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    EudraCT Number:2018-003370-27
    Sponsor's Protocol Code Number:GWND18064
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2018-12-24
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2018-003370-27
    A.3Full title of the trial
    A randomized, double-blind, placebo-controlled trial to investigate the efficacy and safety of cannabidiol oral solution (GWP42003-P; CBD-OS) in patients with Rett syndrome.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A randomized, double-blind, placebo-controlled trial to investigate the efficacy and safety of cannabidiol oral solution (GWP42003-P; CBD-OS) in patients with Rett syndrome.
    A.4.1Sponsor's protocol code numberGWND18064
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/095/2020
    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 CBD - Oral Solution, is known as Epidyolex, and is the approved name in the USA.
    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 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 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
    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
    Rett syndrome (RTT) [typical or atypical]
    E.1.1.1Medical condition in easily understood language
    Rett syndrome (RTT) [typical or atypical]
    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 efficacy of 15 mg/kg/day GWP42003-P, compared with placebo, at the end of 24 weeks’ treatment in reducing symptom severity in patients with RTT using the Rett Syndrome Behaviour Questionnaire (RSBQ).
    E.2.2Secondary objectives of the trial
    Secondary Objectives:
    - To evaluate the efficacy of 15 mg/kg/day GWP42003-P, compared with placebo, at the end of 24 weeks' treatment in reducing symptom severity in patients with RTT using the CGI-I.
    - To evaluate the efficacy of 5 mg/kg/day GWP42003-P, compared with placebo in: RSBQ, CGI-I.
    - To evaluate the effect of GWP42003-P, compared with placebo, in other measures of disease severity: RSBQ subscales, CGI-S, MBA-9, CSHQ.
    - To evaluate the safety of GWP42003-P, compared with placebo, in patients with RTT.
    Exploratory Objectives:
    - To evaluate the effect of GWP42003-P on caregiver and patient QoL: SF-36 and CHQ-PF50.
    - To evaluate the effect of GWP42003-P on HSUQ.
    - Caregiver assessment of Rett symptoms (symptom diary).
    -To determine the plasma concentrations of CBD and its major metabolites with the aim of evaluating exposure versus efficacy and safety and collecting data for population PK analysis.
    -To evaluate the effect of GWP42003-P on exploratory biomarkers.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    For inclusion in the trial, patients must fulfil ALL of the following
    • Patient is female or male aged 2–18 years (inclusive).
    • Patient must weigh at least 10 kg.
    • Patient (if possessing adequate understanding, in the investigator’s opinion) and/or the patient's parent(s)/legal representative is willing and able to give informed consent/assent for participation in the trial.
    • Patient and the patient's 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 a clinical diagnosis of RTT (typical or atypical), defined according to RettSearch Consortium criteria.
    • Patient must have a confirmed pathogenic genetic mutation of the MECP2 gene.
    • Patient must be post-regression (≥ 6 months since last loss of hand use or verbal language or gross motor regression).
    • Patient must have a disease severity of between 10 and 36, defined according to the CSS.
    • All medications or interventions (including antiepileptic drugs [AEDs] and non-pharmacological interventions - dietary supplements, probiotics, physical therapy, speech therapy, etc.) for RTT-related symptoms must have been stable for 4 weeks prior to screening and the patient/caregiver should be willing to maintain a stable regimen throughout the trial.
    • Patient must have the ability to swallow the IMP provided as a liquid solution, or the ability for IMP to be delivered via gastrostomy (G) or nasogastric (NG) feeding tube (use of G-or NG-tubes is only allowed after discussion with the Medical Monitor to confirm suitability of the tubes being used).
    • 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 the patient has one) and consultant (if the patient has one) to be notified of
    participation in the trial, if the primary care practitioner/consultant is different to the investigator.
    E.4Principal exclusion criteria
    The patient may not enter the trial if ANY of the following apply:
    • Patient meets exclusion criteria for RTT diagnosis (traumatic brain injury, neurometabolic disease, or severe infection that causes neurological problems; grossly abnormal psychomotor development in the first 6 months of life).
    • Patient has clinically significant abnormal laboratory values, in the investigator’s opinion.
    • Patient experiences more than weekly seizures (based on history over the last 2 months prior to screening) i.e., CSS ‘epilepsy/seizure’ score of 4 or 5.
    • Patient is taking more than 2 concurrent AEDs.
    • Any history of suicidal behavior or any suicidal ideation in the last month or at screening.
    • Patient has clinically relevant abnormalities in the ECG measured at screening or randomization (including QT interval, corrected by Bazett's correction formula [QTcB] interval > 450 msec, average of 3 measurements).
