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    Summary
    EudraCT Number:2019-004254-28
    Sponsor's Protocol Code Number:CAEL101-301
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-06-01
    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 number2019-004254-28
    A.3Full title of the trial
    A Phase 3, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of CAEL-101 and Plasma Cell Dyscrasia Treatment Versus Placebo and Plasma Cell Dyscrasia Treatment in Plasma Cell Dyscrasia Treatment-Naïve Patients with Mayo Stage IIIb AL Amyloidosis
    Studio di Fase 3, in doppio cieco, multicentrico per valutare l’efficacia e la sicurezza di CAEL-101 e del trattamento per discrasia plasmacellulare rispetto a placebo e al trattamento per discrasia plasmacellulare in pazienti naïve al trattamento per discrasia plasmacellulare con amiloidosi AL in stadio IIIb Mayo
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A double-blind study to evaluate the effectiveness and safety of CAEL-101 in patients with AL amyloidosis.
    Studio in doppio cieco per valutare l’efficacia e la sicurezza di CAEL-101 nel trattamento per discrasia plasmacellulare
    A.3.2Name or abbreviated title of the trial where available
    CAEL 101-301
    CAEL 101-301
    A.4.1Sponsor's protocol code numberCAEL101-301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04504825
    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 SponsorALEXION PHARMACEUTICALS INCORPORATED
    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 supportALEXION PHARMACEUTICALS INCORPORATED
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationALEXION Europe SAS
    B.5.2Functional name of contact pointEuropean Clinical Trial information
    B.5.3 Address:
    B.5.3.1Street Address103-105, rue Anatole France
    B.5.3.2Town/ cityLevallois-Perret
    B.5.3.3Post code92300
    B.5.3.4CountryFrance
    B.5.4Telephone number+33787148158
    B.5.5Fax number+33787148158
    B.5.6E-mailclinicaltrials.eu@alexion.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 numberEU/3/19/2222
    D.3 Description of the IMP
    D.3.1Product nameCAEL-101
    D.3.2Product code [CAEL-101]
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeCAEL-101
    D.3.9.3Other descriptive nameCh11-1F4 (NSC-711516)
    D.3.9.4EV Substance CodeSUB198710
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    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 placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    stage IIIb cardiac AL amyloidosis
    amiloidosi AL cardiaca di stadio IIIb
    E.1.1.1Medical condition in easily understood language
    Cardiac amyloidosis is a disorder caused by deposits of an abnormal protein (amyloid) in the heart tissue. These deposits make it hard for the heart to work properly.
    L'amiloidosi cardiaca è una malattia causata da depositi di una proteina anormale (amiloide) nel tessuto cardiaco. Questi depositi rendono difficile il corretto funzionamento del cuore
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 27.0
    E.1.2Level PT
    E.1.2Classification code 10007509
    E.1.2Term Cardiac amyloidosis
    E.1.2System Organ Class 10007541 - Cardiac 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 objectives are:
    • To determine if CAEL-101 and treatment for plasma cell dyscrasia (PCD) improves overall survival in Mayo stage IIIb AL amyloidosis patients who are treatment naïve compared to treatment for PCD alone
    • To evaluate the safety and tolerability of CAEL-101 in combination with treatment for PCD
    Gli obiettivi principali sono:
    • Stabilire se CAEL-101 e il trattamento per discrasia plasmacellulare (PCD) migliori la sopravvivenza complessiva nei pazienti con amiloidosi AL in stadio IIIb Mayo, che sono naïve al trattamento rispetto al trattamento per PCD in monoterapia
    • Valutare la sicurezza e la tollerabilità di CAEL-101 in combinazione con il trattamento per PCD.
    E.2.2Secondary objectives of the trial
    The key secondary objectives in this study are:
    •To assess quality of life (QoL) as measured by the Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS)
    •To assess cardiac improvement as measured by percent Global Longitudinal Strain (GLS%)
    •To assess functional improvement as measured by the distance walked in the six-minute walk test (6MWT)
    •To assess quality of life as measured by the Short Form 36 Health Survey version 2 Physical Component Score (SF-36v2® PCS)
    I principali obiettivi secondari di questo studio sono:
    • Valutare la qualità della vita (QoL) misurata da Kansas City Punteggio complessivo del questionario sulla cardiomiopatia (KCCQ-OS)
    • Per valutare il miglioramento cardiaco misurato in percentuale globale Deformazione longitudinale (GLS%)
    • Valutare il miglioramento funzionale misurato dalla distanza percorsa nel test del cammino dei sei minuti (6MWT)
    • Valutare la qualità della vita misurata , misurata tramite il Punteggio della componente fisica del
    Questionario sulla salute breve a 36 voci, versione 2 (SF-36v2® PCS).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. AL amyloidosis stage IIIb based on the 2013 European Modification of the 2004 Standard Mayo Clinic Staging in patients with advanced cardiac involvement at the time of Screening which includes NY-proBNP > 8,500 ng/L.
