or myoclonic) for at least six months.
    ? Patients who have a history of slow (<2.5 Hz) spike-and-wave pattern in an EEG prior to the enrollment into the baseline period.
    ? Patients must have at least two drop seizures each week during the 28-day baseline period.
    ? Patients should be refractory; that is having documented failures on more than one antiepileptic drug (AED).
    ? Patient must be 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 vagus nerve stimulation [VNS]) must have been stable for four weeks prior to screening and patient is willing to maintain a stable regimen throughout the study. The ketogenic diet and VNS treatments are not accounted 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.
    ?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 y su cuidador deben estar dispuestos y ser capaces de (según la opinión del investigador) cumplir con todos los requerimientos del estudio.
    ?El paciente debe ser hombre o mujer de entre dos a 55 años de edad (inclusive).
    ?El paciente debe tener un historial documentado de SLG. Esto debe incluir documentación escrita de cumplir criterios de diagnóstico de electroencefalograma (EEG) durante el historial del paciente y evidencia de al menos un tipo generalizado de convulsión, incluyendo convulsiones con episodio de caída (atónica, tónica, tonicoclónica, clónica o mioclónica) durante un periodo mínimo de seis meses.
    ?Pacientes con un historial de patrón punta onda lenta (<2,5 Hz) en un EEG antes de su participación en el periodo selección.
    ?Los pacientes deben sufrir al menos dos convulsiones con episodio de caída cada semana durante el periodo de selección de 28 días.
    ?Los pacientes deberían ser refractarios; es decir, deberían tener fracasos documentados con más de un fármaco antiepiléptico ( FAE).
    ?Los pacientes deben estar tomando una dosis estable de uno o más FAEs durante al menos cuatro semanas antes de la visita de selección.
    ?Todos los medicamentos o las intervenciones para la epilepsia (incluyendo la dieta cetogénica o el tratamiento de estimulación del nervio vago [ENV]) deben haber sido estables durante cuatro semanas antes de la selección y el paciente y el cuidador están dispuestos a mantener un régimen estable durante todo el estudio. La dieta cetogénica y los tratamientos ENV no cuentan como medicamentos FAE.
    ?El paciente y/o sus padres/representante legal deben estar dispuestos a permitir que se notifique de su participación en el estudio a su médico de atención primaria y especialista.
    E.4Principal exclusion criteria
    ? Etiology of patient's seizures is a progressive neurologic disease. Patients with tuberous sclerosis will not be excluded from study participation, unless there is a progressive tumor.
    ? Patient has had an anoxic episode requiring resuscitation within six months of screening.
    ? 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 investigators 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 any known or suspected hypersensitivity to cannabinoids or any of the excipients of the IMP, such as 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 effective contraception, for example, oral contraception, double barrier, intra-uterine device, during the study and for three months thereafter (however a male condom should not be used in conjunction with a female condom).
    ? Female patient is pregnant, 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.
    ? 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) (Alanine aminotransferase [ALT] >5 x upper limit of normal [ULN] or total bilirubin [TBL] >2 x ULN) OR the ALT or Aspartate aminotransferase (AST) >3 x ULN and (TBL >2 x ULN or international normalized ratio [INR] >1.5). This criterion can only be confirmed once the laboratory results are available; patients randomized into the study who are later found to meet this criterion should be withdrawn from 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.
    ? Any history of suicidal behavior or any suicidal ideation of type four or five on the C-SSRS in the last month or at screening.
    ? 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.
    ? Patient is taking more than four concurrent AEDs.
    ? Patient has taken corticotropins in the six months prior to screening.
    ? Patient is currently taking long-term systemic steroids (excluding inhaled medication for asthma treatment) or any other daily medication known to exacerbate epilepsy. An exception will be made of prophylactic medication, for example, idiopathic nephrotic syndrome or asthma.
    ? Patient is taking felbamate, and they have been taking it for less than one year prior to screening.
