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    Summary
    EudraCT Number:2020-003880-24
    Sponsor's Protocol Code Number:ACT16832
    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:2021-06-04
    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 number2020-003880-24
    A.3Full title of the trial
    A multicenter, open-label, non-randomized, Phase 1b/2 study to evaluate the safety, pharmacokinetics,and efficacy of subcutaneous isatuximab in adults with warm autoimmune hemolytic anemia
    Studio di Fase 1b/2 multicentrico, in aperto, non randomizzato per la valutazione di sicurezza, farmacocinetica ed efficacia di isatuximab per via sottocutanea in adulti con anemia emolitica autoimmune calda
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety, pharmacokinetics, and efficacy of subcutaneous isatuximab in adults with warm autoimmune hemolytic anemia
    Sicurezza, farmacocinetica ed efficacia di isatuximab sottocutaneo in adulti con anemia emolitica autoimmune calda
    A.3.2Name or abbreviated title of the trial where available
    .
    .
    A.4.1Sponsor's protocol code numberACT16832
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1255-5350
    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 SponsorSANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
    B.1.3.4CountryFrance
    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 supportSanofi-Aventis Recherche & Développement
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSanofi S.r.l.
    B.5.2Functional name of contact pointContact point
    B.5.3 Address:
    B.5.3.1Street AddressViale L. Bodio, 37/B
    B.5.3.2Town/ cityMilano
    B.5.3.3Post code20158
    B.5.3.4CountryItaly
    B.5.4Telephone number800226343
    B.5.5Fax number0239394168
    B.5.6E-mailinformazioni.medicoscientifiche@sanofi.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 Yes
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameMontelukast
    D.3.2Product code [.]
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMONTELUKAST
    D.3.9.2Current sponsor code.
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameIsatuximab
    D.3.2Product code [SAR650984]
    D.3.4Pharmaceutical form Solution for injection/infusion
    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 INNIsatuximab
    D.3.9.2Current sponsor codeSAR650984
    D.3.9.4EV Substance CodeSUB119676
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number140
    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.IMP: 3
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameParacetamolo
    D.3.2Product code [.]
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPARACETAMOLO
    D.3.9.2Current sponsor code.
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 4
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameFamotidina
    D.3.2Product code [.]
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNFAMOTIDINA
    D.3.9.2Current sponsor code.
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 5
    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.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameLevocitirizina
    D.3.2Product code [.]
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNLEVOCETIRIZINA DICLORIDRATO
    D.3.9.2Current sponsor code.
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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
    Warm autoimmune hemolytic anemia
    anemia emolitica autoimmune calda
    E.1.1.1Medical condition in easily understood language
    Blood and lymphatic diseases
    Malattie linfatiche e del sangue
    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 PT
    E.1.2Classification code 10047822
    E.1.2Term Warm type haemolytic anaemia
    E.1.2System Organ Class 10005329 - Blood and lymphatic 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
    • Part A: To evaluate the safety and tolerability of subcutaneous injections of isatuximab in adults with wAIHA
    • Part B: To evaluate the efficacy of the selected dose in adults with wAIHA
    • Parte A: Valutare la sicurezza e la tollerabilità delle iniezioni sottocutanee di isatuximab negli adulti con wAIHA
    • Parte B: Valutare l’efficacia della dose selezionata negli adulti con wAIHA.
    E.2.2Secondary objectives of the trial
    Part A (Cohorts 2 and 3 only)
    -To evaluate the efficacy of isatuximab in adults with wAIHA
    -To evaluate the durability of response to isatuximab and time to response
    -To evaluate the impact of isatuximab treatment on fatigue
    • Part B
    -To evaluate the safety and tolerability of isatuximab in adults with wAIHA
    -To evaluate the durability of response to isatuximab and time to response
    -To evaluate the impact of isatuximab treatment on fatigue
    • Parts A (all Cohorts) and B
    -To evaluate the effect of isatuximab on markers of hemolysis
    -To characterize the pharmacokinetic profile of isatuximab in adults with wAIHA
    -To evaluate the immunogenicity of isatuximab
    • Parte A (solo Coorti 2 e 3)
    - Valutare l’efficacia di isatuximab negli adulti con wAIHA.
