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    Summary
    EudraCT Number:2019-003997-25
    Sponsor's Protocol Code Number:MDS0519
    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:2020-10-07
    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 number2019-003997-25
    A.3Full title of the trial
    Prospective randomized study on the feasibility of allogeneic stem cell transplantation in higher-risk-myelodysplastic syndromes, performed upfront or preceded by azacitidine or conventional chemotherapy, according to the BM-blast proportion (ACROBAT trial)
    Studio randomizzato, prospettico, sulla fattibilità del trapianto allogenico di cellule staminali in pazienti affetti da Sindrome mielodisplastica ad alto rischio, eseguito upfront o preceduto da azacitidina o chemioterapia convenzionale, secondo la percentuale di blasti midollari (studio ACROBAT)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The study is aimed at evaluating the feasibility of a hypomethylating treatment combined with hematopoietic stem cell transplantation in comparison to hematopoietic stem cell transplantation in patients with high-risk myelodysplastic syndrome.
    Lo studio è volto a valutare la fattibilità di un trattamento ipometilante unito al trapianto di cellule staminali ematopoietiche in confronto al solo trapianto di cellule staminali ematopoietiche in pazienti affetti da sindrome mielodisplastica ad alto rischio.
    A.3.2Name or abbreviated title of the trial where available
    ACROBAT
    ACROBAT
    A.4.1Sponsor's protocol code numberMDS0519
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04184505
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorFONDAZIONE GIMEMA (GRUPPO ITALIANO MALATTIE EMATOLOGICHE DELL' ADULTO) FRANCO MANDELLI ONLUS
    B.1.3.4CountryItaly
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAIFA - Italian Medicines Agency
    B.4.2CountryItaly
    B.4.1Name of organisation providing supportFondazione GIMEMA (Gruppo Italiano Malattie EMatologiche dell'Adulto) Franco Mandelli Onlus
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFondazione GIMEMA
    B.5.2Functional name of contact pointCentro Dati
    B.5.3 Address:
    B.5.3.1Street Addressvia Casilina, 5 Roma
    B.5.3.2Town/ cityRoma
    B.5.3.3Post code00182
    B.5.3.4CountryItaly
    B.5.4Telephone number0670390526
    B.5.5Fax number0670390540
    B.5.6E-mailgimema@gimema.it
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Azacitidina Accord 25 mg/mL polvere per sospensione iniettabile
    D.2.1.1.2Name of the Marketing Authorisation holderAccord Healthcare S.L.U.
    D.2.1.2Country which granted the Marketing AuthorisationSwitzerland
    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 nameAzacitidina
    D.3.2Product code [NA]
    D.3.4Pharmaceutical form Powder for suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAzacitidina
    D.3.9.1CAS number 320-67-2
    D.3.9.2Current sponsor codeNA
    D.3.9.3Other descriptive nameAzacytidine
    D.3.10 Strength
    D.3.10.1Concentration unit mg/m2 milligram(s)/square meter
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number75
    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
    Higher-risk-myelodysplastic syndromes
    Sindrome mielodisplastica ad alto rischio
    E.1.1.1Medical condition in easily understood language
    Blood disease caused by damage to stem cells in the bone marrow that fail to produce adequate amounts of functional blood cells causing them to lack.
    Malattia del sangue causata dal danneggiamento di cellule staminali nel midollo osseo che non riescono a produrre una quantità adeguata di cellule del sangue funzionali provocandone carenza.
    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 21.1
    E.1.2Level PT
    E.1.2Classification code 10028533
    E.1.2Term Myelodysplastic syndrome
    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 21.1
    E.1.2Level PT
    E.1.2Classification code 10028533
    E.1.2Term Myelodysplastic syndrome
    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 primary objective of this study has been split in two categories:
    the feasibility of HSCT (ITT) in patients with HR-MDS with a proportion of bone marrow blasts below 10% and in patients with a proportion of BM blasts equal or greater than 10%.
    L’obiettivo primario di questo studio è stato separato in due categorie:
    valutare la fattibilità del trapianto (ITT, Intention To Treat) nei pazienti affetti da HR-MDS con una percentuale di blasti midollari inferiore al 10% e nei pazienti con una percentuale uguale o maggiore al 10%.
