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    EudraCT Number:2019-001605-24
    Sponsor's Protocol Code Number:GWND19002
    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:2020-02-21
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2019-001605-24
    A.3Full title of the trial
    An open-label extension trial to investigate the long-term safety of cannabidiol oral solution (GWP42003-P, CBD-OS) in patients with Rett Syndrome
    Un ensayo de extensión abierta para investigar la seguridad a largo plazo de la solución oral de Cannabidiol (GWP42003-P, CBD-OS) en pacientes con síndrome de Rett
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An open label extension study for long-term safety of GWP42003-P in patients with Rett Syndrome
    Estudio de extensión de seguridad a largo plazo de GPW42003-P en pacientes con syndrome de Rett
    A.4.1Sponsor's protocol code numberGWND19002
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorGW Research Ltd
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportGW Research Ltd
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGW Research Ltd
    B.5.2Functional name of contact pointGW Research Ltd Switchboard
    B.5.3 Address:
    B.5.3.1Street AddressSovereign House, Vision Park, Chivers Way
    B.5.3.2Town/ cityHiston, Cambridge
    B.5.3.3Post codeCB24 9BZ
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+441223266800
    B.5.5Fax number+441223235667
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D. name Epidyolex and is the approved name in the EU
    D. of the Marketing Authorisation holderGW Pharma B.V (International)
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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/3/17/1855; EU/3/14/1339
    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 INNCannabidiol
    D.3.9.1CAS number 13956-29-1
    D.3.9.2Current sponsor codeGWP42003-P
    D.3.9.3Other descriptive nameCANNABIDIOL
    D.3.9.4EV Substance CodeSUB26600
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product Yes
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product Yes
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Rett syndrome (RTT) [typical or atypical]
    Síndrome de Rett (RTT) (Típico o atípico)
    E.1.1.1Medical condition in easily understood language
    Rett syndrome (RTT) [typical or atypical]
    Síndrome de Rett (RTT) (Típico o atípico)
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10077709
    E.1.2Term Rett syndrome
    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 of GWP42003-P in patients with RTT
    Evaluar la seguridad a largo plazo GWP42003-P en pacientes con RTT
    E.2.2Secondary objectives of the trial
    •To evaluate the effect of GWP42003-P in measures of disease severity
    o Rett Syndrome Behaviour Questionnaire (RSBQ)
    o Clinical Global Impressions - Improvement (CGI-I)
    o Clinical Global Impressions - Severity (CGI-S)
    o 9-items Motor Behavioral Assessment (MBA-9)
    o Children’s Sleep Habits Questionnaire (CSHQ)

    Exploratory objectives:
    •To evaluate the effect of GWP42003-P on caregiver and patient quality of life (QoL)
    o 36-item Short Form [SF-36] and Child Health Questionnaire Parent Form 50 [CHQ-PF50], respectively
    • To evaluate the effect of GWP42003-P on health utilization
    o Hospital Services Use Questionnaire
    o Caregiver Assessment of Rett Symptoms
    •Evaluar el efecto de GWP42003-P en medidas de gravedad de la enfermedad
    oCuestionario de conducta en síndrome de Rett (RSBQ)
    oImpresiones globales clínicas: mejora (CGI-I)
    oImpresiones globales clínicas: gravedad (CGI-I)
    oEvaluación de conducta motora de 9 puntos (MBA-9)
    oCuestionario sobre los hábitos del sueño de los niños
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    For inclusion in the trial, patients must fulfil all of the following criteria:
    • Patient has completed all scheduled visits of the treatment phase of the RCT, GWND18064, and has transitioned to OLE by the point of RCT follow-up (Visit 11).
    • Patient (if possessing adequate understanding, in the investigator’s opinion) and/or her parent(s)/legal representative is willing and able to give informed consent/assent for participation in the trial.
    • Patient and her caregiver are willing and able (in the investigator’s opinion) to comply with all trial requirements (including the completion of all caregiver assessments by the same caregiver throughout the trial).
    • Patient must have the ability to swallow the investigational medicinal product (IMP) provided as a liquid solution or the ability for the IMP to be delivered via gastrostomy (G) or nasogastric (NG) feeding tube (only G- or NG-tubes made from polyurethane or silicon are allowed).
    • Patient and/or parent(s)/legal representative is willing to allow the responsible authorities to be notified of participation in the trial, if mandated by local law.
    • Patient and/or parent(s)/legal representative is willing to allow the patient’s primary care practitioner (if she has one) and consultant (if she has one) to be notified of participation in the trial if the primary care practitioner/consultant is different from the investigator.
    Para ser incluidos en este studio los pacientes Deben cumplir estos criterios
    •El paciente ha completado todas las visitas programadas de la fase de tratamiento del ensayo aleatorizado controlado, GWND18064 y ha pasado a OLE en el momento del seguimiento de ensayo aleatorizado controlado (Visita 11).
    •El paciente (si posee el entendimiento adecuado en opinión del investigador) y/o sus padres/ representante legal está dispuesto y puede dar el consentimiento/asentimiento informado para la participación en el ensayo.
    •El paciente y su cuidador están dispuestos y pueden (en opinión del investigador) cumplir todos los requisitos del ensayo (incluida la conclusión de todas las evaluaciones del cuidador por parte del mismo cuidador a lo largo del ensayo).
    •El paciente debe tener la capacidad de tragar el medicamento en fase de investigación proporcionado como solución líquida o
    la capacidad para que se le administre el medicamento en fase de investigación por sonda de alimentación de gastrostomía (G) o nasogástrica (NG) (solo están permitidos las sondas G o NG de poliuretano o silicona).