    - Valutare la durata della risposta a isatuximab e il tempo alla risposta.
    - Valutare l’impatto del trattamento con isatuximab sull’affaticamento.
    • Parte B
    - Valutare la sicurezza e la tollerabilità di isatuximab negli adulti con wAIHA.
    - Valutare la durata della risposta a isatuximab e il tempo alla risposta.
    - Valutare l’impatto del trattamento con isatuximab sull’affaticamento.
    • Parti A (tutte le Coorti) e B
    - Valutare l’effetto di isatuximab sui marcatori dell’emolisi.
    - Caratterizzare il profilo farmacocinetico di isatuximab negli adulti con wAIHA.
    - Valutare l’immunogenicità di isatuximab.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Participant must be >/=18 to years of age, inclusive, at the time of signing the informed consent.
    - Males and females with a confirmed diagnosis of primary w AIHA or systemic lupus erythematosus (SLE)-associated w AIHA (without other SLE-related manifestations apart from cutaneous and musculoskeletal manifestations) who meet the following criteria:
    a) Hemoglobin level <10 g/dL at screening.
    b) Hemolysis (haptoglobin </=4 mg/dL and total bilirubin above the upper limit of normal).
    c) Positive direct antiglobulin test (DAT) (IgG or IgG + complement C3d pattern or IgM warm autoantibodies (positive dual DAT)).
    - Participants who have previously failed to maintain a sustained response after treatment with corticosteroids (corticosteroid-refractory or corticosteroid-dependent primary wAIHA).
    - Part A only: Participants who have previously failed to maintain a sustained response after treatment with rituximab (or other anti-CD20 monoclonal antibodies). The last dose of the anti-CD20 antibody must have been administered at least 12 weeks before enrollment.
    - Part B: Participants who have had an insufficient response to at least 1 prior therapy in addition to corticosteroids (splenectomy is regarded as a prior therapy).
    - Contraceptive use by men and women
    - Il/La partecipante deve avere un’età >/=18 anni, compiuti, al momento della firma del consenso informato.
    - Soggetti di sesso maschile e femminile con diagnosi confermata di wAIHA primaria o di wAIHA associata a lupus eritematoso sistemico (LES) (senza altre manifestazioni correlate al LES, a parte le manifestazioni cutanee e muscoloscheletriche) che soddisfa i seguenti criteri:
    a) Livello di emoglobina <10 g/dl allo screening.
    b) Emolisi (aptoglobina </=4 mg/dl e bilirubina totale al di sopra del limite superiore della norma).
    c) Positività al test dell’antiglobulina diretto (DAT) (IgG o IgG + complemento C3d o anticorpi anti-IgM caldi [duplice DAT positivo]).
    - I/Le partecipanti che non hanno precedentemente mantenuto una risposta sostenuta dopo il trattamento con corticosteroidi (wAIHA primaria refrattaria a corticosteroidi o corticosteroide-dipendente).
    - Solo Parte A: I/Le partecipanti che non hanno precedentemente mantenuto una risposta sostenuta dopo il trattamento con rituximab (o altri anticorpi monoclonali anti-CD20). L’ultima dose dell’anticorpo anti-CD20 deve essere stata somministrata almeno 12 settimane prima dell’arruolamento.
    - Parte B: I/Le partecipanti che hanno avuto una risposta insufficiente ad almeno 1 terapia precedente in aggiunta ai corticosteroidi (la splenectomia è considerata una terapia precedente).
    - Utilizzo di contraccettivi da parte di uomini e donne
    E.4Principal exclusion criteria
    Participants are excluded from the study if any of the following criteria apply:
    - Clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the participant or compromise the quality of the data derived from his or her participation in the study as determined by the Investigator.
    - Serious infection that required hospitalization within 3 months prior to enrollment.
    - Secondary wAIHA from any cause including drugs, lymphoproliferative disorders, infectious or autoimmune disease (SLE without other SLE-related manifestations apart from cutaneous and musculoskeletal manifestations is allowed), or active hematologic malignancies. Participants with positive antinuclear antibodies but without a definitive diagnosis of an autoimmune disease are allowed.