    E.2.2Secondary objectives of the trial
    1. Overall survival ITT
    2. Event-free survival ITT (including relapse, progression, or death from any cause)
    3. Safety
    4. Changes in HCT-CI at the time of HSCT as compared to the time of enrollment
    5. Pattern of relapse/progression after HSCT
    6. Translational studies with pre- and post-treatment mutational, methylation and cytofluorimetric analysis (patient BM-sampling at enrollment, before HSCT and at 6 months after HSCT)
    7. Quality of life assessment at enrollment, before HSCT and at 6 months after HSCT
    8. Pharmacoeconomic evaluation in terms of duration of hospitalization
    1. Sopravvivenza globale ITT (OS, Overall Survival)
    2. Sopravvivenza libera da eventi ITT (EFS, Event Free Survival) (inclusi ricadute, progressione o decesso per ogni causa)
    3. Sicurezza
    4. Variazioni nell’HCT-Comorbidity Index al momento del trapianto rispetto al momento dell’arruolamento
    5. Pattern di ricaduta/progressione dopo HSCT
    6. Studi Traslazionali con analisi mutazionali, metilazione e citofluorimetria pre- e post- trattamento (campione di midollo all’arruolamento, prima del trapianto e 6 mesi dopo il trapianto)
    7. Valutazione della Qualità di vita all’arruolamento, prima del trapianto e 6 mesi dopo il trapianto.
    8. Valutazione Farmacoeconomica in termini di durata di ospedalizzazione.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives

    Pharmacogenetics
    Version: 1.0
    Date: 24/10/2019
    Title: Translational Research
    Objectives: Translational studies with pre- and post-treatment mutational, methylation and cytofluorimetric analysis

    Farmacogenetica
    Versione: 1.0
    Data: 24/10/2019
    Titolo: Ricerca Traslazionale
    Obiettivi: Analisi mutazionali, metilazione e citofluorimetria pre- e post- trattamento
    E.3Principal inclusion criteria
    1. Patients with newly diagnosed higher-risk MDS, including IPSS Intermediate-2 and high, and IPSS-R intermediate to very-high
    2. Age 18-70 years
    3. Previously untreated for HR-MDS
    4. HSCT – eligible
    5. Life expectancy greater than or equal to 3 months;
    6. Signed written informed consent according to ICH/EU/GCP and national local laws
    7. Eastern Cooperative Oncology Group Performance Status Grade of 0-2
    1. Pazienti affetti da HR-MDS di nuova diagnosi, inclusi IPSS Intermediate-2/high, o IPSS-R da intermediate a very-high.
    2. Età 18-70 anni
    3. Pazienti non precedentemente trattati per HR-MDS
    4. Idoneità per HSCT
    5. Aspettativa di vita maggiore o uguale a 3 mesi;
    6. Firma del consenso informato scritto in accordo con le normative ICH/EU/GCP e le leggi nazionali
    7. Eastern Cooperative Oncology Group Performance Status Grade 0-2
    E.4Principal exclusion criteria
    1. Acute myeloid leukaemia with >20% blasts in BM or peripheral blood (PB);
    2. concurrent malignancy diagnosed in the past 12 months (with the exception of skin basalioma);
    3. severe renal, cardiac, liver or lung impairment; 4. pregnant or lactating or potentially fertile (both males and females), who have not agreed to avoid pregnancy during the trial period; Women of childbearing potential and men must agree to use effective contraception during and up to 3 months after treatment with azacitidine.
    5. HIV infection; active, uncontrolled HCV or HBV infections or liver cirrhosis;
    6. clinically relevant neurological or psychiatric diseases;
    7. hypersensitivity (known or suspected) to AZA;
    8. prior Treatments:
    a) prior investigational drugs (within 30 days);
    b) radiotherapy, chemotherapy, or cytotoxic therapy for non-MDS conditions within the previous 6 months;
    c) growth factors (EPO, G-CSF or GM-CSF) during the previous 21 days;
    d) androgenic hormones during the previous 14 days;
    e) prior transplantation or cytotoxic therapy, including azacitidine, AZA or chemotherapy, administered to treat MDS.
    1. Leucemia mieloide acuta con >20% blasti midollari o periferici;
    2. Malignità concomitante diagnosticata negli ultimi 12 mesi (ad eccezione del basalioma cutaneo);
    3. Grave compromissione renale, cardiaca, epatica o polmonare;
    4. Pazienti in gravidanza, allattamento o potenzialmente fertili (sia maschi che femmine) che non hanno accettato di prevenire la gravidanza durante il periodo di studio; Le donne potenzialmente fertili e gli uomini devono accettare l’utilizzo di una contraccezione efficace durante in trattamento con azacitidina e fino ai 3 mesi successivi.