    ?La etiología de las convulsiones del paciente es una enfermedad neurológica progresiva. Los pacientes que sufran esclerosis tuberosa no quedarán excluidos de participar en el estudio, a menos que presenten un tumor progresivo.
    ?El paciente ha sufrido un episodio anóxico que haya requerido resucitación en un periodo de seis meses de la visita de selección.
    ?El paciente sufre alguna afección médica inestable clínicamente significativa, exceptuando epilepsia.
    ?El paciente ha sufrido síntomas clínicamente relevantes o una enfermedad clínicamente significativa en las cuatro semanas antes de la visita de selección o aleatorización, exceptuando epilepsia.
    ?Si, según la opinión del investigador, durante la visita de selección o aleatorización el paciente tiene valores de laboratorio significativamente anormales.
    ?El paciente sufre alguna anormalidad clínica significativa según se haya medido en el ECG durante la visita de selección o aleatorización.
    ?El paciente sufre alguna afección cardiovascular concurrente que, en la opinión del investigador, podría interferir con la capacidad para evaluar el ECG del paciente.
    ?El paciente tiene historial o presenta signos de abuso de alcohol u otras sustancias en los últimos dos años antes del estudio o consume diariamente más de cinco bebidas que contienen alcohol.
    ?El paciente utiliza en la actualidad o ha utilizado en el pasado, cannabis recreativo, cannabis medicinal o medicamentos sintéticos a base de cannabinoides (incluyendo Sativex®) tres meses antes de su participación en el estudio.
    ?El paciente no está dispuesto a abstenerse de utilizar cannabis recreativo o medicinal o medicamentos a base de cannabinoides sintéticos (incluyendo Sativex®) durante el estudio.
    ?El paciente tiene un historial de síntomas (p. ej., vértigo, mareos, visión borrosa, palpitaciones, debilidad, síncope) relacionados con una caída en la presión arterial debida a cambios posturales.
    ?El paciente sufre o se sospecha que sufre alguna hipersensibilidad conocida a los cannabinoides o a alguno de los excipientes del MI, por ejemplo, al aceite de sésamo.
    ?Una paciente con potencial para procrear o un paciente cuya pareja tenga potencial para procrear; a menos que estén dispuestos a asegurar que tanto ella como su pareja utilizarán métodos anticonceptivos eficaces, por ejemplo anticonceptivos orales, de doble barrera, dispositivos intrauterinos, durante el estudio y tres meses después del estudio (no obstante el condón masculino no debería utilizarse en conjunción con el condón femenino).
    ?Una paciente embarazada, que esté dando pecho o planificando un embarazo durante el transcurso del estudio y tres meses después del estudio.
    ?El paciente ha participado en algún ensayo clínico asociado con algún otro MI en los seis meses anteriores a este estudio.
    ?Cualquier otra enfermedad o trastorno significativo que, según la opinión del investigador, podría poner en peligro al paciente al participar en el estudio, podría influir en el resultado del estudio o afectar la capacidad del paciente para participar en el estudio.
    ?El paciente sufre disfunción hepática importante en la visita de selección (visita 1) o en la visita de aleatorización (visita 2) (alanina aminotransferasa [ALT] >5 xlímite superior normal [ULN] o bilirrubina total [TBL] >2 x ULN) O ALT o aspartato aminotransferasa (AST) >3 x ULN y (TBL >2 x ULN o razón internacional normalizada [INR] >1.5). Este criterio solamente puede confirmarse una vez de que estén disponibles los resultados de laboratorio; los pacientes que participen en el estudio y que posteriormente se descubra que cumplen este criterio deberían ser retirados del estudio.
    ?Después de un examen médico el paciente tiene alguna anormalidad que, según la opinión del investigador, pondría en riesgo la participación del paciente en el estudio de forma segura.
    ?Historial de comportamiento suicida o cualquier ideación suicida de tipo cuatro o cinco en la escala C-SSRS, durante el último mes o en la visita de selección.
    ?El paciente es reacio a abstenerse de donar sangre durante el estudio.