    2. Measurable hematologic disease at Screening as defined by at least one of the following:
    a. Involved/uninvolved free light chain difference (dFLC) > 4 mg/dL
    or
    b. Involved free light chain (iFLC) > 4 mg/dL with abnormal Kappa/Lambda ratio OR
    c. Serum protein electrophoresis (SPEP) m-spike > 0.5 g/dL
    3. Histopathological diagnosis of amyloidosis based on polarizing light microscopy of green bi-refringent material in Congo red stained tissue specimens
    AND confirmation of AL derived amyloid deposits by at least one of the following:
    a. Immunohistochemistry /Immunofluorescence OR
    b. Mass spectrometry OR
    c. Characteristic electron microscopy appearance/ Immunoelectron microscopy
    4. Cardiac involvement as defined by:
    a. Documented clinical signs and symptoms supportive of a diagnosis of heart failure in the setting of a confirmed diagnosis of AL amyloidosis in the absence of an alternative explanation for heart failure
    AND
    b. At least one of the following:
    i. Endomyocardial biopsy demonstrating AL cardiac amyloidosis OR
    ii. Echocardiogram demonstrating a mean left ventricular wall thickness (calculated as [IVSd+LPWd]/2) of > 12 mm at diastole in the absence of other causes (e.g., severe hypertension, aortic stenosis), which would adequately explain the degree of wall thickening OR
    iii. Cardiac magnetic resonance imaging (MRI) with gadolinium contrast agent diagnostic of cardiac amyloidosis
    5. Planned first-line treatment for plasma cell dyscrasia is cyclophosphamide-bortezomib-dexamethasone (CyBorD)-based regimen administered as SoC
    6. Women of childbearing potential (WOCBP) must have a negative pregnancy test during Screening and must agree to use highly effective contraception from Screening to at least 5 months following the last study drug administration or 12 months following the last dose of her anti-PCD therapy, whichever is longer
    7. Men must be surgically sterile or must agree to use highly effective contraception and refrain from donating sperm from Screening to at least 5 months following the last study drug administration or 12 months following the last dose of their PCD therapy, whichever is longer
    1. Amiloidosi AL stadio IIIb sulla base della modifica europea del 2013 dello standard Mayo Clinic Staging del 2004 in pazienti con coinvolgimento cardiaco avanzato al momento dello screening , che include NT- proBNP >
    8.500 ng/l.
    2. Malattia ematologica misurabile allo screening come definita da almeno uno dei seguenti:
    a. Differenza di catene leggere libere coinvolte/non coinvolte (dFLC) > 4 mg/dL
    o
    b. Catena leggera libera coinvolta (iFLC) > 4 mg/dL con rapporto anomalo Kappa/Lambda o
    c. Picco m dell'elettroforesi delle proteine sieriche (SPEP) > 0,5 g/dL
    3. Diagnosi istopatologica di amiloidosi basata sulla microscopia a luce polarizzante di materiale birifrangente verde in campioni di tessuto macchiati di rosso Congo E conferma dei depositi di amiloide derivati da AL da almeno uno dei seguenti:
    a. Immunoistochimica/immunofluorescenza OPPURE
    b. Spettrometria di massa OPPURE
    c. Aspetto caratteristico della microscopia elettronica/ Microscopia immunoelettronica
    4. Coinvolgimento cardiaco come definito da:
    a. Segni e sintomi clinici documentati a supporto di una diagnosi di insufficienza cardiaca nel contesto di una diagnosi confermata di amiloidosi AL in assenza di una spiegazione alternativa per l'insufficienza cardiaca
    E
    b. Almeno uno dei seguenti:
    i. Biopsia endomiocardica che dimostra l'amiloidosi cardiaca AL OPPURE
    ii. Ecocardiogramma che dimostra uno spessore medio della parete del ventricolo sinistro (calcolato come [IVSd+LPWd]/2) di > 12 mm alla diastole in assenza di altre cause (p. es., grave ipertensione, stenosi aortica), che spiegherebbe adeguatamente il grado di ispessimento della parete OPPURE
    iii. Risonanza magnetica cardiaca (MRI) con mezzo di contrasto al gadolinio diagnostico dell'amiloidosi cardiaca
    5. Il trattamento di prima linea pianificato per la discrasia plasmacellulare è il regime a base di ciclofosfamide-bortezomib-desametasone (CyBorD) somministrato come SoC
    6. Le donne in età fertile (WOCBP) devono avere un test di gravidanza negativo durante lo screening e devono accettare di utilizzare un metodo contraccettivo altamente efficace dallo screening fino ad almeno 5 mesi dopo l'ultima somministrazione del farmaco in studio o 12 mesi dopo l'ultimo dose della sua terapia anti-PCD, a seconda di quale sia la più lunga.