    •El paciente y/o padres/representante legal está dispuesto a permitir que se le notifique a las autoridades responsables la participación en el ensayo, si es obligatorio por la ley local.
    •El paciente y/o padres/representante local está dispuesto a permitir que se notifique al médico de familia del paciente (si tiene uno) y al especialista (si tiene uno) su participación en el ensayo si el médico de familia /especialista es distinto del investigador
    E.4Principal exclusion criteria
    • Patient meets the withdrawal criteria (including clinically significant abnormal laboratory values), in the investigator’s opinion.
    • Patient met during the RCT the criteria for permanent IMP discontinuation (unless in case of an AE, if AE was not considered related with the IMP; patients that met alanine aminotransferase (ALT)/aspartate aminotransferase (AST) elevations discontinuation criteria must be excluded).
    • Patient is of childbearing potential, unless willing to ensure that they or their partner use a highly effective method of birth control (e.g., combined [estrogen and progestogen containing] hormonal contraceptiona associated with inhibition of ovulation [oral, intravaginal or transdermal], progestogen-only hormonal contraceptiona associated with inhibition of ovulation [oral, injectable or implantableb] intrauterine devices/hormone-releasing systemsc, bilateral tubal occlusiona, vasectomized partnera, a, sexual abstinenceb during the trial and for 3 months thereafter.
    • Patient has been previously enrolled and dosed in this trial.
    • Patient is unwilling to abstain from donation of blood during the trial.
    •El paciente cumple los criterios de retirada (incluidos valores anómalos de laboratorio clínicamente significativos) en opinión del investigador.
    •El paciente cumple durante el ensayo aleatorizado controlado el criterio para interrupción permanente del medicamento en fase de investigación ( a menos en el caso de AA, si el AA no se consideraba relacionado con el medicamento en fase de investigación; los pacientes que cumplen los criterios de interrupción por elevaciones de alanina aminotransferasa (ALT)/Aspartato aminotransferasa (AST) deben ser excluidos).
    •La paciente en edad fértil, a menos que esté dispuesta a garantizar que ellas o sus parejas utilizan un método anticonceptivo sumamente eficaz (p. ej. un anticonceptivo hormonal [con estrógenos y progesterona] relacionado con la inhibición de la ovulación [oral, intravaginal o dérmico], anticonceptivo hormonal solo progesterona relacionada con la inhibición de la ovulación [oral, inyectable o implantable ] dispositivos intrauterinos/sistemas liberadoras de hormonas oclusión tubárica bilateral , pareja vasectomizadac, abstinencia sexual durante el ensayo y los 3 meses posteriores al mismo.
    •El paciente se ha inscrito y se le ha administrado el medicamento previamente en este ensayo.
    El paciente no está dispuesto a abstenerse de donar sangre durante el ensayo.
    E.5 End points
    E.5.1Primary end point(s)
    The long-term safety profile of GWP42003-P will be assessed by evaluating changes in the following, relative to the prerandomization baseline of the RCT:
    • AEs
    • Clinical laboratory parameters
    • Vital signs
    • Physical examination procedures
    • 12-lead ECG
    • Effects on menstruation cycles
    • Suicidality
    • Change in growth and development by measurement of height, weight, IGF-1 levels, and Tanner Staging (for patients aged ≥ 7 years or earlier, if clinically indicated by the onset of menarche or other signs of precocious puberty)
    El perfil de seguridad a largo plazo de GWP42003-P será valorado evaluando los cambios en lo siguiente, en relación a la referencia inicial previa a la aleatorización del ensayo aleatorizado controlado:
    •Parámetros de laboratorio clínico
    •Constantes vitales
    •Procedimientos de exploración física
    •Electrocardiograma (ECG) de 12 derivaciones
    •Efectos en la menstruación
    •Tendencias suicidas
    •Cambio en crecimiento y desarrollo midiendo la altura, peso, niveles del factor 1 de crecimiento parecido a la insulina en suero (IGF-1), y escala de Tanner (para pacientes con edades ≥ 7 años o anteriores, si está indicado clínicamente por la aparición de la menarquía u otros signos de pubertad precoz)
    E.5.1.1Timepoint(s) of evaluation of this end point
    at all visits
    En todas las visitas
    E.5.2Secondary end point(s)
    The following will be assessed by evaluating changes relative to the prerandomization baseline of the RCT:
    • RSBQ
    • CGI-I
    • CGI-S
    • MBA-9
    • CSHQ
    The following will be assessed by evaluating changes relative to the prerandomization baseline of the RCT:
    • RSBQ
    • CGI-I
    • CGI-S
    • MBA-9
    • CSHQ
    E.5.2.1Timepoint(s) of evaluation of this end point
    Please refer to Protocol schedule of events table for detailed information
    Por favor consulte la table de eventos del protocol para una información más detallada
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy No
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E. trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled 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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United 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
    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 months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months7
    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: 252
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F. of subjects for this age range: 135
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 105
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 12
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male No
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F. of subjects incapable of giving consent
    Rett Syndrome
    Síndrome de Rett
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state35
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 85
    F.4.2.2In the whole clinical trial 252
    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)
    Subjects will continue on standard of care after the study end.
    Lo pacientes continuarán con el tratamiento estándar después del final del estudio
    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 Decision2020-03-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-02-26
    P. End of Trial
    P.End of Trial StatusOngoing
    The status of studies in GB is no longer updated from 1.1.2021
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