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    Summary
    EudraCT Number:2021-002482-17
    Sponsor's Protocol Code Number:TAK-935-3003
    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:2022-01-27
    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 number2021-002482-17
    A.3Full title of the trial
    A Phase 3, Prospective, Open-Label, Multisite, Extension of Phase 3 Studies To Assess the Long-Term Safety and Tolerability of Soticlestat as Adjunctive Therapy in Subjects With Dravet Syndrome or Lennox-Gastaut Syndrome (ENDYMION 2).
    Estensione di fase 3, prospettica, in aperto, multicentrica di studi di fase 3 per valutare la sicurezza e la tollerabilità a lungo termine di soticlestat come terapia aggiuntiva in soggetti con sindrome di Dravet o sindrome di Lennox-Gastaut (ENDYMION 2)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Open-Label Extension Study of Soticlestat in Dravet and Lennox-Gastaut Syndromes
    Studio di estensione in aperto di soticlestat nelle sindromi di Dravet e Lennox-Gastaut
    A.3.2Name or abbreviated title of the trial where available
    ENDYMION 2
    ENDYMION 2
    A.4.1Sponsor's protocol code numberTAK-935-3003
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/317/2020
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorTAKEDA DEVELOPMENT CENTER AMERICAS INC.
    B.1.3.4CountryUnited States
    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 supportTakeda Development Center Americas, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationTakeda Development Center Americas, Inc.
    B.5.2Functional name of contact pointMedical Director
    B.5.3 Address:
    B.5.3.1Street Address95 Hayden Avenue
    B.5.3.2Town/ cityLexington, MA
    B.5.3.3Post code02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number0016174441422
    B.5.5Fax number000000
    B.5.6E-mailDimitrios.Arkilo@takeda.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 No
    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 namesoticlestat
    D.3.2Product code [TAK-935]
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPGastroenteral use
    Oral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSOTICLESTAT
    D.3.9.1CAS number 1429505-03-2
    D.3.9.2Current sponsor codeTAK-935
    D.3.9.4EV Substance CodeSUB198078
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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.IMP: 2
    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.2.5The 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 namesoticlestat
    D.3.2Product code [TAK-935]
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPGastroenteral use
    Oral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSOTICLESTAT
    D.3.9.1CAS number 1429505-03-2
    D.3.9.2Current sponsor codeTAK-935
    D.3.9.4EV Substance CodeSUB198078
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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
    Dravet Syndrome (DS)
    Lennox-Gastaut Syndrome (LGS)
    Sindrome di Dravet (DS)
    Sindrome di Lennox-Gastaut (LGS)
    E.1.1.1Medical condition in easily understood language
    seizures
    Convulsioni
    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 LLT
    E.1.2Classification code 10073682
    E.1.2Term Dravet syndrome
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10048816
    E.1.2Term Lennox-Gastaut syndrome
    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
    - To assess the long-term safety and tolerability of soticlestat when administered as adjunctive therapy to SOC (eg, ASMs, vagus nerve stimulation, ketogenic diet, modified Atkins diet) in subjects with DS or LGS.
    - Valutare la sicurezza e la tollerabilità a lungo termine di soticlestat quando somministrato come terapia aggiuntiva allo standard di cura (Standard Of Care, [SOC]) (ad es., farmaci anticonvulsivanti [Antiseizure Medication, ASM], stimolazione del nervo vago, dieta chetogenica, dieta Atkins modificata) in soggetti affetti da SD o SLG.
    E.2.2Secondary objectives of the trial
    - To assess the effect of soticlestat on seizure frequency (convulsive seizures for the DS cohort, MMD seizures for the LGS cohort, and total seizure count for each cohort).
    - To assess the effect of soticlestat on the Clinical Global Impression of Improvement (CGI-I) (clinician) and Caregiver Global Impression of Improvement (Care GI-I).
    - To assess the effect of soticlestat on CGI-I Seizure Intensity and Duration.
    - To assess the effect of soticlestat on the CGI-I Nonseizure Symptoms completed by clinician with input from the caregiver.
    - To assess the effect on Quality of Life Inventory-Disability (QI-Disability).
    - Valutare l’effetto di soticlestat sulla frequenza delle crisi convulsive (crisi convulsive per la coorte SD, crisi convulsive con gravi cadute motorie [Major Motor Drop, MMD] per la coorte SLG e conta totale delle crisi convulsive per ciascuna coorte).
