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    Summary
    EudraCT Number:2020-004001-32
    Sponsor's Protocol Code Number:EMN27
    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:2021-08-02
    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 number2020-004001-32
    A.3Full title of the trial
    A phase 2 study of Belantamab Mafodotin in patients with relapsed or refractory AL amyloidosis.
    Studio di fase 2 con belantamab nafodotin in pazienti con amiloidosi AL redicivata o refrattaria.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Belantamab Mafodotin in patients with relapsed or refractory AL amyloidosis
    Belantamab mafodotin in pazienti con amiloidosi AL redicivata o refrattaria.
    A.3.2Name or abbreviated title of the trial where available
    EMN27
    EMN27
    A.4.1Sponsor's protocol code numberEMN27
    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 SponsorSTICHTING EUROPEAN MYELOMA NETWORK
    B.1.3.4CountryNetherlands
    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 supportGlaxoSmithKline Research and Development Ltd
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationEuropean Myeloma Network - EMN
    B.5.2Functional name of contact pointEMN
    B.5.3 Address:
    B.5.3.1Street AddressErasmus MC, dr. Molewaterplein 40
    B.5.3.2Town/ cityRotterdam
    B.5.3.3Post code3015 CE
    B.5.3.4CountryNetherlands
    B.5.4Telephone number00447767565020
    B.5.5Fax number00447767565020
    B.5.6E-mailsarah.lonergan@emn.life
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameBelantamab mafodotin
    D.3.2Product code [GSK2857916]
    D.3.4Pharmaceutical form Concentrate and solvent for concentrate for 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.3.8INN - Proposed INNbelantamab mafodotin
    D.3.9.1CAS number 2050232-20-5
    D.3.9.2Current sponsor codebelantamab mafodotin
    D.3.9.4EV Substance CodeSUB130430
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) 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 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 Yes
    D.3.11.13.1Other medicinal product typeBelantamab mafodotin is a humanized afucosylated, maleimidocaproyl monomethyl auristatin phenylalanine (mcMMAF) conjugated IgG1 antibody
    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
    Relapsed or refractory AL amyloidosis.
    Amiloidosi AL redicivata o refrattaria.
    E.1.1.1Medical condition in easily understood language
    Amyloidosis
    Amiloidosi.
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10002022
    E.1.2Term Amyloidosis
    E.1.2System Organ Class 10021428 - Immune system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of BLMF in patients with relapsed or refractory AL amyloidosis.
    Valutare l’efficacia di BLMF in pazienti con amiloidosi AL recidivante o refrattaria.
    E.2.2Secondary objectives of the trial
    1.The safety of blmf in patients with relapsed or refractory AL amyloidosis.
    a.Rates of AEs grade 3 or higher, related to blmf therapy
    b.Rates of treatment discontinuation due to toxicity related to blmf
    c.Dose reduction due to toxicity of blmf therapy
    d.Any grade hematologic AEs
    e.Any grade non-hematologic AEs
    f.Rates of AEs of special interest

    2.Secondary efficacy evaluations:
    a.Overall hematologic response rates
    b.Organ response rates per individual organ
    c.Time to first hematologic response
    d.Time to at least a very good hematologic response
    e.Time to at least a low-dFLC response
    f.Duration of hematologic response (time from first response to
    hematologic progression, for those who achieved at least a hematologic
    PR or low-dFLC response)
    g.Time to a subsequent therapy, either due to progression or inadequate
    response
    h.Time to hematologic progression or major organ deterioration or death
    i.Overall survival
    j.Quality of life.
    1. Sicurezza di BLMF in pazienti con amiloidosi AL recidivante o refrattaria.
    a. Tassi di EA di grado >=3, correlati alla terapia con BLMF
    b. Tassi di interruzione del trattamento per tossicità correlata a BLMF
    c. Riduzione della dose per tossicità con BLMF
    d. EA ematologici di qualsiasi grado
    e. EA non ematologici di qualsiasi grado
    f. Tassi di EA di interesse speciale

    2. Valutazioni di efficacia secondarie:
    a. Tassi complessivi di risposta ematologica (CR+VGPR+dFLC ridotta+PR)
    b. Tassi di risposta organica per singolo organo
    c. Tempo alla prima risposta ematologica (CR/VGPR/dFLC ridotta/PR)
    d. Tempo ad almeno una risposta ematologica molto buona (VGPR)
    e. Tempo ad almeno una risposta con dFLC ridotta (dFLC ridotta o CR)
    f. Durata della risposta ematologica
    g. Tempo a una terapia successiva
    h. Tempo alla progressione ematologica o a un peggioramento organico maggiore o al decesso
    i. Sopravvivenza globale
    j. Qualità della vita
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Diagnosis of AL amyloidosis, confirmed by histology and typed with immunohistochemistry, immunoelectron microscopy or mass spectrometry, or if not available, for patients with biopsy confirmed amyloidosis and cardiac involvement alone, if they also have a negative PYP- or DPD-Tc99m bone scan.
