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    Summary
    EudraCT Number:2021-001959-13
    Sponsor's Protocol Code Number:EPICDKL5
    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:2021-07-27
    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 number2021-001959-13
    A.3Full title of the trial
    Adjunctive cannabidiol therapy in patients with CDKL5 deficiency disorder. Interventional drug study on efficacy and safety with focus on seizure effects
    Terapia con cannabidiolo in add-on nei pazienti con sindrome CDKL5. Studio interventistico con farmaco su efficacia e sicurezza con un focus sugli effetti delle crisi epilettiche.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Adjunctive cannabidiol therapy in patients with CDKL5 deficiency disorder. Interventional drug study on efficacy and safety with focus on seizure effects
    Terapia con cannabidiolo in add-on nei pazienti con sindrome CDKL5. Studio interventistico con farmaco su efficacia e sicurezza con un focus sugli effetti delle crisi epilettiche.
    A.3.2Name or abbreviated title of the trial where available
    EPICDKL5
    EPICDKL5
    A.4.1Sponsor's protocol code numberEPICDKL5
    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 SponsorFONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE
    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 Researched Limited Cambridge
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFONDAZIONE POLICLINICO UNIV A.GEMELLI IRCCS
    B.5.2Functional name of contact pointDIREZIONE SCIENTIFICA
    B.5.3 Address:
    B.5.3.1Street AddressLARGO A. GEMELLI 8
    B.5.3.2Town/ cityROMA
    B.5.3.3Post code00168
    B.5.3.4CountryItaly
    B.5.4Telephone number0630155701
    B.5.5Fax number0630155701
    B.5.6E-maildirezione.scientifica@policlinicogemelli.it
    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 Epidyolex
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameEpidyolex
    D.3.2Product code [Epidyolex]
    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 codeEpidyolex
    D.3.10 Strength
    D.3.10.1Concentration unit 1X 100 milligrams/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 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
    patients with CDKL5
    pazienti con sindrome CDKL5
    E.1.1.1Medical condition in easily understood language
    patients with CDKL5
    pazienti con sindrome CDKL5
    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 10015039
    E.1.2Term Epilepsy congenital
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • effectiveness and safety of cannabidiol adjunctive therapy in patients with CDKL5 deficiency disorder presenting uncontrolled seizures.
    - Efficacia e sicurezza del cannabidiolo nei pazienti con patologia disordine CDKL5 che presentano epilessia non adeguatamente controllata
    E.2.2Secondary objectives of the trial
    •to estimate the change of pre-/on-CBD EEGs, analysed for the following criteria: background frequency, focal slowing, reactivity, frequency of interictal epileptiform discharges (IED) and total number of recorded seizures;
    •to estimate the incidence of the following on subjects receiving CBD:
    -use of rescue medication;
    -hospitalization to treat seizures;
    -status epilepticus (SE);
    •to assess the effect of CBD on the following measures:
    - mean changes versus baseline of CGI-Improvement Score-CGI-I at 3, 6, and 12 months and of CBCL, DBC-P, sleep questionnaire at 12 months.
    • cambiamento degli EEG (pre-trattamento/ in trattamento) analizzando i seguenti criteri: frequenza dell’attività di fondo, rallentamento focale, reattività, frequenza delle anomalie epilettiche intercritiche e numero totale delle crisi registrate.
    • incidenza dei seguenti eventi nei pazienti che assumono CBD:
    utilizzo dei farmaci per le emergenze
    ospedalizzazioni per la gestione delle crisi epilettiche
    stato di male epilettico (SE)
    • variazione media rispetto al baseline del punteggio del CGI Improvement Score (CGI-I) a 3, 6 e 12 mesi, della CBCL, del DBC-P e del questionario del sonno a 12 mesi,
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    •Male and female patients aged =1 year and = 30 years
    •Patients with a confirmed diagnosis of CDKL5 deficiency disorder
    •Patients with = 4 motor seizures/month in the last 6 months at baseline
    • Patients with 12-week baseline seizure frequency data available either from seizure diaries or medical records
    • Based on the physician's clinical judgment, seizures not adequately controlled with the current treatment
    • Pazienti di sesso maschile e femminile = 1 anno e = 30 anni
    • Pazienti con diagnosi confermata di encefalopatia epilettica correlata a CDKL5
    • Pazienti con = 4 crisi motorie al mese al baseline negli ultimi 6 mesi
    • Pazienti con almeno 12 settimane di dati sulla frequenza delle crisi, desumibili dai diari delle famiglie o dalla documentazione medica
    • Pazienti con crisi epilettiche con adeguatamente controllate dall’attuale terapia secondo il giudizio medico
    E.4Principal exclusion criteria
    • Patients on an investigational drug or device within 30 days prior to the initiation of the present study
    •Patients on an artisanal cannabis derivative treatment
    •Patients with severe hepatic impairment
    • Patients contraindicated for cannabidiol use (according to SmPC), including suspected sesame seed allergy or history of hypersensitivity to cannabidiol or any ingredients in the product
    •Pregnant or lactating females
    • Pazienti che hanno introdotto un farmaco o un dispositivo sperimentale nei 30 giorni antecedenti lo studio
    • Pazienti in trattamento con altri preparati di cannabis terapeutica nel corso dello studio
    • Pazienti con danno epatico severo
    • Pazienti che presentano controindicazioni al trattamento con cannabidiolo, inclusa sospetta allergia ai semi di sesamo o storia di ipersensibilità ai componenti del cannabidiolo o ad altri ingredienti presenti nel prodotto
    • Donne in gravidanza o in allattamento.