    - History of coagulation or bleeding disorders (Evans Syndrome is allowed).
    - Uncontrolled or active HBV or HCV infection
    - HIV infection.
    - Serum gammaglobulin levels <3 g/L.
    - Females who are pregnant, lactating, or considered unreliable with respect to contraceptive practice.
    - Concurrent treatment with corticosteroids, unless the participant has been on a stable daily dose for >/= 30 days prior to enrollment.
    - Treatment with cyclophosphamide within 4 weeks prior to enrollment.
    - Treatment with cytotoxic drugs (other than cyclophosphamide) within 12 weeks prior to enrollment.
    In presenza di uno dei seguenti criteri i partecipanti saranno esclusi dallo studio:
    - Anamnesi medica clinicamente significativa o malattia cronica in corso che comprometterebbe la sicurezza del/la partecipante o comprometterebbe la qualità dei dati derivati dalla sua partecipazione allo studio, come stabilito dallo sperimentatore.
    - Infezione seria che ha richiesto il ricovero ospedaliero nei 3 mesi precedenti l’arruolamento.
    - wAIHA secondaria per qualsiasi causa, compresi farmaci, disturbi linfoproliferativi, malattie infettive o autoimmuni (il lupus eritematoso sistemico [LES] senza altre manifestazioni correlate al LES, a parte le manifestazioni cutanee e muscoloscheletriche è consentito) o tumori maligni ematologici attivi. Sono consentiti i/le partecipanti con anticorpi antinucleari positivi, ma senza diagnosi definitiva di malattia autoimmune.
    - Anamnesi di coagulazione o disturbi emorragici (è consentita la sindrome di Evans).
    - Infezione non controllata o attiva da HBV o da HCV
    - Infezione da HIV.
    - Livelli di gammaglobulina sierica <3 g/l.
    - Donne in gravidanza, in allattamento o considerate inaffidabili in relazione alla pratica di contraccezione.
    - Trattamento concomitante con corticosteroidi, a meno che il/la partecipante non abbia assunto una dose stabile giornaliera per >/= 30 giorni prima dell’arruolamento.
    - Trattamento con ciclofosfamide nelle 4 settimane precedenti l’arruolamento.
    - Trattamento con farmaci citotossici (diversi da ciclofosfamide) nelle 12 settimane precedenti l’arruolamento.
    E.5 End points
    E.5.1Primary end point(s)
    Part A: To assess safety and tolerability
    Standard clinical and laboratory parameters and adverse events
    Part B
    -To evaluate overall response rate (R) or complete response (CR) at Day 85.
    R is defined as an increase in hemoglobin by >/=2 g/dL from baseline and an absence of transfusion in the last 7 days. Biochemical evidence of hemolysis may still be present.
    CR is defined as hemoglobin >/=11 g/dL (women) or >/=12 g/dL (men), no evidence of hemolysis (normal bilirubin, LDH, haptoglobin, and reticulocytes), and absence of transfusion from 6 weeks after the first isatuximab dose.
    Parte A: Valutare la sicurezza e la tollerabilità
    Parametri clinici e di laboratorio standard ed eventi avversi
    Parte B
    - Valutare il tasso di risposta complessiva (R) o risposta completa (CR) al Giorno 85.
    R è definito come un aumento dell’emoglobina di >/=2 g/dl dal basale e un’assenza di trasfusioni negli ultimi 7 giorni. Potrebbe essere ancora presente evidenza biochimica di emolisi.
    La CR è definita come emoglobina >/=11 g/dl (donne) o >/=12 g/dl (uomini), nessuna evidenza di emolisi (bilirubina , LDH, aptoglobina e reticolociti normali) e assenza di trasfusione a partire dalle 6 settimane dopo la prima dose di isatuximab.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Part A: Through Day 169
    Part B: Through Day 85
    Parte A: fino al Giorno 169
    Parte B: fino al Giorno 85
    E.5.2Secondary end point(s)
    Part A (Cohorts 2 and 3 Only):
    1)To evaluate overall response rate (R) or complete response (CR) at Day 85; R is defined as an increase in hemoglobin by >/=2 g/dL from baseline and an absence of transfusion in the last 7 days. Biochemical evidence of hemolysis may still be present. CR is defined as hemoglobin >/=11 g/dL (women) or >/=12 g/dL (men), no evidence of hemolysis (normal bilirubin, LDH, haptoglobin, and reticulocytes), and absence of transfusion from 6 weeks after the first isatuximab dose.