    2.Previous systemic therapy for AL amyloidosis.
    3.Patients must be 18 years of age or above.
    4.ECOG performance status 0, 1 or 2.
    5.Mayo stage 1 or Mayo stage 2 or Mayo stage 3A1-3 defined as both cTnT < 0.035 ng/mL (or in place of cTnT the cTnI < 0.10 ng/mL or high sensitivity Troponin T < 54 ng/L) AND simultaneous NT-proBNP = 332 ng/L, OR EITHER above threshold, or BOTH above threshold but with NTproBNP < 8500 ng/L (stage 3A disease).
    6.Supine systolic blood pressure = 90 mmHg.
    7.Measurable disease defined by at least one of the following: a.serum FLC =2.0 mg/dL (20 mg/L) with an abnormal kappa:lambda ratio or the difference between involved and uninvolved free light chains (dFLC) =2 mg/dL (20 mg/L). b) presence of a monoclonal spike that is =0.5 g/dl.
    8.Symptomatic organ involvement (heart, kidney, liver/GI tract, peripheral nervous system).
    9. Patients must have adequate organ function
    10.Written informed consent in accordance with local and institutional guidelines.
    11.Female patients: contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. A WOCBP must have a negative highly sensitive serum-pregnancy test (as required by local regulations) 72 hours before the first dose of the study intervention.
    12. Male patients: contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Male patients are eligible to participate if they agree to the following during the intervention period and for 6 months after the last dose of study treatment to allow for clearance of any altered sperm.
    13. All toxicities related to previous treatment (defined by NCICTCAE, v. 5, corneal toxicities are defined according to the Keratopathy Visual Activity [KVA] scale), must be = Grade 1 at the time of enrolment except for alopecia.
    14. Patient must be able to understand the study procedures and agree to participate in the study by providing written informed consent.
    1. Diagnosi di amiloidosi AL, confermata istologicamente e tipizzata mediante indagine immunochimica, microscopia immunoelettronica o spettrometria di massa, oppure se non disponibile, per pazienti con amiloidosi confermata da biopsia e solo interessamento cardiaco, anche una scintigrafia con tracciante osseo PYP- o DPD-Tc99m negativa.
    2. Precedente terapia sistemica per amiloidosi AL.
    3. Età = 18 anni.
    4. Stato di performance ECOG 0, 1 o 2.
    5. Stadio Mayo 1 o stadio Mayo 2 o stadio Mayo 3A1-3 definiti come cTnT <0,035 ng/ml (oppure, al posto di cTnT, cTnI <0,10 ng/ml o Troponina ultrasensibile T <54 ng/l) E contemporaneamente NT-proBNP =332 ng/l, OPPURE UNO DEI DUE oltre la soglia, o ENTRAMBI oltre la soglia ma con TproBNP <8500 ng/l (malattia di stadio 3A)
    6. Pressione arteriosa sistolica in posizione supina =90 mmHg.
    7. Malattia misurabile definita da almeno uno dei seguenti: a. FLC =2,0 mg/dl (20 mg/l) con rapporto kappa:lambda anormale o differenza tra catene leggere libere coinvolte/non coinvolte (dFLC) =2mg/dl (20 mg/l). b. presenza di picco monoclonale =0,5 g/dl.
