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    Summary
    EudraCT Number:2019-001605-24
    Sponsor's Protocol Code Number:GWND19002
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2021-01-21
    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 ConcernedItaly - Italian Medicines Agency
    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
    Studio di estensione in aperto per valutare la sicurezza a lungo termine della soluzione orale di Cannabidiolo (GWP42003-P, CBD-OS) in pazienti affetti dalla sindrome di 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
    Studio di estensione in aperto per valutare la sicurezza a lungo termine di GWP42003-P in pazienti affetti dalla sindrome di Rett
    A.3.2Name or abbreviated title of the trial where available
    NA
    NA
    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 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 Yes
    D.2.1.1.1Trade name CBD soluzione orale nota come Epidyolex e con tale nome è stato approvato in UE
    D.2.1.1.2Name 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 nameCannabidiolo
    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 INNcannabidiolo
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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]
    Sindrome di Rett (RTT) [tipica o atipica]
    E.1.1.1Medical condition in easily understood language
    Rett syndrome (RTT) [typical or atypical]
    Sindrome di Rett (RTT) [tipica o atipica]
    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 (Rett Syndrome)
    Valutare la sicurezza lungo termine di GWP42003-P in pazienti con RTT (Sindrome di Rett)
    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
    • Valutare l’effetto di GWP42003-P nelle misurazioni di gravità della malattia
    o Questionario sul comportamento associato alla Sindrome di Rett (RSBQ)
    o Impressioni cliniche globali - Miglioramento (CGI-I)
    o Impressioni cliniche globali - Gravità (CGI-S)
    o Valutazione del comportamento motorio a 9 voci (MBA-9)
    o Questionario per misurare le abitudini legate al sonno nei bambini(CSHQ)

    Obiettivi esplorativi:
    • Valutare l’effetto di GWP42003-P sulla qualità della vita della paziente e del caregiver (QoL)
    o Versione breve del questionario a 36 voci [SF-36] e il Questionario sulla salute del bambino, modulo per i genitori a 50 voci [CHQ-PF50],
    rispettivamente
    • Valutare l’effetto di GWP42003-P sull’utilizzo delle risorse sanitarie
    o Questionario sull’utilizzo dei servizi ospedalieri
    o Valutazione del caregiver sui sintomi della sindrome di Rett
    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.
    • La paziente ha completato tutte le visite programmate della fase di trattamento di RCT, GWND18064, ed è passata all’OLE al momento della visita di follow-up RCT (Visita 11).
    • La paziente (se in possesso di un adeguato livello di comprensione, secondo l’opinione dello sperimentatore), e/o i suoi genitori/rappresentanti legali sono disposti e in grado di fornire un consenso/assenso informato per la partecipazione allo studio.
    • La paziente e il suo caregiver sono disposti e in grado (secondo l’opinione dello sperimentatore) di soddisfare tutti i requisiti dello studio (compreso il completamento di tutte le valutazioni del caregiver da parte dello stesso caregiver per tutta la durata dello studio).
    • La paziente deve essere in grado di deglutire il prodotto medicinale sperimentale (IMP) fornito come soluzione liquida, o deve essere essere possibile erogare l’IMP mediante gastrostomia (G) o sondino naso-gastrico (NG) (sono consentiti esclusivamente tubi G o NG in poliuretano o silicone).
    • La paziente e/o i genitori/rappresentante legale sono disposti a consentire alle autorità responsabili di essere informate circa la partecipazione allo studio, se richiesto dalle leggi locali.
    • La paziente e/o i genitori/rappresentante legale sono disposti a consentire all’operatore sanitario principale della paziente (se presente) e al consulente (se presente) di essere informati circa la partecipazione allo studio nel caso in cui l’operatore sanitario principale/consulente sia diverso dallo sperimentatore.
    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 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 thereafter.
    • Patient has been previously enrolled and dosed in this trial.
    • Patient is unwilling to abstain from donation of blood during the trial.
    • La paziente soddisfa i criteri di ritiro (inclusi valori clinici di laboratorio significativamente anomali) secondo l’opinione dello sperimentatore.
    • Durante l’RCT la paziente ha soddisfatto i criteri per l’interruzione permanente dell’IMP (a meno che, nel caso di un AE, l’AE non sia stato ritenuto correlato all’IMP; le pazienti che soddisfano i criteri di interruzione per aumento di alanina aminotransferasi (ALT) / aspartato aminotransferasi (AST) devono essere escluse).
    • La paziente è potenzialmente fertile, a meno che non sia disposta a garantire che lei o il suo partner userà un metodo contraccettivo altamente efficace (ad es. contraccettivi ormonali combinati [con estrogeni e progesterone] associati all’inibizione dell’ovulazione [orali, intravaginali o transdermici], contraccettivi a base di solo progestinico associati all’inibizione dell’ovulazione [orali, iniettabili o impiantabili] dispositivi intrauterini/sistemi intrauterini a rilascio ormonale, occlusione tubarica bilaterale, partner sottoposto a vasectomia, astinenza sessuale durante lo studio e per i 3 mesi successivi.
    • La paziente è stata precedentemente arruolata in questo studio e ha già ottenuto il dosaggio del medicinale.
    • La paziente non è disposta a interrompere la donazione di sangue durante la sperimentazione.
    E.5 End points
    E.5.1Primary end point(s)
    Safety:
    The long-term safety profile of GWP42003-P will be assessed by evaluating changes in the following, relative to the prerandomization baseline of the Randomized Controlled Trial:
    • 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)
    Sicurezza:
    Il profilo di sicurezza di GWP42003-P nel lungo periodo sarà giudicato valutando i cambiamenti nei punti sotto elencati, relativi alla baseline pre-randomizzazione dello studio randomizzato controllato:
    • Eventi avversi (AEs)
    • Parametri clinici di laboratorio
    • Segni vitali
    • Procedure della visita medica
    • Elettrocardiogramma a 12 derivazioni (ECG)
    • Effetti sul ciclo mestruale
    • Suicidalità
    • Variazione della crescita e dello sviluppo mediante la misurazione di altezza, peso, livelli del fattore di crescita insulino-simile 1 (IGF-1) e stadi di Tanner (per le pazienti di età = 7 anni o di età inferiore se clinicamente indicato in base alla comparsa del menarca o di altri segni di pubertà precoce)
    E.5.1.1Timepoint(s) of evaluation of this end point
    at all visits
    a tutte le visite
    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
    I seguenti aspetti saranno giudicati valutando le variazioni relative alla baseline di pre-randomizzazione dell’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
    Per informazioni dettagliate sui tempi di rilevazione degli endpoint, si faccia riferimento allo schema delle procedure del protocollo (Schedule of Assessments).
    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.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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 Yes
    E.8.1.7.1Other trial design description
    in aperto
    open
    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 concerned5
    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 Information not present in EudraCT
    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
    Canada
    United States
    Italy
    Spain
    United Kingdom
    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
    LVLS
    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 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.1.1.1In 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: 135
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number 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.3.3.6.1Details of subjects incapable of giving consent
    Rett Syndrome
    Sindrome di Rett
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state40
    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.
    Dopo il termine dello studio i pazienti saranno curati secondo lo standard di cura della patologia in studio.
    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 Decision2020-06-30
    N.Ethics Committee Opinion of the trial applicationWithdrawn
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-11-02
    P. End of Trial
    P.End of Trial Status
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