    ?El paciente tiene pensado viajar fuera de su país de residencia durante el estudio.
    ?El paciente ha estado anteriormente aleatorizado en este estudio.
    ?El paciente toma simultáneamente más de cuatro FAEs.
    ?El paciente ha tomado corticotropinas en los seis meses anteriores a la visita de selección.
    ?El paciente está tomando esteroides sistémicos a largo plazo (excluyendo medicamentos inhalados para el tratamiento del asma) o cualquier otro medicamento diario que se sepa que exacerbara la epilepsia. Se hará una excepción con los medicamentos profilácticos, por ejemplo, el síndrome nefrótico idiopático o el asma.
    ?El paciente está tomando felbamato y lo lleva tomando menos de un año antes de la visita de selección.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the mean percentage change from baseline in number of drop seizures (average per week) during the maintenance period (Day 15 to the end of the evaluable period) in patients taking GWP42003-P compared with placebo.
    La variable principal es el porcentaje medio de cambio respecto al basal en el número de convulsiones con episodio de caída (promedio semanal) durante el periodo de mantenimiento (día 15 hasta el final del periodo de evaluación) en pacientes que hayan tomado GWP42003-P, comparado con el placebo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The baseline period is Screening (V1) - Randomisation (V2).
    Mean percentage change from baseline in number of drop seizures (average per week) from end of titration (V3) to End of Treatment (V8) in patients taking GWP42003-P compared with placebo.
    El periodo de basal es selección (V1)- Aleatorización (V2). Porcentaje medio de cambio respecto al basal en el número de convulsiones con episodio de caída (promedio semanal) desde el fin del periodo de ajuste de dosis (V3) hasta el fin de tratamiento (V8) en pacientes que toman GWP42003-P respecto placebo.
    E.5.2Secondary end point(s)
    The following endpoints will be compared between the three treatment groups over the 12-week, double-blind maintenance period:
    ? Percentage change from baseline in number of drop seizures (average per week) during the Weeks 1?4, 5?8 and 9?12.
    ? Number of patients considered treatment responders, defined as those with a ?25%, ?50%, ?75%, or 100% reduction in drop seizures from baseline. Summaries will be presented overall and four-weekly.
    ? Number of patients experiencing a >25% worsening, ?25 to +25% no change, 25?50% improvement, 50?75% improvement or >75% improvement in drop seizures from baseline.
    ? Percentage change from baseline in number of non-drop seizures (average per week).
    ? Percentage change from baseline in the frequencies of sub-types of seizures (average per week).
    ?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 number of episodes of status epilepticus.
    ? Changes from baseline in number of inpatient hospitalizations due to epilepsy.
    ? Changes from baseline in quality of life as assessed by the Quality of Life in Childhood Epilepsy (QOLCE), for patients aged between two and 18 years of age, or Quality of Life in Epilepsy, version 2 (QOLIE-31-P) for patients aged 19 years and older.
    ? Changes from baseline in the Caregiver Global Impression of Change (CGIC) score.
    ? Change from baseline in adaptive behavior as measured with the Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) score.
    ? Change in cognitive function as measured with the Cognitive Assessment Battery.
    ? 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.
    The safety profile of GWP42003-P compared with placebo will also be assessed at each Dose Level by measuring:
    ? AEs.
    ? Clinical laboratory parameters.
    ? Columbia-Suicide Severity Rating Scale (C-SSRS) score.
    ? Vital signs.
    ? Physical examination parameters.
    ? 12-lead electrocardiogram (ECG).
    ? Cannabis Withdrawal Scale (CWS) or Pediatric Cannabinoid Withdrawal Scale (PCWS) score.
    ? Abuse liability.
    Durante el periodo de mantenimiento doble ciego de 12 semanas, se compararán las siguientes variables entre los tres grupos de tratamiento:
    ?Porcentaje de cambio respecto al basal en el número de convulsiones con episodio de caída (promedio semanal) durante las semanas 1?4, 5?8 y 9?12.
