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    Summary
    EudraCT Number:2019-001633-14
    Sponsor's Protocol Code Number:CBD_DEE
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-05-24
    Trial 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 number2019-001633-14
    A.3Full title of the trial
    A single-centre, open-label pilot study to assess the efficacy and safety of CBD oral solution as an adjunctive treatment for pediatric subjects with Developmental and Epileptic Encephalopathy
    Studio pilota monocentrico in aperto per valutare l’efficacia e la sicurezza di una soluzione orale a base di cannabidiolo (CBD) come adiuvante al trattamento di bambini affetti da Encefalopatia Evolutiva ed Epilettica (DEE)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A pilot study to assess the efficacy and safety of CBD oral solution in addition to standard treatment for pediatric subjects with Developmental and Epileptic Encephalopathy
    Studio pilota per valutare una soluzione orale a base di cannabidiolo (CBD) in aggiunta al trattamento standard di bambini affetti da Encefalopatia Evolutiva ed Epilettica (DEE)
    A.3.2Name or abbreviated title of the trial where available
    CBD_DEE
    CBD_DEE
    A.4.1Sponsor's protocol code numberCBD_DEE
    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 SponsorIRCCS, OSPEDALE PEDIATRICO BAMBINO GESÙ DI ROMA
    B.1.3.4CountryItaly
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportGW Pharmaceuticals
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationContract Research Organization Ospedale Pediatrico Bambino Gesù
    B.5.2Functional name of contact pointCRO
    B.5.3 Address:
    B.5.3.1Street AddressViale di Villa Pamphili 100
    B.5.3.2Town/ cityRoma
    B.5.3.3Post code00167
    B.5.3.4CountryItaly
    B.5.4Telephone number0668593539
    B.5.5Fax number06454037925
    B.5.6E-mailvaleria.antenucci@opbg.net
    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.2.1.1.1Trade name Epidiolex
    D.2.1.1.2Name of the Marketing Authorisation holderGW Pharmaceuticals plc
    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 nameEpidiolex
    D.3.2Product code [Epidiolex]
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Yes
    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 codeCBD
    D.3.9.3Other descriptive namePurified CBD
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number5 to 20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    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
    Developmental and Epileptic Encephalopathy (DEE)
    Encefalopatia Evolutiva ed Epilettica (DEE)
    E.1.1.1Medical condition in easily understood language
    Developmental and Epileptic Encephalopathy (DEE)
    Encefalopatia Evolutiva ed Epilettica (DEE)
    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 10077380
    E.1.2Term Epileptic encephalopathy
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10077380
    E.1.2Term Epileptic encephalopathy
    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
    The primary objective of the study is to demonstrate that CBD therapy reduces the number and/or severity of seizures and reduces or solves EEG abnormalities in child with DEE when taken in addition to current anti-epileptic drugs (AEDs)
    Provare che la terapia con CBD riduce il numero e la gravità delle convulsioni e riduce o risolve le anomalie dell’Elettro Encefalo Gramma (EEG) in bambini con DEE, quando assunta in aggiunta agli attuali farmaci anti epilettici.
    E.2.2Secondary objectives of the trial
    The secondary objective of the study is to assess safety and tolerability of CBD and to assess whether there is a psychomotor or cognitive improvement (depending on age) with CBD therapy
    Valutare la sicurezza e la tollerabilità de trattamento con CBD e verificare il miglioramento psicomotorio o cognitivo (in base all’età) con la terapia
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    o Male or female;
    o Aged between 2 and 7 years;
    o A documented history of DEE [7];
    o Currently taking at least 1 other AED between one and four AEDs, with a stable antiseizure treatment for the previous 4 weeks (including ketogenic diet and vagus nerve stimulation);
    o Inadequately seizure control with 2 or more current or prior AEDs;
    o Written informed consent provided by the participant and/or parent(s)/caregiver(s).
    o Sesso maschile o femminile;
    o Età compresa tra 2 e 7 anni;
    o Documentata storia di Encefaloapatia Evolutiva ed Epilettica (DEE);
    o In trattamento stabile (che includa dieta chetogenica e stimolazione del nervo vago) da non meno di 4 settimane con almeno un altro e non più di quattro altri farmaci antiepilettici (AEDs);
    o Consenso informato scritto ottenuto dal soggetto o da entrambi i genitori/rappresentante legale
    E.4Principal exclusion criteria
    o Clinically significant unstable medical or psychiatric conditions that may place patient’s safety at risk;
    o Clinically significant liver disease or serum aminotransferase (ALT or AST) >3 times the upper limit of the normal range or total bilirubin >2 times the upper limit of the normal range or international normalized ratio (INR) >1.5;
    o Known or suspected intolerance or hypersensitivity to cannabinoids or any of the excipients of the medicinal product such as sesame oil;
    o Current use of ACTH or other systemic steroids;
    o Current use (or use in the previous 2 months) of medicinal cannabis, or synthetic cannabinoid-based medications;
    o Inadequate supervision by parent(s)/caregiver(s) in the judgment of investigators
    o Stable felbamate dosing = 1 year;
    o Subjects who have been part of a clinical trial involving another investigational product in the previous six months;
    o Subjects with severe renal impairment: estimated glomerular filtration rate (eGFR) calculated as crCL < 30 ml/min, according to Schwartz equation.