    8. Interessamento organico sintomatico.
    9. I pazienti devono avere una funzionalità organica adeguata.
    10. Consenso informato scritto secondo le linee guida locali e istituzionali.
    11. Pazienti di sesso femminile: l’uso di contraccettivi deve essere conforme ai regolamenti locali relativi ai metodi di contraccezione per i partecipanti agli studi clinici. Una paziente di sesso femminile è idonea a partecipare se non è in stato di gravidanza o se non allatta al seno e se si applica almeno una delle seguenti condizioni: a. Non è in età fertile (WOCBP) o b. è una WOCBP e utilizza, durante il periodo interventistico e per almeno 4 mesi dopo l’ultima dose dello studio, un metodo contraccettivo altamente efficace (tasso di fallimento <1% annuo), preferibilmente con bassa dipendenza dall’utilizzatore. La paziente accetta di non donare ovuli (cellule uovo, ovociti) a scopo di riproduzione durante questo periodo. Una WOCBP deve avere un test di gravidanza sierico altamente sensibile negativo (come richiesto dalle normative locali) 72 ore prima della prima dose dell’intervento dello studio.
    12. Pazienti di sesso maschile: l’uso di contraccettivi deve essere conforme alle normative locali relative ai metodi di contraccezione per i partecipanti agli studi clinici. I pazienti di sesso maschile sono idonei a partecipare se acconsentono a quanto segue durante il periodo di intervento e per 6 mesi dopo l’ultima dose di trattamento dello studio per permettere la pulizia di eventuale sperma alterato: a. Evitare la donazione di sperma PIÙ, uno tra: b. Astinenza da rapporti sessuali eterosessuali come stile di vita preferito e consueto (astinenza a lungo termine e costante) e accettare di proseguire l’astinenza, o c. Accettare di utilizzare un metodo contraccettivo/di barriera come descritto di seguito:
    Accettare di utilizzare un preservativo maschile, anche in seguito a intervento di vasectomia riuscito, e la partner femminile deve accettare di utilizzare un metodo contraccettivo altamente efficace con un tasso di fallimento <1% annuo durante i rapporti con una donna in età fertile non in stato di gravidanza.
    13. Tutte le tossicità correlate al trattamento precedente (NCI-CTCAE v.5) devono essere di Grado =1 al momento dell’arruolamento, tranne che nel caso dell’alopecia.
    14. Il paziente deve essere in grado di comprendere le procedure dello studio e accettare di partecipare allo studio fornendo il consenso informato scritto.
    E.4Principal exclusion criteria
    1.Presence of non-AL amyloidosis.
    2.Presence of lytic bone lesions or active myeloma with hypercalcemia, cast nephropathy, anemia due to marrow infiltration or extramedullary disease > 60% plasma cells in bone marrow.
    3.Previously untreated disease: patients who have had at least 2 cycles of therapy directed against the plasma cell clone; however, patients that have received high dose of melphalan as their only therapy are eligible for the study.
    4.Previous exposure to anti-BMCA agents
    5.Cardiac stage IIIB disease: both cTnT > 0.035 ng/mL (or in place of cTnT the cTnI > 0.10 ng/mL or high sensitivity Troponin T > 54 ng/L) AND simultaneous NT-proBNP >8500 ng/L.
    6.Known repetitive ventricular arrhythmias on 24h Holter ECG despite anti-arrhythmic treatment. Patient must not have evidence of cardiovascular risk including any of the following: a) Evidence of current clinically significant uncontrolled arrhythmias, including clinically significant ECG abnormalities such as 2nd degree (Mobitz Type II) or 3rd degree AV block. b) History of myocardial infarction, acute coronary syndromes (including unstable or uncontrolled angina), coronary angioplasty, or stenting or bypass grafting within three (3) months of screening. c) Class III or IV heart failure as defined by the NYHA classification system (1994). d) Severe uncontrolled ventricular arrhythmias, sick sinus syndrome, electrocardiographic evidence of acute ischemia or Grade 3 conduction system abnormalities (unless patient has a pacemaker). d) Uncontrolled hypertension or hypotension (i.e., supine SBP< 90 mmHg despite supportive therapy with midodrine)
    7.Significant neuropathy (Grades 3–4, or Grade 2 with pain) within 14 days prior to Cycle 1 Day 1.
    8.Pleural effusions requiring thoracentesis or ascites requiring paracentesis within 14 days prior to Cycle 1 Day 1.
    9.Ongoing corneal epithelial disease except mild changes in corneal epithelium.
    10.Current unstable liver or biliary disease defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, persistent jaundice, or cirrhosis.
    11.Active renal condition (infection, requirement for dialysis or any other condition that could affect the patient's safety) unrelated to AL amyloidosis. Patients with isolated proteinuria resulting from AL are eligible, provided they fulfil other inclusion criteria.
