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    Summary
    EudraCT Number:2020-004011-28
    Sponsor's Protocol Code Number:A4250-012
    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:2021-05-20
    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 number2020-004011-28
    A.3Full title of the trial
    A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT)
    Studio di fase 3 in doppio cieco, randomizzato, controllato verso placebo sulla sicurezza e l’efficacia di odevixibat (A4250) in pazienti affetti da sindrome di Alagille (ASSERT)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome
    Un'indagine sulla sicurezza e l'efficacia di Odevixibat nei pazienti con sindrome di Alagille
    A.3.2Name or abbreviated title of the trial where available
    NA
    NA
    A.4.1Sponsor's protocol code numberA4250-012
    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 SponsorALBIREO AB
    B.1.3.4CountrySweden
    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 supportAlbireo AB
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlbireo AB
    B.5.2Functional name of contact pointClinical Operations Department
    B.5.3 Address:
    B.5.3.1Street AddressArvid Wallgrens backe 20
    B.5.3.2Town/ cityGöteborg
    B.5.3.3Post code41346
    B.5.3.4CountrySweden
    B.5.6E-mailmedinfo@albireopharma.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 Yes
    D.2.5.1Orphan drug designation numberEMA/OD/023/12
    D.3 Description of the IMP
    D.3.1Product nameOdevixibat
    D.3.2Product code [A4250]
    D.3.4Pharmaceutical form Capsule, hard
    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 INNOdevixibat
    D.3.9.1CAS number 501692-44-0
    D.3.9.2Current sponsor codeA4250
    D.3.9.3Other descriptive nameA4250
    D.3.9.4EV Substance CodeSUB126128
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1200
    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 Yes
    D.2.5.1Orphan drug designation numberEMA/OD/023/12
    D.3 Description of the IMP
    D.3.1Product nameOdevixibat
    D.3.2Product code [A4250]
    D.3.4Pharmaceutical form Capsule, hard
    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 INNOdevixibat
    D.3.9.1CAS number 501692-44-0
    D.3.9.2Current sponsor codeA4250
    D.3.9.3Other descriptive nameA4250
    D.3.9.4EV Substance CodeSUB126128
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number600
    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: 3
    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 Yes
    D.2.5.1Orphan drug designation numberEMA/OD/023/12
    D.3 Description of the IMP
    D.3.1Product nameOdevixibat
    D.3.2Product code [A4250]
    D.3.4Pharmaceutical form Capsule, hard
    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 INNOdevixibat
    D.3.9.1CAS number 501692-44-0
    D.3.9.2Current sponsor codeA4250
    D.3.9.3Other descriptive nameA4250
    D.3.9.4EV Substance CodeSUB126128
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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: 4
    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 Yes
    D.2.5.1Orphan drug designation numberEMA/OD/023/12
    D.3 Description of the IMP
    D.3.1Product nameOdevixibat
    D.3.2Product code [A4250]
    D.3.4Pharmaceutical form Capsule, hard
    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 INNOdevixibat
    D.3.9.1CAS number 501692-44-0
    D.3.9.2Current sponsor codeA4250
    D.3.9.3Other descriptive nameA4250
    D.3.9.4EV Substance CodeSUB126128
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number400
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, hard
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, hard
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Alagille Syndrome
    Sindrome di Alagille
    E.1.1.1Medical condition in easily understood language
    Alagille syndrome is a childhood disease that affects the liver and different parts of the body. These children have buildup of a fluid made by the liver which can cause severe itch.
    La sindrome di Alagille è una malattia infantile che colpisce il fegato e diverse parti del corpo.Questi bambini hanno un accumulo di un fluido prodotto dal fegato che può causare un forte prurito.
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10053870
    E.1.2Term Alagille 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
    The primary objective is to demonstrate the efficacy of repeated daily doses of 120 µg/kg/day odevixibat in relieving pruritus in patients with ALGS.