    7. Gli uomini devono essere chirurgicamente sterili o devono accettare di utilizzare metodi contraccettivi altamente efficaci e astenersi dal donare lo sperma dallo screening per almeno 5 mesi dopo l'ultima somministrazione del farmaco in studio o 12 mesi dopo l'ultima dose del suo anti-PCD terapia, qualunque sia la più lunga.
    E.4Principal exclusion criteria
    1. Have any other form of amyloidosis other than AL amyloidosis
    2. Received prior therapy for AL amyloidosis or multiple myeloma. A maximum exposure of 2 weeks of a CyBorD-based PCD treatment after Screening laboratory samples are obtained and prior to randomization is allowed.
    3. Has POEMS syndrome OR multiple myeloma defined as clonal bone marrow plasma cells > 10% from a bone marrow biopsy (performed = 3 months prior to signing the ICF or during screening) OR biopsy-proven (performed = 3 months prior to signing the ICF or during Screening) bony or extramedullary plasmacytoma AND any one or more of the following CRAB features:
    a. Evidence of end organ damage that can be attributed to the underlying plasma cell proliferative disorder, (eg multiple myeloma and POEMS syndrome) specifically:
    i. Hypercalcemia: serum calcium > 0.25 mmol/L (> 1mg/dL) higher than the upper limit of normal (ULN) or > 2.75 mmol/L (> 11mg/dL) OR
    ii. Renal insufficiency: creatinine clearance < 40 mL per minute or serum creatinine > 177 µmol/L (> 2mg/dL) OR
    iii. Anemia: hemoglobin value of > 20g/L below the lowest limit of normal, or a hemoglobin value < 100g/L OR
    iv. Bone lesions: one or more osteolytic lesion on skeletal radiography, computed tomography (CT), or positron emission tomography (PET)/CT.
    If bone marrow has < 10% clonal plasma cells, more than one bone lesion is required to distinguish from solitary plasmacytoma with minimal marrow involvement
    OR
    b. Any one of the following biomarkers of malignancy:
    i. 60% or greater clonal plasma cells on bone marrow examination
    ii. More than one focal lesion on MRI that is at least 5mm or greater insize
    4. Have supine systolic blood pressure < 90 mmHg or symptomatic orthostatic hypotension, defined as a decrease in systolic blood pressure upon standing of > 30 mmHg despite medical management (e.g., midodrine, fludrocortisones) in the absence of volume depletion.
    1. Avere qualsiasi altra forma di amiloidosi diversa dall'amiloidosi AL
    2. Ha ricevuto una precedente terapia per l'amiloidosi AL o il mieloma multiplo. È consentita un'esposizione massima di 2 settimane a un trattamento PCD basato su CyBorD dopo aver ottenuto campioni di laboratorio di screening e prima della randomizzazione.
    3. Presenza di sindrome di POEMS ((polineuropatia, organomegalia, endocrinopatia,
    proteina M e alterazioni cutanee) OPPURE mieloma multiplo definito come plasmacellule clonali del midollo osseo >10% da biopsia del midollo osseo (eseguita =3 mesi prima di firmare il modulo di consenso informato o durante lo screening) o plasmacitoma osseo o extramidollare testato da biopsia (eseguita =3 mesi prima di firmare il modulo di consenso informato o durante lo screening) e una o più delle seguenti caratteristiche CRAB:
    a. Evidenza di danno d'organo finale che può essere attribuito al disturbo proliferativo delle plasmacellule (ovvero, mieloma multiplo e sindrome di POEMS) sottostante, in particolare:
    i. Ipercalcemia: calcio sierico > 0,25 mmol/L (> 1 mg/dL) superiore al limite superiore della norma (ULN) o > 2,75 mmol/L (> 11 mg/dL) OPPURE
    ii. Insufficienza renale: clearance della creatinina < 40 ml al minuto o creatinina sierica > 177 µmol/l (> 2 mg/dl) OPPURE
    iii. Anemia: valore dell'emoglobina > 20 g/l al di sotto del limite più basso della norma o valore dell'emoglobina < 100 g/l OPPURE
    iv. Lesioni ossee: una o più lesioni osteolitiche su radiografia scheletrica, tomografia computerizzata (TC) o tomografia a emissione di positroni (PET)/TC.
