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    Summary
    EudraCT Number:2016-000704-28
    Sponsor's Protocol Code Number:GCP#05.01.020
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-08-29
    Trial results View 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 number2016-000704-28
    A.3Full title of the trial
    A Multicenter, Randomized, Phase III Registration Trial of Transplantation of NiCord®, Ex Vivo Expanded, Umbilical Cord Blood-derived, Stem and Progenitor Cells, versus Unmanipulated Umbilical Cord Blood for Patients with Hematological Malignancies
    Studio Clinico Multicentrico, Randomizzato, Registrativo di Fase III sul Trapianto di NiCord®, Cellule Staminali e Cellule Progenitrici derivate da Sangue del Cordone Ombelicale, Espanse Ex-Vivo, verso Sangue da Cordone Ombelicale Non Manipolato per Pazienti con Tumori Ematologici.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A multicenter trial of transplantation of NiCord, stem cells from donated umbilical cord blood that were grown in a laboratory to expand their number versus unmanipulated umbilical cord blood for patients with cancers of the blood and lymph
    Uno studio multicentrico di trapianto di NiCord, cellule staminali da sangue del cordone ombelicale donato che vengono coltivate in un laboratorio per espandere il loro numero verso sangue da cordone ombelicale non manipolato per i pazienti con tumori del sangue e linfatici
    A.4.1Sponsor's protocol code numberGCP#05.01.020
    A.5.4Other Identifiers
    Name:naNumber:na
    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 SponsorGamida Cell Ltd
    B.1.3.4CountryIsrael
    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 supportGamida Cell Ltd
    B.4.2CountryIsrael
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGamida Cell Ltd
    B.5.2Functional name of contact pointClinical trial department
    B.5.3 Address:
    B.5.3.1Street Address5, Nahum Hafzadi, Beit Ofer
    B.5.3.2Town/ cityJerusalem
    B.5.3.3Post code9548401
    B.5.3.4CountryIsrael
    B.5.4Telephone number97226595666
    B.5.5Fax number97226595666
    B.5.6E-mailIrit@gamida-cell.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/3/17/1852
    D.3 Description of the IMP
    D.3.1Product nameNiCord (CF cultured fraction and NF non-cultured fraction)
    D.3.4Pharmaceutical form Dispersion 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.3.8INN - Proposed INNAllogeneic, ex vivo expanded, umbilical cord blood-derived, hematopoietic CD34+ progenitor cells
    D.3.9.3Other descriptive nameALLOGENEIC, EX VIVO EXPANDED, UMBILICAL CORD BLOOD-DERIVED, HEMATOPOIETIC CD34+ PROGENITOR CELLS
    D.3.9.4EV Substance CodeSUB181698
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number800000000
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAllogeneic, non-expanded, umbilical cord blood-derived, hematopoietic mature myeloid and lymphoid cells
    D.3.9.3Other descriptive nameALLOGENEIC, NON-EXPANDED, UMBILICAL CORD BLOOD-DERIVED, HEMATOPOIETIC MATURE MYELOID AND LYMPHOID CELLS
    D.3.9.4EV Substance CodeSUB181699
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number400000000
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product Yes
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    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
    High risk haematological malignancies
    Neoplasie Ematologiche ad alto rischio
    E.1.1.1Medical condition in easily understood language
    cancer affecting blood, bone marrow, lymph nodes
    Tumore maligno che colpisce il sangue, il modollo osseo e i linfonodi
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10000845
    E.1.2Term Acute lymphoblastic leukemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10000886
    E.1.2Term Acute myeloid leukemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10009015
    E.1.2Term Chronic myeloid leukemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10028534
    E.1.2Term Myelodysplastic syndrome NOS
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10006597
    E.1.2Term Burkitt's lymphoma NOS
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10076434
    E.1.2Term Hepatosplenic gamma-delta T-cell lymphoma
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10025315
    E.1.2Term Lymphoma malignant
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10036889
    E.1.2Term Prolymphocytic leukemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10073481
    E.1.2Term Enteropathy-associated T-cell lymphoma
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10054481
    E.1.2Term Natural killer-cell leukemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10054655
    E.1.2Term T-cell type acute leukemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10000836
    E.1.2Term Acute leukemia NOS
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10067837
    E.1.2Term Acute biphenotypic leukemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10054681
    E.1.2Term Undifferentiated type acute leukemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The overall study objective is to compare the safety and efficacy of NiCord single ex-vivo expanded cord blood unit transplantation to unmanipulated cord blood unit transplantation in patients with hematological malignancies following conditioning therapy
    L'obiettivo generale dello studio è confrontare la sicurezza e l'efficacia del trapianto di NiCord® singola unità di sangue cordonale espansa ex vivo rispetto al trapianto di unità di sangue cordonale non manipolato in pazienti con tumori maligni ematologici dopo terapia di condizionamento come segue
    E.2.2Secondary objectives of the trial
    Not applicable - see primary and secondary endpoints sections
    NA, si vedano le sezioni relative agli endpoints primari e secondari
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.12-65 years of age
    2.Patients with one of the following HM: Acute Lymphoblastic Leukemia (ALL), Acute Myeloid Leukemia (AML), Chronic Myeloid Leukemia (CML), Myelodysplastic Syndrome (MDS) and Lymphoma as defined in the protocol. Biphenotypic/undifferentiated/Prolymphocytic/Dendritic Cell Leukemias and Natural Killer Cell Malignancies in first or subsequent CR, adult T-cell leukemia/lymphoma in first or subsequent CR. Patients with CLL are not eligible regardless of disease status.