    E.5 End points
    E.5.1Primary end point(s)
    Primary endpoint of this study:
    •to assess the effect of CBD using the following effectiveness measures:
    - median percentage change from baseline in motor seizures frequency at 6 and 12 months;
    -% withdrawal due to lack of efficacy;
    -mean change in the number of concomitant AEDs;
    - 50% responder rates for motor generalized seizures (patients with =50% reduction of all generalized seizures) at each time point visit;
    - median percentage change from baseline in all seizures type frequency at 3 and 12 months;
    - seizure freedom rate considering the last three months of treatment for motor generalized seizures and for all seizure types;
    -% change of days free from seizures for absences and myoclonic seizures;
    - score change from baseline on National Hospital Seizure Severity Scale (NHS3) for each seizure type at 6 and 12 months;
    •to assess the safety of CBD using the following measures:
    -retention rate at 6 and 12 months;
    -% withdrawal due to adverse reactions;
    - percentage of patients experiencing adverse reactions (ADRs) and serious adverse reactions
    Valutazione dell’efficacia attraverso gli effetti del CBD sulle seguenti misure:
    • variazione percentuale rispetto al baseline della frequenza mediana delle crisi motorie a 6 mesi e a 12 mesi,
    • percentuale di interruzione della terapia dovuta a mancanza di efficacia,
    • cambiamento medio nel numero dei farmaci antiepilettici concomitanti,
    • tasso di risposta del 50% per le crisi generalizzate motorie ad ogni controllo,
    • variazione percentuale rispetto al baseline della frequenza mediana di tutti i tipi di crisi epilettiche a 3 mesi e a 12 mesi,
    • tasso di libertà dalle crisi considerando gli ultimi 3 mesi di trattamento per le crisi generalizzate motorie e per tutti i tipi di crisi,
    • variazione percentuale rispetto al baseline dei giorni liberi da crisi per crisi di assenza e crisi miocloniche,
    • variazione dello score al National Hospital Seizure Severity Scale (NHS3) per ogni tipo di crisi a 6 e 12 mesi;
    Valutazione della sicurezza e tollerabilità attraverso gli effetti del CBD sulle seguenti misure:
    • Retention rate a 6 mesi e 12 mesi,
    • Percentuale di interruzione della terapia dovuta ad effetti collaterali
    • Percentuale di pazienti che hanno avuto esperienza di reazioni avverse o di serie reazioni avverse.
    E.5.1.1Timepoint(s) of evaluation of this end point
    6 and 12 months
    6 e 12 mesi
    E.5.2Secondary end point(s)
    •to estimate the change of pre-/on-CBD EEGs, analysed for the following criteria: background frequency, focal slowing, reactivity, frequency of interictal epileptiform discharges (IED) and total number of recorded seizures;
    •to estimate the incidence of the following on subjects receiving CBD:
    -use of rescue medication;
    -hospitalization to treat seizures;
    -status epilepticus (SE);
    •to assess the effect of CBD on the following measures:
    - mean changes versus baseline of CGI-Improvement Score-CGI-I at 3, 6, and 12 months and of CBCL, DBC-P, sleep questionnaire at 12 months.
    • cambiamento degli EEG (pre-trattamento/ in trattamento) analizzando i seguenti criteri: frequenza dell’attività di fondo, rallentamento focale, reattività, frequenza delle anomalie epilettiche intercritiche e numero totale delle crisi registrate.
    • incidenza dei seguenti eventi nei pazienti che assumono CBD:
    utilizzo dei farmaci per le emergenze
    ospedalizzazioni per la gestione delle crisi epilettiche
    stato di male epilettico (SE)
    • variazione media rispetto al baseline del punteggio del CGI Improvement Score (CGI-I) a 3, 6 e 12 mesi, della CBCL, del DBC-P e del questionario del sonno a 12 mesi,
    E.5.2.1Timepoint(s) of evaluation of this end point
    3, 6 e 12 mesi
    3, 6, and 12 months
    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 months0
    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 months0
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 1
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 3
    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.3.3.6.1Details of subjects incapable of giving consent
    INTELLETTUAL DISABILITY, CHILDREN
    DISABILITA' INTELLETTIVA, MINORI
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    F.4.2.2In the whole clinical trial 15
    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)
    BEST CLINICAL PRACTICE
    MIGLIOR PRATICA CLINICA
    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 Decision2021-11-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-12-02
    P. End of Trial
    P.End of Trial StatusOngoing
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