    - Valutare l’effetto di soticlestat sull’impressione clinica globale di miglioramento (Clinical Global Impression of Improvement, [CGI-I]) (da parte del medico) e sull’Impressione globale di miglioramento da parte del caregiver (Caregiver Global Impression of Improvement, [Care GI-I]).
    - Valutare l’effetto di soticlestat sull’intensità e sulla durata delle crisi convulsive secondo la scala CGI-I.
    - Valutare l’effetto di soticlestat sui sintomi delle crisi non convulsive secondo la scala CGI-I compilata dal medico con il contributo del caregiver.
    - Valutare l’effetto sul Questionario per misurare la qualità della vita (Quality of life Inventory, [QI]) - Invalidità.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subject must have:
    ¿ Been previously enrolled in soticlestat clinical study TAK-935-3001 or TAK-935-3002.
    1. Received at least 12 weeks of treatment (combined titration and Maintenance Period) with the study drug in the antecedent study and not have a serious or severe AE that, in the investigator’s or sponsor’s opinion, was related to the study drug and would make it unsafe for the patient to continue receiving the study drug.
    2. In the opinion of the investigator, the subject has the potential to benefit from the administration of soticlestat.
    3. In the opinion of the investigator, the subject and/or parent or legal guardian is capable of understanding and complying with protocol requirements including the use of digital tools, complete appropriate assessments, maintain an accurate and complete daily seizure diary and take study drug for the duration of the study.
    4. The subject or the subject’s parent or legal guardian are willing and able to read, understand, and sign and date a written or an electronic informed consent form (ICF), assent form (if applicable), and any required privacy authorization prior to the initiation of any study procedures.
    5. Female subjects of childbearing potential (defined as first menarche) must have a negative pregnancy test and agree to use an effective method of birth control during the study and for 30 days following the last dose of study drug.
    Effective contraceptive methods are as follows:
    ¿ Double-barrier method (contraceptive sponge, diaphragm, or cervical cap with spermicidal jellies or creams PLUS male condom).
    ¿ Partner with male condom.
    ¿ Combined or progestogen-only hormonal contraception
    ¿ Intrauterine device (Copper T PLUS condom)
    ¿ Intrauterine hormone-releasing system
    ¿ Sexual abstinence:
    i. Sexual abstinence may be considered as a method only if defined as refraining from heterosexual intercourse and determined to be the usual lifestyle before entering the study with reliability of abstinence for the duration of the study participation and for 30 days after last dose of study drug.
    ¿ Sterilization:
    ii. Bilateral tubal occlusion.
    iii. Vasectomized partner (provided that the partner is the sole sexual partner of the subject and the absence of sperm in the ejaculate has been confirmed).
    Il soggetto deve:
    ¿ Essere stato precedentemente arruolato nello studio clinico su soticlestat TAK-935-3001 o TAK-935-3002.
    1. Aver ricevuto almeno 12 settimane di trattamento (periodi di titolazione e di mantenimento combinati) con il farmaco dello studio nell’ambito dello studio precedente e non aver manifestato un evento avverso (EA) serio o grave che, a giudizio dello sperimentatore o dello sponsor, fosse correlato al farmaco dello studio e rendesse non sicuro per il paziente continuare a ricevere il farmaco dello studio.
    2. A giudizio dello sperimentatore, il soggetto ha il potenziale di trarre beneficio dalla somministrazione di soticlestat.
    3. A giudizio dello sperimentatore, il soggetto e/o il genitore o tutore legale è/sono in grado di comprendere e attenersi ai requisiti del protocollo, compreso l’uso di strumenti digitali, completare le valutazioni appropriate, mantenere un diario delle crisi convulsive giornaliero accurato e completo e assumere il farmaco dello studio per tutta la durata dello studio.
    4. Il soggetto o il genitore o tutore legale è disposto e in grado di leggere, comprendere, firmare e datare un modulo di consenso informato (Informed Consent Form, [ICF]) in formato elettronico o cartaceo, un modulo di assenso (se pertinente) e qualsiasi autorizzazione sulla privacy richiesta prima dell’avvio di qualsiasi procedura dello studio.
    5. I soggetti di sesso femminile in età fertile (definiti come pazienti che hanno avuto il primo menarca) devono presentare un test di gravidanza negativo e devono accettare di utilizzare un metodo contraccettivo efficace durante lo studio e per 30 giorni dopo l’ultima dose di farmaco dello studio.