    3.CBU criteria as described in the protocol;
    4.Patients who will be starting conditioning prior to NiCord release for infusion must have an additional partially HLA-matched CBU reserved as a BU to the NiCord arm in case of production failure. The BU CBU must be HLA-matched at 4-6/6 HLA class I(HLA-A&HLA-B, low resolution) and II (HLA-DRB1,high resolution) loci with the patient. A second BU CBU is recommended to be added in the below cases: If the BU CBU is HLA matched at 5-or 6/6, and contains a pre-cryopreserved total nucleated cell dose of<2.5x10^7 TNC/kg, OR a pre- cryopreserved CD34+ cell dose
    of<1.2 x10^5CD34+ cells/kg. If the BU CBU is HLA-matched at 4/6, and contains a pre-cryopreserved total nucleated cell dose of<3.5x10^7 TNC/kg, OR a pre cryopreserved CD34+ cell dose of
    <1.7x10^5CD34+cells/kg. In case of two BU CBUs, the second BU CBU must also be HLA-matched at 4-6/6 HLA class I(HLA-A&HLA-B, low resolution) and II (HLA-DRB1,high resolution) loci with the patient. The BU CBUs are recommended to have a combined pre-cryopreserved total
    nucleated cell dose of at least 3x10^7 TNC/kg.
    5.Patient's Performance score ≥70% by Karnofsky or Lansky
    6. Patient has sufficient physiologic reserves including: a.Cardiac: LVEF of ≥40% by echocardiogram, radionuclide scan or cardiac MRI b.PFT demonstrating FVC and FEV1 of >50% of predicted for age and cDLCO>50% of predicted c.Renal: Creatinine clearance test ≥60mL/min d.Hepatic: Serum Bilirubin<2.0 mg/dl; Hepatic transaminases<3xupper limit of normal range
    7.Females of childbearing potential, defined as any female who has experienced menarche and is not postmenopausal or permanently sterilized, agree to use an appropriate method of contraception from at least 7 days prior to conditioning regimen therapy until completion of FU procedures.
    8.Patient signs the written informed consent after being aware of the nature of the patient's disease and willingly consents to the treatment program after being informed of alternative treatments, potential risks, benefits, and discomforts
    11. Un’età di 12-65 anni
    2. Pazienti con uno dei seguenti tumori maligni ematologici:• Leucemia linfoblastica acuta (ALL), Leucemia mieloide acuta (AML), Leucemia mielocitica cronica (CML), Sindrome mielodisplastica (MDS) e Linfoma come definito nel protocollo.
    Leucemia a cellule dendritiche/prolinfocitica/indifferenziata/bifenotipica e tumori maligni delle cellule natural killer in prima o successiva CR, leucemia/linfoma dell'adulto a cellule T in prima o successiva CR. I pazienti affetti da CLL non sono eleggibili a prescindere dallo stato della malattia.
    , 3. Criteri per CBU come descritti nel protocollo;
    4. I pazienti che inizieranno il condizionamento prima del rilascio di NiCord per infusione devono avere una CBU aggiuntiva parzialmente compatibile per HLA a disposizione come backup nel braccio con NiCord in caso di insuccesso della produzione. La CBU di backup deve essere compatibile per HLA in misura 4-6/6 ai loci della regione HLA di classe I (HLA-A e HLA-B, bassa risoluzione) e II (HLA-DRB1, alta risoluzione) con il paziente. Si raccomanda di aggiungere una seconda CBU di backup nei seguenti casi: Qualora la CBU di backup sia compatibile per HLA in misura 5 o 6/6 e contenga una dose totale di cellule nucleate pre-crioconservate di <2,5x107 TNC/kg, O una dose di cellule CD34+ pre-crioconservate di <1,2x105 cellule CD34+/kg. Nel caso di due CBU di backup, la seconda CBU di backup deve anche essere compatibile per HLA in misura 4-6/6 ai loci della regione HLA di classe I (HLA-A e HLA-B, bassa risoluzione) e II (HLA-DRB1, alta risoluzione) con il paziente. Si raccomanda che le CBU di backup presentino una dose combinata totale di cellule nucleate pre-crioconservate (post trattamento) di almeno 3x107 TNC/kg.