    o Condizione medica o psichica instabile in modo clinicamente significativo che possa mettere a rischio la sicurezza del paziente;
    o Epatopatia clinicamente significativa, valore delle aminotransferasi seriche (ALT o AST) superiore di tre volte il limite superiore di normalità, valore della bilirubina totale superiore di 2 volte il limite superiore di normalità o valore di INR (International Normalized Ratio) superiore a 1.5;
    o Nota o sospetta intolleranza o ipersensibilità ai cannabinoidi o ad un qualsiasi eccipiente del prodotto sperimentale, come ad esempio l’olio di sesamo;
    o Attuale uso di ACTH o altro steroide sistemico;
    o Attuale uso (o uso nei due mesi precedenti) di medicinali a base di cannabis o a base di cannabinoidi sintetici;
    o Genitori o rappresentanti legali, giudicati dal medico di studio, non adatti a supervisionare adeguatamente il soggetto;
    o Trattamento stabile con falbamato da un anno o meno;
    o Partecipazione a qualsiasi altro studio che coinvolga prodotti sperimentali nei precedenti sei mesi;
    o Soggetti con severa compromissione della funzionalità renale: velocità di filtrazione glomerulare calcolata come CLcr < 30 ml/min secondo l’equazione di Schwartz.
    E.5 End points
    E.5.1Primary end point(s)
    • Assess the percentage change per 28 days from the 4-week baseline period in motor seizure frequency during the 24-week treatment period;
    • Assess EEG improvement from baseline during treatment period in a blind senior epileptologists evaluation procedure; a score will be established for each patient, based on review and comparison of all baseline-EEG/8-weeks control-EEG and baseline-EEG/14-weeks control-EEG, with values ranging from 0 (= worsened EEG), to a maximum of 2 (= improved); 1 will be assigned if the EEG trace is unmodified.
    - Valutare la percentuale di cambiamento nella frequenza delle convulsioni motorie nei 28 giorni successivi al periodo di screening di 4 settimane.
    - Valutare il miglioramento dei parametri di EEG durante il periodo di trattamento, in confronto al baseline, secondo una procedura di valutazione in cieco da parte di un neurologo; sarà stabilito un punteggio per ciascun paziente, sulla base della valutazione del confronto dell’EEG-basale rispetto all’EEG di confronto a: 8 settimane, 14 settimane, sulla base di valori he variano da 0 (EEG peggiorato) ad un massimo di 2 (EEG migliorato), con il valore 1 assegnato in caso di variazioni non riscontrate
    E.5.1.1Timepoint(s) of evaluation of this end point
    • 28 days within period from week 4 to week 24;
    • from baseline to 8 and 14 weeks;
    • 28 giorni entro il periodo dalla settimana 4 alla settimana 24;
    • dal basale a 8 e 14 settimane;
    E.5.2Secondary end point(s)
    • Assess the safety and tolerability of CBD as an adjunctive therapy based on safety variables (adverse events, laboratory tests, ECG, vital signs, body weight, physical examination, neurological examination);
    • Number of subjects considered treatment responders, defined as those with a =25%, =50% or =75% reduction in convulsive seizures from baseline;
    • Number of subjects who are convulsive seizure free
    • Change in number of seizures by subtype
    • Change in severity of seizures
    • Change in number of seizure-free days
    • Change from baseline in cognitive score
    • Change from baseline in adaptive score
    • Change from baseline in Behaviour score
    • Change from baseline in quality of life score
    • Change from baseline in parent evaluation
    • Change from baseline in Investigator evaluation
    • Valutazione della sicurezza e tollerabilità di CBD come terapia antiepilettica aggiuntiva, sulla base delle seguenti variabili di sicurezza: eventi avversi, parametri di laboratorio, Elettrocardiogramma (RCG), segni vitali, peso corporeo, esame fisico, valutazione neurologica;
    • Numero di soggetti che presentano una riduzione delle convulsioni epilettiche dal basale;
    • Numero di soggetti che sono liberi da convulsioni epilettiche
    • Modifica nel numero del sottotipo di crisi epilettiche
    • Modifica nella gravità delle crisi epilettiche
    • Modifica del numero di giorni liberi da crisi epilettiche
    • Modifica, dal baseline, del punteggio della valutazione cognitiva
    • Modifica, dal baseline, del punteggio adattivo
    • Modifica, dal baseline del,, punteggio comportamentale
    • Modifica dal baseline del punteggio sulla qualità della vita
    • Modifica dal baseline del punteggio della valutazione dei sintomi da parte del genitore/rappresentante legale
    • Modifica dal baseline del punteggio della valutazione da parte del medico
    E.5.2.1Timepoint(s) of evaluation of this end point
    20 weeks
    20 settimane
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.7Trial has a data monitoring committee No
    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 years1
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months8
    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: 20
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    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 No
    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
    Minors
    Minori
    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 20
    F.4.2.2In the whole clinical trial 20
    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)
    Standard of care or possibility to participate in a long term forllow up separate study.
    Cura Standard o possibilità di partecipare ad uno studio di estensione di follow-up.
    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 Decision2019-12-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-03-05
    P. End of Trial
    P.End of Trial StatusOngoing
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