    Dimostrare l’efficacia di dosi giornaliere ripetute di 120 µg/kg/die di odevixibat nell’alleviare il prurito nei pazienti affetti da ALGS.
    E.2.2Secondary objectives of the trial
    To assess the impact of odevixibat on serum bile acid levels in patients with ALGS.
    To evaluate the safety and tolerability of odevixibat in patients with ALGS.
    Valutare l’impatto di odevixibat sui livelli di acidi biliari nel siero nei pazienti affetti da ALGS.
    Valutare la sicurezza e la tollerabilità di odevixibat in pazienti affetti da ALGS.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. A male or female patient (of any age) with genetically confirmed diagnosis of ALGS. A patient may be randomized based on genetic testing results in the medical record. If genetic testing results are not available, testing will be performed at Screening Visit 1, and the patient may not be randomized until the genetic diagnosis is confirmed.
    2. Patient must have a history of significant pruritus and a caregiver reported observed scratching or a patient-reported pruritus score at an average of =2 (on 0 to 4 scale), as measured by the Albireo ObsRO instrument (for patients <18 years of age) or the PRO instrument (for patients >18 years of age) in the 14 days prior to randomization. For each AM and PM weekly assessment a minimum of 4 out of 7 expected scores must be recorded. The mean of the weekly AM and the mean of the weekly PM scores will be averaged to determine the pruritus score as measured by ObsRO or PRO, if the patient is =18 years of age.
    3. Patient must have an elevated baseline serum bile acid level. Each of the serum bile acid levels obtained at Screening Visit 1 and Screening Visit 2 must be greater than the upper limit of normal (>ULN).
    4. Patient and/or legal guardian must sign informed consent (and assent) as appropriate. Patients who turn 18 years of age (or legal age per country) during the study will be required to re-consent in order to remain in the study.
    5. Caregivers must be willing and able to use an eDiary device as required by the study and patients =8 years of age must be willing to use an eDiary if able to do so.
    6. Sexually active males and females must agree to use a reliable contraceptive method with =1% failure rate (such as hormonal contraception, intrauterine device, or complete abstinence) throughout the duration of the study and 90 days thereafter (from signed informed consent through 90 days after last dose of study drug).
    1. Paziente uomo o donna (di qualsiasi età) con diagnosi di ALGS confermata geneticamente. Un/a paziente può essere randomizzato/a sulla base dei risultati dei test genetici presenti nella cartella clinica. Se non sono disponibili i risultati dei test genetici, questi saranno eseguiti durante la visita di screening 1 e il paziente non potrà essere randomizzato fino a quando non sarà confermata la diagnosi genetica.
    2. Il paziente deve presentare una anamnesi di prurito significativo e un grattamento osservato da chi lo assiste o un punteggio di prurito riferito dal paziente di =2 (su una scala da 0 a 4) in media, come misurato dallo strumento Albireo ObsRO (per pazienti di età <18 anni) o dallo strumento PRO (per pazienti di età > 18 anni) nei 14 giorni precedenti la randomizzazione. Per ogni valutazione settimanale della mattina e della sera è necessario registrare un minimo di 4 dei 7 punteggi previsti. In base ai punteggi settimanali della mattina e ai punteggi settimanali della sera sarà calcolata una media per determinare il punteggio del prurito misurato tramite ObsRO o PRO, se il paziente ha un’età =18 anni.
    3. Il paziente deve presentare un livello elevato di acidi biliari nel siero al basale. Ciascuno dei livelli di acidi biliari nel siero ottenuti alla visita di screening 1 e alla visita di screening 2 deve essere maggiore del limite superiore della norma (>ULN).
    4. Il paziente e/o il tutore legale devono firmare il consenso informato (e l’assenso) come del caso. I pazienti che compiono 18 anni (o l'età legale secondo il Paese) durante lo studio dovranno rinnovare il consenso per rimanere nello studio.