    5. Punteggio di performance del paziente di ≥70% secondo Karnofsky o Lansky
    6. Il paziente ha sufficienti riserve fisiologiche che includono: a. Cardiache: Frazione di eiezione ventricolare sinistra (LVEF) di ≥40% da ecocardiogramma, scansione con radionuclidi o RMI cardiaca, b. Test di funzionalità polmonare (prima del trattamento con broncodilatatori) che mostrano FVC e FEV1 di > 50% del valore previsto in base all'età e cDLCO di > 50% del valore previsto.c. Renali: Test della clearance della creatinina ≥60 ml/min. d. Epatiche: Bilirubina sierica < 2,0 mg/dl; transaminasi epatiche (ALT e AST) < 3 volte il limite superiore del range normale.
    7. Le donne in età fertile, definite come donne che hanno raggiunto il menarca e non si trovano in stato di post-menopausa o non sono sterilizzate in modo permanente, accettano di usare un metodo contraccettivo appropriato da almeno 7 giorni prima del regime di condizionamento fino al completamento delle procedure di follow-up.
    8. Firma del paziente del modulo di consenso informato scritto in seguito all’identificazione della natura della malattia del paziente e consenso volontario al programma di trattamento dopo essere stato informato in merito a trattamenti alternativi, potenziali rischi, benefici e disagi.
    E.4Principal exclusion criteria
    1.MDS or CML with "marked" or "3+" fibrosis
    2.CMMoL or MDS/CMMoL overlap
    3.Fewer than 21 days have elapsed since initiation of the patient's last
    chemotherapy cycle and the initiation of the stem cell transplant
    preparative regimen (radiotherapy,intrathecal agents, hydroxyurea,
    tyrosine kinase inhibitors, hypomethylating agents, rituximab and
    lenalidomide are not considered chemotherapy)
    4.Persistent clinically significant toxicities that, in the investigator's
    opinion, make the patient unsuitable for transplant
    5.Evidence of donor specific anti-HLA antibodies to the selected
    treatment CBU #1 (MFI>3000 to HLA A, B, C, or DRB1)
    6.Evidence of HIV infection or HIV positive serology
    7.Evidence of active Hepatitis B or Hepatitis C as determined by serology
    or PCR
    8.Pregnancy, as indicated by a positive serum or urine human chorionic
    gonadotrophin (HCG) test, or lactation
    9.Active malignancy other than that for which the UCB transplant is being performed within 12 months of enrollment. Fully resected
    cutaneous squamous cell or basal cell carcinoma or cervical carcinoma in
    situ within 12 months of enrollment will be permitted.
    10.Evidence of uncontrolled bacterial, fungal or viral infections or severe
    concomitant diseases, which in the judgment of the Principal
    investigator indicate that the patient could not tolerate transplantation
    11.Patients with presence of leukemic blasts in the central nervous
    system (CNS)
    12.Patients with an 8/8 allele level HLA-matched and readily available
    related or unrelated donor (whose stem cells can be collected in a timely
    manner without jeopardizing recipient outcome). Patients who have
    haploidentical related donors will not be excluded
    13.Prior allogeneic hematopoietic stem cell transplant
    14.Allergy to bovine products, gentamicin, or to any other product that
    may interfere with the treatment
    15.Psychologically incapable of undergoing bone marrow transplant
    (BMT) with associated strict isolation or documented history of medical
    non-compliance and/or psychiatric illness and/or social situations that
    would limit compliance with study requirements
    16.Enrolled in another interventional clinical trial or received an
    investigational treatment within 30 days prior to the approved date of
    randomization, unless documented approval obtained from Sponsor prior
    to the anticipated date of randomization, unless documented approval
    obtained from Sponsor prior to randomization
    1. MDS o CML affetti da fibrosi con "marcatori" o " 3+"
    2. CMMoL o sovrapposizione MDS/CMMoL
    3. Sono trascorsi meno di 21 giorni dall'inizio dell'ultimo ciclo di chemioterapia del paziente e l'avvio del regime di preparazione del trapianto di cellule staminali (radioterapia, agenti intratecali, idrossiurea, inibitori della tirosin-chinasi, agenti ipometilanti, rituximab e lenalidomide non vengono considerati chemioterapia)
    4. Tossicità clinicamente significative persistenti che, secondo il parere dello sperimentatore, rendono il paziente non idoneo per il trapianto
    5. Evidenza di anticorpi anti-ALD403 specifici del donatore alla CBU n.1 per il trattamento selezionata (MFI>3000 a HLA A, B, C, o DRB1)
    6. Evidenza di infezione da HIV o sierologia positiva per HIV
    7. Evidenza di epatite B o epatite C come determinato da sierologia o PCR
    8. Gravidanza, indicata da test positivo della gonadotropina corionica umana (HCG) su siero o urine, o allattamento
    9. Tumore maligno attivo diverso da quello per cui viene eseguito il trapianto di UCB entro 12 mesi dall'arruolamento. Sarà ammesso carcinoma cutaneo a cellule squamose o carcinoma a cellule basali o carcinoma cervicale in situ completamente rescisso entro 12 mesi dall'arruolamento.