    12.Patient must not use contact lenses while participating in this study.
    13.Patient must not be simultaneously enrolled in any interventional clinical trial.
    14.Use of an investigational drug or approved systemic anti-myeloma therapy (including systemic steroids) within 14 days or five half-lives,
    whichever is shorter, prior to the first dose of study drug.
    15.Plasmapheresis within seven days prior to the first dose of the study treatment.
    16.Treatment with a monoclonal antibody within 30 days prior to the first dose of the study treatment
    17.Major surgery = 4 weeks prior to initiating study treatment.
    18.Evidence of active mucosal or internal bleeding
    19.Known immediate or delayed hypersensitivity reaction or idiosyncratic reactions to blmf or drugs chemically related to blmf, or any of the components of the study treatment.
    20.Active infection requiring treatment.
    21.Known HIV infection (defined as positive testing for HIV antibodies).
    22.Positive test for HBsAg, or HBcAb at screening or within 3 months prior to the first dose of study treatment.
    23.Positive test for hepatitis C antibody hepatitis C RNA at screening or within 3 months prior to the first dose of the study treatment.
    24.Invasive malignancies other than the disease under study.
    25.Any serious and/or unstable pre-existing medical, psychiatric disorder, or other conditions that could interfere with the patient's safety, informed consent or compliance to the study procedures.
    26.Patients must not be pregnant or breast-feeding
    1. Presenza di amiloidosi non AL.
    2. Presenza di lesioni osteolitiche o mieloma attivo con ipercalcemia, nefropatia associata al mieloma, anemia da infiltrazione midollare o patologia extramidollare >60% di plasmacellule nel midollo osseo.
    3. Patologia non trattata in precedenza: pazienti che hanno ricevuto almeno 2 cicli di terapia contro il clone plasmacellulare; tuttavia, i pazienti che hanno ricevuto un’alta dose di melfalan come unica terapia sono idonei per lo studio.
    4. Precedente esposizione ad agenti anti-BMCA
    5. Patologia cardiaca di stadio IIIB: cTnT >0,035 ng/ml (oppure, al posto di cTnT, cTnI >0,10 ng/ml o Troponina ultrasensibile T >54 ng/l) E contemporaneamente NT-proBNP >8500 ng/l.
    6. Aritmie ventricolari note ripetute evidenziate mediante Holter ECG delle 24 ore nonostante trattamento antiaritmico. Il paziente non deve avere evidenza di rischio cardiovascolare, compreso quanto segue: a) Evidenza di attuali aritmie non controllate clinicamente significative; b) Anamnesi di infarto del miocardio, sindromi coronariche acute (NYHA, 1994). c) Aritmia ventricolare grave non controllata, sindrome del seno malato, evidenza ECG di ischemia acuta o anomalie del sistema di conduzione cardiaco di Grado 3; d) Ipertensione o ipotensione non controllata
    7. Neuropatia significativa (Gradi 3–4 o Grado 2 con dolore) nei 14 giorni precedenti il Ciclo 1 Giorno 1.
    8. Effusione pleurica che richiede toracentesi o ascite che richiede paracentesi nei 14 giorni prima del Ciclo 1 Giorno 1.
    9. Attuale patologia epiteliale corneale, con l’eccezione di lievi variazioni dell’epitelio corneale.
    10. Attuale patologia epatica o biliare instabile definita dalla presenza di ascite, encefalopatia, coagulopatia, ipoalbuminemia, varici esofagee o gastriche, ittero persistente o cirrosi. Nota: Una patologia epatica cronica non cirrotica stabile.
    11. Patologia renale attiva non correlata all’amiloidosi AL. Pazienti con proteinuria isolata derivante da AL sono idonei, a condizione che soddisfino gli altri criteri di inclusione.
    12. Il paziente non deve usare lenti a contatto durante la partecipazione a questo studio.
    13. Il paziente non deve essere contemporaneamente arruolato in un’altra sperimentazione clinica interventistica.
    14. Uso di un farmaco sperimentale o di una terapia sistemica anti-mieloma approvata entro 14 giorni o cinque emivite, qualunque dei due periodi sia più breve, prima della prima dose di farmaco dello studio.