    5. Le persone che assistono i pazienti devono essere disposte e in grado di utilizzare un dispositivo eDiary come richiesto dallo studio e i pazienti di età =8 anni devono essere disposti a utilizzare un eDiary se in grado di farlo.
    6. I soggetti di sesso maschile e femminile sessualmente attivi devono accettare di utilizzare un metodo contraccettivo affidabile con un tasso di insuccesso =1% (come contraccezione ormonale, dispositivo intrauterino o astinenza completa) per tutta la durata dello studio e per i 90 giorni successivi (a partire dalla firma del consenso informato e per 90 giorni dopo l’ultima dose del farmaco in studio).
    E.4Principal exclusion criteria
    1. Patient with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following:
    a) Biliary atresia
    b) Progressive Familial Intrahepatic Cholestasis (PFIC)
    c) Benign recurrent intrahepatic cholestasis
    d) Suspected or proven liver cancer or metastasis to the liver on imaging
    studies
    2. Patient with a past medical history or ongoing presence of any other disease or condition known to interfere with the absorption, distribution, metabolism (specifically bile acid metabolism), or excretion of drugs in the intestine, including but not limited to, inflammatory bowel disease
    3. Patient with past medical history or ongoing chronic (i.e. >3 months) diarrhea requiring intravenous fluid or nutritional intervention for treatment of the diarrhea and/or its sequelae
    4. Patient has a confirmed past diagnosis of infection with human immunodeficiency virus or other present and active, clinically significant chronic infection
    5. Recent infection requiring hospitalization or treatment with parenteral anti-infective within 4 weeks of randomization (Study Day 1) or completion of oral anti-infective treatment within 2 weeks prior to start of Screening Period
    6. Cancer within the last 5 years except for basal cell carcinoma
    7. Cancer >5 years prior to screening except for non-liver cancers with no evidence of recurrence
    8. Chronic kidney disease with an impaired renal function and a glomerular filtration rate <70 mL/min/1.73 m2
    9. Patient with surgical history of disruption of the enterohepatic circulation (biliary diversion surgery) within 6 months prior to start of Screening Period
    10. Patient has had a liver transplant or a liver transplant is planned within 6 months of randomization
    11. Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
    12. International normalized ratio (INR) >1.4 (the patient may be treated with Vitamin K intravenously, and if INR is =1.4 at resampling the patient may be randomized)
    13. Serum alanine aminotransferase (ALT) >10 × ULN at Screening
    14. Serum ALT >15 × ULN at any time point during the last 6 months unless an alternate etiology was confirmed for the elevation
    15. Total bilirubin >15 × ULN at Screening
    16. Patient suffers from uncontrolled, recalcitrant pruritic condition other than ALGS. Examples include, but not limited to, refractory atopic dermatitis or other primary pruritic skin diseases
    1. Pazienti con anamnesi pregressa o presenza attuale di altri tipi di malattia epatica tra cui, a titolo esemplificativo e non esaustivo:
    a) atresia biliare
    b) colestasi intraepatica familiare progressiva (PFIC)
    c) colestasi intraepatica ricorrente benigna
    d) tumore al fegato sospetto o accertato o metastasi al fegato evidenziate da esami di diagnostica per immagini
    2. Pazienti con anamnesi pregressa o presenza attuale di un’altra malattia o condizione che interferisce con l’assorbimento, la distribuzione, il metabolismo (in particolare il metabolismo degli acidi biliari) o l’escrezione di farmaci nell’intestino, inclusa a titolo esemplificativo e non esaustivo la malattia infiammatoria intestinale
    3. Pazienti con anamnesi pregressa o presenza attuale di diarrea cronica (ossia > 3 mesi) che richiede infusione di liquidi per via endovenosa o intervento nutrizionale per il trattamento della diarrea e/o delle sue conseguenze
    4. Pazienti con diagnosi pregressa confermata di infezione da virus dell’immunodeficienza umana o di altra infezione cronica clinicamente significativa presente e attiva
    5. Infezione recente che richiede il ricovero in ospedale o il trattamento con una terapia antinfettiva parenterale entro 4 settimane dalla randomizzazione (giorno 1 dello studio) o completamento della terapia antinfettiva orale nelle 2 settimane prima dell’inizio del periodo di screening