    10. Evidenza di infezioni micotiche, batteriche o virali non controllate o gravi malattie concomitanti, che, secondo il parere dello sperimentatore principale, indicano che il paziente potrebbe non tollerare il trapianto
    11. Pazienti con presenza di blasti leucemici nel sistema nervoso centrale (SNC)
    12. Pazienti con donatore non consanguineo o consanguineo subito disponibile e compatibile per HLA per un allele in misura 8/8 (le cui cellule staminali possono essere prelevate tempestivamente senza compromettere l’esito nel ricevente). I pazienti che presentano donatori consanguinei aploidentici non saranno esclusi
    13. Precedente trapianto allogenico di cellule staminali ematopoietiche
    14. Allergia ai prodotti di origine bovina, gentamicina, o a qualsiasi altro prodotto che potrebbe interferire con il trattamento
    15. Incapacità psicologica di sottoposti a trapianto di midollo osseo (BMT) con il relativo stretto isolamento o anamnesi documentata di mancata compliance medica e/o malattia psichiatrica e/o situazioni sociali che limiterebbero la compliance ai requisiti dello studio

    16. Arruolamento in un’altra sperimentazione clinica interventistica o assunzione di trattamento sperimentale entro 30 giorni prima della data prevista di randomizzazione, salvo approvazione documentata ottenuta dallo Sponsor prima della randomizzazione
    E.5 End points
    E.5.1Primary end point(s)
    Assess the time to neutrophil engraftment following transplantation.
    Neutrophil engraftment is defined as achieving an absolute neutrophil
    count (ANC) ≥ 0.5 x 10^9/L on 3 consecutive measurements on
    different days with subsequent donor chimerism (≤10% host cells by
    peripheral blood chimerism or bone marrow chimerism if peripheral
    blood chimerism is not available). The day of neutrophil engraftment is
    designated as the first of the 3 consecutive measurements and must
    occur on or before 42 days post transplant (and also prior to infusion of
    any additional stem cell product).
    Valutare il tempo all'attecchimento dei neutrofili dopo il trapianto.
    L’attecchimento dei neutrofili è definito come raggiungimento di una conta assoluta dei neutrofili (ANC) ≥ 0,5 x 109/l su 3 misurazioni consecutive in giorni diversi con seguente chimerismo del donatore (≤10% nelle cellule ospiti con chimerismo nel sangue periferico o chimerismo nel midollo osseo se non è disponibile chimerismo nel sangue periferico). Il giorno dell'attecchimento dei neutrofili è definito come la prima delle 3 misurazioni consecutive e deve avvenire 42 giorni o entro 42 giorni dopo il trapianto (e anche prima dell'infusione di eventuali altri prodotti a base di cellule staminali).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Not relevant
    Non Rilevante
    E.5.2Secondary end point(s)
    Secondary Endpoints:
    •Incidence of grade 2/3 bacterial or invasive fungal infections by 100
    days following transplantation
    •Days alive and out of hospital in the first 100 days following
    transplantation
    •Platelet engraftment by 42 days following transplantation
    Tertiary Endpoint:
    •Non-relapse mortality by 210 days following randomization
    Exploratory Endpoints:
    •Neutrophil engraftment by 16 days following transplantation
    •Time from transplantation to platelet engraftment
    •Duration of primary hospitalization
    •Non-relapse mortality by 130 days and 15 months following
    randomization
    •Overall survival at 210 days and 15 months following randomization
    •Disease-free survival at 15 months following randomization
    •Neutrophil engraftment by 42 days following transplantation
    •Acute GvHD grade II-IV and III-IV by 100 days following transplantation
    •Chronic GvHD (mild/moderate/severe) by 180 days and 1 year
    following transplantation
    •Secondary graft failure by 1 year following transplantation
    •Grade 3 viral infections by 180 days and 1 year following
    transplantation
    •Safety and tolerability of NiCord® transplantation
    •Relapse by 15 months following randomization
    •Relapse mortality by 15 months following randomization
    •Immune reconstitution at 28, 70, 100, 180, and 365 days following transplantation
    •Health-related quality of life
    End point secondario (ripetere se necessario):