    ?Número de pacientes que se ha considerado que han respondido al tratamiento, definido como aquellos que han experimentado una reducción en convulsiones con episodio de caída de un ?25%, ?50%, ?75% o 100%, respecto al basal. Se presentarán resúmenes generales y cada cuatro semanas.
    ?Número de pacientes que han experimentado un empeoramiento de >25%, ?25 a +25% sin cambio, una mejoría de 25?50%, una mejoría de 50?75% o una mejoría de >75% en la incidencia de convulsiones con episodio de caía, respecto al basal.
    ?Porcentaje de cambio respecto al basal en el número de convulsiones sin episodio de caída (promedio semanal).
    ?Porcentaje de cambio respecto al basal en las frecuencias de otros tipos de convulsiones (promedio semanal).
    ?Cambios respecto al basal en la duración de otros tipos de ataques según se haya valorado en la Impresión General del Cuidador del Cambio en la Duración de los Ataques (CGICSD).
    ?Cambio respecto al basal en el número de episodios de estado epiléptico.
    ?Cambio respecto al basal en el número de hospitalizaciones asociadas con la epilepsia.
    ?Cambio respecto al basal en la calidad de vida según se haya medido con Calidad de vida en niños con epilepsia (QOLCE), para pacientes de entre dos y 18 años de edad o Calidad de vida en pacientes con epilepsia, versión 2 (QOLIE-31-P) para pacientes de 19 años de edad y más mayores.
    ?Cambio respecto al basal en la puntuación de la impresión general del cambio del cuidador (CGIC).
    ?Cambio respecto al basal en la puntuación de conducta adaptiva según se haya medido en las escalas de conducta adaptiva de Vineland, segunda edición (Vineland-II).
    ?Cambio respecto al basal en la función cognitiva según se haya medido con la batería de evaluación cognitiva.
    ?Cambio respecto al basal en la puntuación de la alteración del sueño según se haya medido en una escala de clasificación numérica 0?10 (0?10 NRS).
    ?Cambios respecto al basal en la puntuación en la escala de somnolencia de Epworth (EDSS).
    Para cada nivel de dosis también se evaluará el perfil de seguridad de GWP42003-P comparado con el placebo, midiendo:
    ?AAs.
    ?Parámetros clínicos de laboratorio.
    ?Puntuación en la escala de calificación de la severidad del suicidio de Columbia (C-SSRS).
    ?Signos vitales.
    ?Parámetros del examen físico.
    ?Electrocardiograma de 12 derivaciones (ECG).
    ?Puntuación en la escala de abstinencia al cannabis (CWS) o en la escala pediátrica de abstinencia a los cannabinoides (PCWS), según sea apropiado.
    ?Propensión al abuso del uso del fármaco
    E.5.2.1Timepoint(s) of evaluation of this end point
    The baseline period is Screening (V1) - Randomisation (V2).
    Mean percentage change from baseline will be measured from end of titration (V3) to End of Treatment (V8) in patients taking GWP42003-P compared with placebo.
    El periodo de basal es selección (V1)- Aleatorización (V2). Porcentaje medio de cambio respecto al basal se medirá desde el fin del periodo de ajuste de dosis (V3) hasta el fin de tratamiento (V8) en pacientes tomando GWP42003-P comparado con placebo.
    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 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 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 EEA15
    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
    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
    Completion of the treatment period Visit 10, Day 137.
    Finalización del periodo de tratamiento visita 10, día 137.
    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 months11
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months11
    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: 120
    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: 40
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 40
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 40
    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 LGS Subjects will have some level of cognitive impairment
    Algunos pacientes con LGS tendrán algun nivel de deterioro cognitivo.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 65
    F.4.2.2In the whole clinical trial 120
    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).
    Tras la finalización del estudio, se le ofrecerá a todos los pacientes la posibilidad de continuar recibiendo GWP42003-P en un estudio de extensión abierto (OLE) (bajo otro protocolo).
    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 Decision2015-04-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-01-14
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-05-02
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