    6. Cancro negli ultimi 5 anni ad eccezione del carcinoma basocellulare
    7. Cancro >5 anni prima dello screening ad eccezione dei tumori non epatici senza evidenza di recidiva
    8. Malattia renale cronica con funzione renale compromessa e velocità di filtrazione glomerulare <70 ml/min/1,73 m2
    9. Pazienti con anamnesi chirurgica di interruzione della circolazione enteroepatica (chirurgia di diversione biliare) nei 6 mesi prima dell’inizio del periodo di screening
    10. Pazienti che abbiano subito un trapianto di fegato o che abbiano in programma di sottoporsi a trapianto di fegato entro 6 mesi dalla randomizzazione
    11. Malattia epatica scompensata, anamnesi o presenza di ascite clinicamente significativa, emorragia da varici e/o encefalopatia
    12. Rapporto internazionale normalizzato (INR) >1,4 (è possibile un trattamento con vitamina K per via endovenosa e se l’INR è =1,4 all’esame successivo si può procedere alla randomizzazione)
    13. Alanina aminotransferasi (ALT) sierica >10 × ULN allo screening
    14. ALT sierica >15 × ULN in qualsiasi momento durante gli ultimi 6 mesi a meno che non sia stata confermata un’eziologia alternativa per l’aumento
    15. Bilirubina totale > 15 × ULN allo screening
    16. Pazienti affetti da una condizione pruriginosa non controllata e recalcitrante diversa da ALGS (compresa, a titolo esemplificativo e non esaustivo, dermatite atopica refrattaria o altra malattia cutanea pruriginosa primaria)
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in scratching to Month 6 (Weeks 21 to 24) as measured by the Albireo ObsRO caregiver instrument
    Variazione del prurito dal basale al mese 6 (settimane da 21 a 24) come misurato dallo strumento Albireo ObsRO per l’assistente del paziente
    E.5.1.1Timepoint(s) of evaluation of this end point
    Change from baseline in scratching to Month 6 (Weeks 21 to 24)
    Modifica dal basale nello scratching al mese 6 (settimane da 21 a 24)
    E.5.2Secondary end point(s)
    Change in serum bile acid levels from baseline to the average of Week 20 and Week 24.
    Variazione dei livelli sierici di acidi biliari dal basale alla media della settimana 20 e della settimana 24.
    E.5.2.1Timepoint(s) of evaluation of this end point
    From baseline to the average of Week 20 and Week 24.
    Dal basale alla media della Settimana 20 e della Settimana 24.
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA17
    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
    Denmark
    France
    Germany
    Italy
    Netherlands
    Poland
    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
    The end of the study is defined as follows:
    • End of study in one country: last patient last visit (LPLV) in the country and sites in the country are closed
    • End of study globally: LPLV globally and all study sites closed
    La fine dello studio è definita come segue:
    • Fine dello studio in un paese: l'ultima visita dell'ultimo paziente (LPLV) nel paese e i centri nel paese sono chiusi
    • Fine dello studio a livello globale: LPLV a livello globale e tutti i centri studio chiusi
    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 months11
    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 months11
    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: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 22
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 21
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 18
    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
    Subjects under age incapable of giving consent personally.
    Soggetti minorenni incapaci di prestare personalmente il consenso.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 60
    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)
    Following completion of this study, patients have the option to continue in an open-label extension study, provided they meet eligibility criteria for that study.
    Dopo il completamento di questo studio, i pazienti hanno la possibilità di continuare uno studio di estensione in aperto, a condizione che soddisfino i criteri di ammissibilità per tale 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 Decision2021-02-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-12-15
    P. End of Trial
    P.End of Trial StatusOngoing
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