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    EudraCT Number:2019-001605-24
    Sponsor's Protocol Code Number:GWND19002
    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:2019-10-23
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    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
    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
    A.4.1Sponsor's protocol code numberGWND19002
    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 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 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
    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 long-term safety of GWP42003-P in patients with 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
    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 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 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 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 from the investigator.

    Per Protocol Annex 1, with the exception of the inclusion criterion that requires patients must have completed all scheduled visits of the treatment phase of the RCT, GWND18064, and transitioned to OLE by the point of RCT follow-up (Visit 11), all inclusion and exclusion criteria defined in the GWND19002 protocol must be met. In addition, the following inclusion criteria apply:
    Inclusion criteria
    • Patient has completed all scheduled visits of the treatment phase of the RCT, either in clinic or completed as many scheduled assessments as feasible remotely, or has withdrawn from GWND18064 after discussion with the medical monitor due to inability to adequately monitor safety and benefit risk.
    • Visit 1 is taking place no later than 3 months after screening of new subjects for study GWND18064 has reopened at their trial site.
    E.4Principal exclusion criteria
    The patient may not enter the trial if ANY of the following apply:
    • 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).
    • Females 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 contraception 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 after the last dose.
    • Patient has been previously enrolled and dosed in this trial.
    • Patient is unwilling to abstain from donation of blood during the trial.
    • Male participants who are fertile (i.e., after puberty unless permanently sterile by bilateral orchidectomy) 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.

    Per Protocol Annex 1, with the exception of the inclusion criterion that requires patients must have completed all scheduled visits of the treatment phase of the RCT, GWND18064, and transitioned to OLE by the point of RCT follow-up (Visit 11), all inclusion and exclusion criteria defined in the GWND19002 protocol must be met. In addition, the following exclusion criteria apply:
    • Any history of suicidal behavior or any suicidal ideation in the last month or at Visit 1.
    • Patient has clinically relevant abnormalities in the electrocardiogram measured at Visit 1 (including QT interval, corrected by Bazett's correction formula [QTcB] > 450 msec, average of 3 measurements).
    • Patient has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the IMP (active and 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. If Visit 1 laboratory results indicate the patient is not eligible, the patient must be withdrawn. Prior to withdrawal for the transaminase elevations noted above, the investigator may choose to confirm the transaminase elevations by repeating the following laboratory tests within 24 to 48 hours: ALT, AST, TBL, international normalized ratio, % eosinophils, gamma-glutamyl transferase, alkaline phosphatase, and eosinophils. Should the above transaminase elevation criteria be confirmed, the patient must be withdrawn from the trial.
    • Patient has received an IMP (other than GWND18064 IMP) since participation in GWND18064.
    • Patient has been taking felbamate for less than 1 year prior to Visit 1
    • Patient is currently using or has used recreational or medicinal cannabis, cannabinoid-based medications (including Sativex
    ® [nabiximols]) or cannabidiol oral solutions (excluding GWND18064 IMP) within the 3 months prior to Visit 1 and is unwilling to abstain for the duration of the trial.
    • 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 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.
    • Female patient is pregnant (positive pregnancy test) or lactating.
    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)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Please refer to the schedule of the assessment within the protocol (Appendix 1).
    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
    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 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) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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 concerned3
    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
    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 years3
    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 years3
    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: 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.1.3.1Number of subjects for this age range: 0
    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 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.
    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 Decision2019-11-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-01-15
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
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