    • Patient has any concurrent cardiovascular conditions which will, in the investigator’s opinion, interfere with the ability to assess the patient's ECGs or put the patient at risk because of participation in the trial.
    • Patient’s first or second degree relative has a history of significant ECG abnormalities, in the opinion of the investigator (e.g. premature cardiac arrest, sudden death).
    • Patient has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the IMP (active or placebo), such as sesame oil.
    • Patient has moderately impaired hepatic function at screening, defined as serum ALT or AST > 3 × ULN or total bilirubin [TBL] > 2 × ULN.
    This criterion can only be confirmed once the Visit 1 laboratory results are available.
    • Female 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 contraceptives, contraception a associated with inhibition of ovulation [oral, intravaginal or transdermal], progestogen-only hormonal contraception associated with inhibition of ovulation [oral, injectable or implantable intrauterine devices/hormone-releasing systems, bilateral tubal occlusion, vasectomized partner, sexual abstinence) during the trial and for 3 months thereafter.
    • Female patient is pregnant (positive pregnancy test) or lactating.
    • Male patient is fertile (i.e., after puberty unless permanently sterile by bilateral orchiectomy) and with a partner of childbearing potential unless agree to ensure that they use male contraception (e.g., condom) or remain sexually abstinent during the trial and for 3 months after the last dose.
    • Patient has received an IMP within the 3 months prior to the screening visit.
    • Patient has been taking felbamate for less than 1 year prior to screening.
    • Patient is currently using or has used recreational or medicinal cannabis, cannabinoid-based medications (including Sativex®), or cannabidiol oral solutions (including CBD-OS [GWP42003-P]) within the 3 months prior to screening and is unwilling to abstain for the duration of the trial.
    • Patient has a positive Δ9-tetrahydrocannabinol (THC) test at screening.
    • Patient has any other systemic dysfunction (e.g., gastrointestinal, renal, respiratory) or significant disease or disorder which, in the opinion of the investigator, may either put the patient, other participants, or site staff at risk because of participation in the trial, may influence the result of the trial, or the patient’s ability to participate in the trial.
    • Any abnormalities identified following a physical examination of the patient that, in the opinion of the investigator, would jeopardize the safety of the patient if the patient took part in the trial.
    • Patient has been previously randomized into this trial.
    • Patient has travel outside the country of residence planned during the trial.
    E.5 End points
    E.5.1Primary end point(s)
    RSBQ (total score) for the 15 mg/kg/day GWP42003-P dose level compared with placebo at the end of 24 weeks.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Visit 2, Visit 5, Visit 6, Visit 7, Visit 8, Visit 9
    E.5.2Secondary end point(s)
    CGI-I for the 15 mg/kg/day GWP42003-P dose level compared with placebo at the end of 24 weeks.

    Other Secondary Efficacy Endpoints:
    • To compare 5 mg/kg/day GWP42003-P with placebo using:
    o RSBQ (total score)
    o CGI-I
    • To compare 15 mg/kg/day GWP42003-P and 5 mg/kg/day GWP42003-P with placebo using:
    o RSBQ subscales.
    o CGI-S.
    o MBA-9.
    o CSHQ.

    Exploratory endpoints:
    • Caregiver QoL questionnaire (SF-36).
    • Patient QoL questionnaire (CHQ-PF50)
    • Hospital Services Use Questionnaire.
    • Caregiver assessment of Rett symptoms (symptom diary).
    • Plasma concentrations of CBD and its main metabolites.
    • Blood levels of exploratory biomarkers.
    The safety profile of GWP42003-P compared with placebo will be
    assessed by measuring:
    • AEs.
    • Clinical laboratory parameters.
    • Vital signs.
    • Physical examination procedures.
    • 12-lead electrocardiogram (ECG).
    • Effects on menstruation cycles.
    • Suicidality.
    • Change in growth and development by measurement of
    height, weight, serum insulin-like growth factor-1 (IGF-1)
    levels and Tanner Staging (for patients aged ≥ 7 years, or
    earlier if clinically indicated by onset of menarche or other
    signs of precocious puberty).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Please refer to Protocol schedule of events table for detailed information
    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 Yes
    E.6.6Pharmacokinetic Yes
    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) Yes
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA16
    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 months5
    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 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: 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 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. of subjects incapable of giving consent
    Rett Syndrome
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 126
    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 may be invited to continue to receive GWP42003-P in an open-label extension (OLE) study under a separate protocol
    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 Decision2019-02-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-04-09
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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