    15. Plasmaferesi entro sette giorni prima della prima dose di trattamento dello studio.
    16. Trattamento con un anticorpo monoclonale entro sette giorni prima della prima dose di trattamento dello studio
    17. Intervento chirurgico maggiore =4 settimane prima di iniziare il trattamento dello studio.
    18. Evidenza di emorragia della mucosa o interna attiva
    19. Reazione da ipersensibilità nota immediata o ritardata o reazioni idiosincrasiche a BLMF o a farmaci chimicamente correlati a BLMF o ad altri componenti del trattamento dello studio.
    20. Infezione attiva che richiede trattamento.
    21. Infezione da HIV nota.
    22. Test positivo per HBsAg o HBcAb allo screening o entro 3 mesi prima della prima dose di trattamento dello studio.
    23. Test per l’anticorpo dell’epatite C o test RNA epatite C positivo allo screening o entro 3 mesi prima della prima dose di trattamento dello studio. 24. Patologie maligne invasive diverse dalla patologia in studio, a meno che la seconda patologia maligna non sia stata clinicamente stabile per almeno 2 anni e, secondo lo sperimentatore principale, non influenzerà la valutazione degli effetti dei trattamenti della sperimentazione clinica della patologia maligna attualmente in oggetto.
    25. Qualsiasi patologia clinica, psichiatrica o di altro tipo grave e/o instabile preesistente che possa interferire con la sicurezza del paziente, il consenso informato o l’aderenza alle procedure dello studio.
    26. Le pazienti non devono essere in stato di gravidanza o allattare.
    E.5 End points
    E.5.1Primary end point(s)
    The primary end point of the study is the CR/VGPR/low-dFLC response rate at 6 months/ 4 cycles from start of therapy with blmf, according to consensus response criteria.
    L’endpoint primario dello studio è il tasso di CR/VGPR/risposta con dFLC ridotta a 6 mesi/4 cicli dall’inizio della terapia con BLMF, secondo i criteri di risposta stabiliti mediante consenso.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At 6 months/ 4 cycles from start of therapy with BLFM.
    A 6 mesi / 4 cicli dall'inizio del trattamento con BLMF.
    E.5.2Secondary end point(s)
    a) Evaluation of safety (grade 3 or higher adverse events rates related to blmf therapy, treatment discontinuation rates, any grade hematologic adverse events, any grade non-hematologic adverse events) and b) Further evaluation of efficacy (Overall hematologic response rates at 6 months (CR+VGPR+low-dFLC response+ PR), Organ response rates per individual organ (heart, kidney, liver) at 3, 6, 12, 18 and 24 months from start of therapy, Time to first hematologic response (PR or better), Time to at least a very good hematologic response (VGPR or better), Time to at least a low-dFLC response, Duration of hematologic response (time
    from first response to hematologic progression, for those who achieved at least a hemPR or low-dFLC response), Time to a subsequent therapy, either due to progression or inadequate response, Time to hematologic progression or major organ deterioration or death, overall survival and quality of life)); Valutazione della sicurezza (tassi di EA correlati alla terapia con BLMF di grado 3 o superiore, tassi di interruzione del trattamento, EA ematologici di qualunque grado, EA non ematologici di qualunque grado) e
    • Ulteriore valutazione dell’efficacia (tassi complessivi di risposta ematologica a 6 mesi (CR+VGPR+risposta con dFLC ridotta+PR), tassi di risposta organica per singolo organo (cuore, reni, fegato) a 3, 6, 12, 18 e 24 mesi dall’inizio della terapia, tempo alla prima risposta ematologica (PR o superiore), tempo ad almeno una risposta ematologica molto buona (VGPR o superiore), tempo ad almeno una risposta con dFLC ridotta, durata della risposta ematologica (tempo dalla prima risposta alla progressione ematologica, per coloro che raggiungono almeno una hemPR o risposta con dFLC ridotta), tempo alla terapia successiva, a causa della progressione oppure di una risposta inadeguata, tempo alla progressione ematologica o a un peggioramento organico maggiore o al decesso, sopravvivenza globale e qualità della vita).
    E.5.2.1Timepoint(s) of evaluation of this end point
    For the entire duration of the study
    Per l'intera durata 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 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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other 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 No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA5
    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
    France
    Germany
    Italy
    Netherlands
    United Kingdom
    Greece
    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 years4
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 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: 18
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 18
    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 30
    F.4.2.2In the whole clinical trial 36
    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)
    None
    Nessuno
    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 Decision2021-05-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-01-13
    P. End of Trial
    P.End of Trial StatusOngoing
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