    I metodi contraccettivi efficaci sono definiti come segue:
    ¿ Metodo a doppia barriera (spugna, diaframma o cappuccio cervicale contraccettivo con gel o creme spermicide PIÙ preservativo maschile).
    ¿ Partner con preservativo maschile.
    ¿ Contraccezione ormonale combinata o solo progestinica
    ¿ Dispositivo intrauterino (spirale in rame T PLUS)
    ¿ Sistema intrauterino a rilascio di ormoni
    ¿ Astinenza sessuale:
    i. L’astinenza sessuale può essere considerata un metodo solo se definita come astensione dai rapporti eterosessuali e se accertata come stile di vita abituale prima di entrare nello studio, con affidabilità dell’astinenza per la durata della partecipazione allo studio e per 30 giorni dopo l’ultima dose del farmaco dello studio.
    ¿ Sterilizzazione:
    ii. Occlusione tubarica bilaterale.
    iii. Partner vasectomizzato (a condizione che il partner sia l’unico partner sessuale del soggetto e che sia stata confermata l’assenza di spermatozoi nell’eiaculato).
    E.4Principal exclusion criteria
    1. Unstable, clinically significant neurologic (other than the disease being studied), psychiatric, cardiovascular, ophthalmologic, pulmonary, hepatic, renal, metabolic, gastrointestinal, urologic, immunologic, hematopoietic, endocrine disease, malignancy including progressive tumors, or other abnormality that may impact the ability to participate in the study or that may potentially confound the study results. It is the responsibility of the investigator to assess the clinical significance; however, consultation with the medical monitor may be warranted.
    2. Abnormal and clinically significant ECG abnormality at Visit 1 including QT interval with Fridericia correction method (QTcF) >450 ms confirmed with a repeat ECG using manual measurement of QTcF. Clinically significant ECG abnormalities should be discussed with the medical monitor.
    3. Subject is currently pregnant or breastfeeding or is planning to become pregnant during the study or within 30 days of the last dose of study drug.
    4. Subject is considered by the investigator to be at imminent risk of suicide or injury to self, others, or property. Subjects who have positive answers on item numbers 4 or 5 on the CSSRS before dosing are excluded. This scale will only be administered to subjects aged =6 years.
    1. Malattia neurologica instabile, clinicamente significativa (diversa dalla malattia oggetto di studio), psichiatrica, cardiovascolare, oftalmologica, polmonare, epatica, renale, metabolica, gastrointestinale, urologica, immunologica, ematopoietica, endocrina, neoplasia maligna, inclusi i tumori progressivi, o altra anomalia che potrebbe influire sulla capacità di partecipare allo studio o che potrebbe potenzialmente confondere i risultati dello studio. È responsabilità dello sperimentatore valutare la significatività clinica; tuttavia, può essere giustificato un consulto con il medical monitor.
    2. Anomalia dell’ECG clinicamente significativa alla Visita 1, compreso l’intervallo tra l’onda Q e l’onda T (Q-wave/T-wave, [QT]) con il metodo di correzione di Fridericia (QTcF) >450 ms, confermata da un ECG ripetuto utilizzando la misurazione manuale del QTcF. Le anomalie dell’ECG clinicamente significative devono essere discusse con il medical monitor.
    3. Il soggetto è attualmente in gravidanza o sta allattando al seno o ha in programma di avviare una gravidanza durante lo studio o entro 30 giorni dall’ultima dose del farmaco dello studio.
    4. Lo sperimentatore ritiene che il soggetto sia a rischio imminente di suicidio o lesione a se stesso, agli altri o alla proprietà. Sono esclusi i soggetti che hanno fornito risposte positive alla voce n. 4 o 5 della Scala della Columbia University per la valutazione della gravità del rischio di suicidio (Columbia Suicide Severity Rating Scale, [C-SSRS]) prima della somministrazione. Questa scala sarà somministrata solo a soggetti di età =6 anni.
    E.5 End points
    E.5.1Primary end point(s)
    - Incidence of TEAEs.
    - Change from baseline in the Columbia-Suicide Severity Rating Scale (C-SSRS) categorization based on Columbia Classification Algorithm of Suicide Assessment categories 1, 2, 3, 4, and 5 for patients =6 years of age.
    - Incidence of clinically significant abnormal safety laboratory test values, vital signs and electrocardiogram (ECG) evaluations.