    • Incidenza di infezioni batteriche o micotiche invasive di grado 2/3 entro 100 giorni dopo
    il trapianto
    • Giorni di sopravvivenza e senza ricovero nei primi 100 giorni dopo il trapianto
    • Attecchimento delle piastrine entro 42 giorni dopo il trapianto

    Endpoint terziario:
    • Mortalità non causata da recidiva entro 210 giorni dopo la randomizzazione
    • Attecchimento dei neutrofili entro 16 giorni dopo il trapianto
    • Tempo dal trapianto all'attecchimento delle piastrine
    • Durata del ricovero primario
    • Mortalità non causata da recidiva entro 130 giorni e 15 mesi dopo la randomizzazione
    • Sopravvivenza globale a 210 giorni e 15 mesi dopo la randomizzazione
    • Sopravvivenza libera da malattia a 15 mesi dopo la randomizzazione
    • Attecchimento dei neutrofili entro 42 giorni dopo il trapianto
    • GVHD acuta di grado II-IV e III-IV entro 100 giorni dopo il trapianto
    • GVHD cronica (lieve/moderata/grave) entro 180 giorni e a 1 anno dopo il trapianto
    • Fallimento del trapianto secondario entro 1 anno dopo il trapianto
    • Infezioni virali di grado 3 entro 180 giorni e a 1 anno dopo il trapianto
    • Sicurezza e tollerabilità del trapianto di NiCord®
    • Recidiva entro 15 mesi dopo la randomizzazione
    • Mortalità causata da recidiva entro 15 mesi dopo la randomizzazione
    • Immunoricostituzione a 28, 70, 100, 180 e 365 giorni dopo il trapianto
    • Qualità della vita correlata allo stato di salute
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary Endpoints
    day 100 following transplantation
    day 100 following transplantation
    day 42 following transplantation
    Tertiary Endpoint:
    day 210 following randomization
    Exploratory Endpoints
    day 16 following transplantation
    Not relevant
    Not relevant
    day 130 and 15 months following randomization
    day 210 and 15 months following randomization
    15 months following randomization
    day 42 following transplantation
    day 100 following transplantation
    day 180 and 1 year following transplantation
    1 year following transplantation
    day 180 and 1 year following transplantation
    not relevant
    15 months following randomization
    15 months following randomization
    day 28, 70, 100, 180 and 365 following transplantation
    not relevant
    Endpoints Secondari
    - Giorno 100 dopo il trapianto
    - Giorno 100 dopo il trapianto
    - Giorno 42 dopo il trapianto
    Endpoint Terziario
    - Giorno 210 dopo la randomizzazione
    Endpoints Esplorativi
    - Giorno 16 dopo il trapianto
    - Non Rilevante
    - Non Rilevante
    - Giorno 30 e 15 mesi dopo la randomizzazione
    - Giorno 210 e 15 mesi dopo la randomizzazione
    - 15 mesi dopo la randomizzazione
    - Giorno 42 dopo il trapianto
    - Giorno 100 dopo il trapianto
    - Giorno 180 e 1 anno dopo il trapianto
    - 1 anno dopo il trapianto
    - Giorno 180 e 1 anno dopo il trapianto
    - Non rilevante
    - 15 mesi dopo la randomizzazione
    - 15 mesi dopo la randomizzazione
    - Giorno 28, 70, 100, 180 e 365 dopo il trapianto
    - Non rilevante
    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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    one or two Unmanipulated Umbilical Cord Blood Unit(s)
    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 concerned2
    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 No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Italy
    Netherlands
    Portugal
    Singapore
    Spain
    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
    LVLS
    LVLS
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 30
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 30
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 80
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 50
    F.4.2.2In the whole clinical trial 120
    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)
    When subject completes the study visits he will be offered to
    participate in long term follow up study.
    Quando il soggetto completa le visite dello studio gli/le sarà offerto di partecipare allo studio di follow-up a lungo termine.
    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 Decision2018-12-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-07-24
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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