    Se il midollo osseo ha < 10% di plasmacellule clonali, è necessaria più di una lesione ossea per distinguerla dal plasmocitoma solitario con un coinvolgimento minimo del midollo
    OPPURE
    b. Uno qualsiasi dei seguenti biomarcatori di malignità:
    i. 60% o più di plasmacellule clonali all'esame del midollo osseo
    ii. Più di una lesione focale alla risonanza magnetica di dimensioni almeno pari o superiori a 5 mm
    4. Avere pressione arteriosa sistolica in posizione supina < 90 mmHg o ipotensione ortostatica sintomatica, definita come una diminuzione della pressione arteriosa sistolica in posizione eretta di > 30 mmHg nonostante il trattamento medico (ad es. midodrina, fludrocortisone) in assenza di deplezione di volume.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is the time to all-cause mortality and will be assessed from randomization to the date of death (for patients who died) or to the end of the Primary Evaluation Treatment Period (PETP). Patients living at the end of the study (EoS) will be censored at their last known date recorded. Patients who prematurely discontinue study treatment or withdraw from the study will be included in the analysis (even after discontinuation of study treatment or the study) using the date of death or censoring time point (i.e., last known date recorded), as appropriate.
    L’endpoint di efficacia primario è il tempo alla mortalità per tutte le cause e sarà valutato
    dalla randomizzazione fino alla data del decesso (per i pazienti deceduti) o alla fine del PETP. I pazienti che sono vivi alla fine dello studioEOS saranno censiti all’ultimo giorno di vita noto registrato. I pazienti che interrompono prematuramente il trattamento dello studio o si ritirano dallo studio saranno inclusi nell’analisi (anche dopo l’interruzione del trattamento in studio o dello studio) utilizzando la data di decesso o il punto temporale del censimento (ovvero l’ultima data nota registrata), a seconda dei casi.
    E.5.1.1Timepoint(s) of evaluation of this end point
    End of Treatment (EoT) and Follow-up every 12 weeks thereafter.The primary endpoint is also evaluated during the Treatment Period.
    Fine del trattamento e follow-up ogni 12 settimane successive. l'endpoint primario è inoltre valutato durante il periodo di trattamento
    E.5.2Secondary end point(s)
    The four key secondary efficacy endpoints are:
    • Changes from baseline to Week 50 in the KCCQ-OS
    • Changes from baseline to Week 50 in GLS%
    • Changes from baseline to Week 50 in the 6MWT distance
    • Changes from baseline to Week 50 in the SF-36 v2 PCS
    I quattro principali endpoint secondari di efficacia sono i seguenti:
    • Variazioni dal basale alla Settimana 50 nel KCCQ-OS
    • Variazioni dal basale alla Settimana 50 nel GLS%
    • Variazioni dal basale alla Settimana 50 nella distanza 6MWT
    • Variazioni dal basale alla Settimana 50 nel SF-36v2® PCS
    E.5.2.1Timepoint(s) of evaluation of this end point
    • KCCQ-QS & SF-36: Weeks 1, 14, 26, 38 & 50
    • GLS% (echocardiogram): Screening (Week -4 to 0), Weeks 14, 26 & 50
    • 6MWT : Screening (Week-4 to 0), Weeks 14, 26, 38 & 50.
    • KCCQ-QS & SF-36: settimana 1, 14, 26, 38 e 50
    • GLS% (ecocardiogramma): Screening (settimane da 4 a 0), settimana 14, 26 e 50
    • 6MWT : Screening (settimane da 4 a 0), settimana 14, 26, 38 e 50
    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 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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA50
    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
    Switzerland
    Australia
    Brazil
    Canada
    China
    Israel
    Japan
    Korea, Republic of
    Russian Federation
    United Kingdom
    United States
    Austria
    Belgium
    Czechia
    France
    Germany
    Greece
    Italy
    Netherlands
    Poland
    Spain
    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 study will be considered complete at the time when the expected number of deaths have been observed, the last surviving patient to start study drug completes at least 50 weeks of treatment or Sponsor decision to terminate the study, whichever comes first.
    Lo studio sarà considerato completo nel momento in cui il numero atteso di decessi sarà stato osservato, l'ultimo paziente sopravvissuto ad iniziare il farmaco in studio avrà completato almeno 50 settimane di trattamento o la decisione dello Sponsor di terminare lo studio, a seconda dell'evento che si verifica per primo.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months10
    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 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 74
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 50
    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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 39
    F.4.2.2In the whole clinical trial 124
    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)
    Patients who are still receiving treatment with study drug when the study is complete may be eligible for enrollment into an open-label, extension study of CAEL-101. Patients enrolling into the extension study will complete all assessments required at the 14-day EOT visit. Patients randomized to placebo in this study will be permitted to enroll
    in extension study and will start CAEL-101 in that trial.
    Pazienti che stanno ancora ricevendo il trattamento con il farmaco in studio quando il lo studio è completo possono essere idonei per l'iarruolamento in uno studio di estensione open-label, di CAEL-101. Pazienti arruolati nello studio di estensione completeranno tutte le valutazioni richieste alla visita EOT di 14 giorni.
    I pazienti randomizzati al placebo in questo studio potranno essere arruolati in studio di estensione e inizieranno CAEL-101 in 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 Decision2021-12-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-11-17
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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