    - Change from baseline in safety laboratory test values, vital signs and ECG evaluations.
    - Change from baseline in height and weight for all age groups, and in absolute value for Tanner stage for children 6 to 17 years and insulin-like growth factor 1 (IGF 1) for children 2 to 17 years of age during the study.
    - Incidenza di eventi avversi emergenti dal trattamento (Treatment Emergent Adverse Event, [TEAE]).
    - Variazione rispetto al basale nella classificazione secondo la Scala della Columbia University per la valutazione della gravità del rischio di suicidio (C-SSRS) basata sull’Algoritmo di classificazione Columbia delle categorie di valutazione del rischio di suicidio 1, 2, 3, 4 e 5 per i pazienti di età =6 anni.
    - Incidenza di valori anomali clinicamente significativi negli esami di laboratorio per la sicurezza, nei segni vitali e nelle valutazioni elettrocardiografiche (ECG).
    - Variazione rispetto al basale nei valori degli esami di laboratorio per la sicurezza, nei segni vitali e nelle valutazioni ECG.
    - Variazione rispetto al basale nell’altezza e nel peso per tutte le fasce di età e nel valore assoluto per lo stadio di Tanner per i bambini dai 6 ai 17 anni e per il fattore di crescita insulino-simile 1 (Insulin-like Growth Factor 1, [IGF 1]) per i bambini dai 2 ai 17 anni di età durante lo studio.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Primary end points will be assessed throughout the study.
    Gli endpoint primari saranno valutati durante tutto lo studio.
    E.5.2Secondary end point(s)
    - Percent change from baseline in total seizure frequency per 28 days for each (DS and LGS) cohort.
    - Percent change from baseline in convulsive seizure frequency (DS) per 28 days.
    - Percent change from baseline in MMD seizure frequency (LGS) per 28 days.
    - Effect on the CGI-I and Care GI-I.
    - Effect on CGI-I Seizure Intensity and Duration.
    - Effect on the CGI-I Nonseizure Symptoms completed by clinician with input from the caregivers.
    - Effect on QI-Disability.
    - Variazione percentuale rispetto al basale nella frequenza totale delle crisi convulsive nell’arco di 28 giorni per ciascuna coorte (SD e SLG).
    - Variazione percentuale rispetto al basale nella frequenza delle crisi convulsive (SD) nell’arco di 28 giorni.
    - Variazione percentuale rispetto al basale nella frequenza delle crisi convulsive con MMD (SLG) nell’arco di 28 giorni.
    - Effetto sulle scale CGI-I e Care GI-I.
    - Effetto sull’intensità e sulla durata delle crisi convulsive secondo la scala CGI-I.
    - Effetto sui sintomi delle crisi non convulsive secondo la scala CGI-I compilata dal medico con il contributo dei caregiver.
    - Effetto sul questionario QI - Invalidità.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary end points will be assessed throughout the study.
    Gli endpoint secondari saranno valutati durante tutto lo studio.
    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 Yes
    E.6.7Pharmacodynamic Yes
    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) 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
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA25
    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
    Belgium
    Canada
    China
    France
    Greece
    Hungary
    Italy
    Japan
    Latvia
    Netherlands
    Poland
    Russian Federation
    Serbia
    Spain
    Ukraine
    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
    The end of the study for an individual subject is defined as the last protocol-specified contact with that subject.
    The overall end of the study is defined as the last protocol-specified contact with the last subject ongoing in the study.
    La fine dello studio per un singolo soggetto è definita come l’ultimo contatto specificato nel protocollo con quel soggetto.
    La fine complessiva dello studio è definita come l’ultimo contatto specificato nel protocollo con l’ultimo soggetto ancora attivo nello studio.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    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 years4
    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) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 169
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 95
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 112
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state18
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 135
    F.4.2.2In the whole clinical trial 376
    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)
    At the end of the maintenance period, whether after the full duration or for early termination, the dose will be tapered for approximately 1 week (unless already at the lowest dose), followed approximately 2 weeks later by a safety follow-up visit or phone call.
    Alla fine del periodo di mantenimento, sia dopo l’intera durata sia in caso di interruzione anticipata, la dose sarà ridotta gradualmente per circa 1 settimana (a meno che non sia già al suo livello più basso); seguirà quindi una visita o telefonata di follow-up di sicurezza circa 2 settimane dopo.
    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 Decision2022-04-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-01-18
    P. End of Trial
    P.End of Trial StatusOngoing
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