    5. Infezione da HIV; infezioni attive, non controllate da HCV o HBV o cirrosi epatica;
    6. Patologie neurologiche o psichiatriche clinicamente rilevanti;
    7. Ipersensibilità (conosciuta o sospetta) ad azacitina;
    8. Trattamenti precedenti:
    a) farmaci sperimentali (entro 30 giorni);
    b) radioterapia, chemioterapia o terapia citotossica non per il trattamento della MDS entro i 6 mesi precedenti;
    c) fattori di crescita (EPO, G-CSF o GM-CSF) nei precedenti 21 giorni;
    d) ormoni androgeni nei precedenti 14 giorni;
    e) precedente trapianto o terapia citotossica, inclusa azacitidina o chemioterapia, somministrata per il trattamento della MDS.
    E.5 End points
    E.5.1Primary end point(s)
    Feasibility of HSCT (ITT) - Non-inferiority design.
    Primary Endpoint evaluation
    The feasibility of HSCT will be estimated in terms of proportion of patients who receive HSCT of the total number of randomized patients.
    For the primary endpoint of feasibility of HSCT, all patients who perform HSCT will be considered as "successes", and all others, as "failures".
    For the primary efficacy endpoint (feasibility of HSCT), sensitivity analyses will be performed adjusting the treatment comparison by factors which appeared to be of prognostic importance
    Fattibilità di HSCT (ITT) - Progettazione di non inferiorità.
    Valutazione dell'endpoint primario
    La fattibilità di HSCT sarà stimata in termini di proporzione di pazienti che ricevono HSCT del numero totale di pazienti randomizzati.
    Per l'endpoint primario di fattibilità dell'HSCT, tutti i pazienti che eseguono l'HSCT saranno considerati "successi" e tutti gli altri come "fallimenti".
    Per l'endpoint primario di efficacia (fattibilità dell'HSCT), verranno eseguite analisi di sensibilità regolando il confronto del trattamento in base a fattori che sembravano essere di importanza prognostica
    E.5.1.1Timepoint(s) of evaluation of this end point
    During the course of the study
    Durante tutto il corso dello studio
    E.5.2Secondary end point(s)
    - Overall survival ITT
    - Event-free survival ITT (including relapse, progression, or death from any cause)
    - Safety in terms of AE/SAEs
    - Pattern of relapse/progression after HSCT
    - Translational studies with mutational, and cytofluorimetric analysis (patient BM-sampling at enrollment, before HSCT and at 6 months after HSCT)
    - Quality of life assessment at enrollment, before HSCT and at 6 months after HSCT
    - Pharmacoeconomic evaluation (i.e. duration of hospitalization, RBC transfusions, etc).
    - Sopravvivenza globale ITT
    - ITT di sopravvivenza libera da eventi (inclusa ricaduta, progressione o morte per qualsiasi causa)
    - Sicurezza in termini di AE / SAE
    - Pattern di ricaduta / progressione dopo HSCT
    - Studi traslazionali con analisi mutazionale e citofluorimetrica (campionamento BM del paziente all'arruolamento, prima dell'HSCT e 6 mesi dopo l'HSCT)
    - Valutazione della qualità della vita al momento dell'arruolamento, prima dell'HSCT e 6 mesi dopo l'HSCT
    - Valutazione farmacoeconomica (ovvero durata del ricovero, trasfusioni di globuli rossi, ecc.).
    E.5.2.1Timepoint(s) of evaluation of this end point
    At the beginning and at the end of the study
    All'inizio e alla fine dello 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 Yes
    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 Yes
    E.6.13.1Other scope of the trial description
    Not inferiority
    Non inferiorità
    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 Yes
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo No
    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 concerned25
    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 No
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    Last Visit of the last Subject
    Ultima Visita dell'ultimo Paziente
    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 months0
    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 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 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: 137
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 137
    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 state273
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 274
    F.4.2.2In the whole clinical trial 274
    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 will continue to be followed according to the normal care activity provided by good clinical practice.
    I pazienti continueranno ad essere seguiti secondo la normale attività assistenziale prevista dalla buona pratica clinica.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Fondazione GIMEMA (Gruppo Italiano Malattie EMatologiche dell'Adulto)
    G.4.3.4Network Country Italy
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2020-06-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-02-05
    P. End of Trial
    P.End of Trial StatusOngoing
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