    2)Overall response rate at Day 169, time to R or CR, time to loss of R or CR, proportion of participants requiring rescue therapy (any wAIHA-directed therapy other than prednisone or transfusion) or splenectomy; Overall response rate at Day 169, time to R or CR, time to loss of R or CR (loss of R defined as hemoglobin <10 g/dL at two consecutive visits at least 7 days apart and initiation of new treatment for anemia or increase in steroid dose; loss of CR is defined as hemoglobin <11 g/dL (women) or <12 g/dL (men) at two consecutive visits at least 7 days apart), proportion of participants requiring rescue therapy (any wAIHA-directed therapy other than prednisone or transfusion) or splenectomy
    3)-FACIT-fatigue scale score
    Part B:
    4)-To assess safety and tolerability; Standard clinical and laboratory parameters and adverse events.
    5)Overall response rate at Day 169, time to R or CR, time to loss of R or CR, proportion of participants requiring rescue therapy (any wAIHA-directed therapy other than prednisone or transfusion) or splenectomy; Overall response rate at Day 169, time to R or CR, time to loss of R or CR (loss of R defined as hemoglobin <10 g/dL at two consecutive visits at least 7 days apart and initiation of new treatment for anemia or increase in steroid dose; loss of CR is defined as hemoglobin <11 g/dL (women) or <12 g/dL (men) at two consecutive visits at least 7 days apart), proportion of participants requiring rescue therapy (any wAIHA-directed therapy other than prednisone or transfusion) or splenectomy
    6)-FACIT-fatigue scale score
    Part A (All Cohorts) and B:
    7)-Change from baseline in LDH, haptoglobin, reticulocytes, and total Bilirubin
    8)-PK parameters after subcutaneous Administrations; Includes maximum concentration received after injection (Cmax) and area under the plasma concentration versus time curve calculated over the dosing interval T (336 h) (AUC0-2week)
    9)Incidence and titer (if relevant) of antiisatuximab antibodies.
    Parte A (solo Coorti 2 e 3):
    1) Tasso di risposta complessiva (R) o risposta completa (CR) al Giorno 85; R è definito come un aumento dell’emoglobina di >/=2 g/dl dal basale e un’assenza di trasfusioni negli ultimi 7 giorni. Potrebbe essere ancora presente evidenza biochimica di emolisi. La CR è definita come emoglobina >/=11 g/dl (donne) o >/=12 g/dl (uomini), nessuna evidenza di emolisi (bilirubina, LDH, aptoglobina e reticolociti normali) e assenza di trasfusione a partire dalle 6 settimane dopo la prima dose di isatuximab.
    2) Tasso di risposta complessiva al Giorno 169, tempo alla R o alla CR, tempo alla perdita di R o CR, percentuale di partecipanti che richiede terapia di salvataggio (qualsiasi terapia diretta a wAIHA diversa da prednisone o trasfusione) o splenectomia; Tasso di risposta complessiva al Giorno 169, tempo alla R o alla CR, tempo alla perdita di R o CR (perdita di R definita come emoglobina <10 g/dl in due visite consecutive ad almeno 7 giorni l’una dall’altra e inizio di nuovo trattamento per l’anemia o aumento della dose di steroidi; la perdita di CR è definita come emoglobina <11 g/dl (donne) o <12 g/dl (uomini) in due visite consecutive ad almeno 7 giorni di distanza, la percentuale di partecipanti che richiede terapia di salvataggio (qualsiasi terapia diretta a wAIHA diversa dal prednisone o trasfusione) o splenectomia.
    3) Punteggio della scala FACIT-affaticamento
    Parte B:
    4) Valutare la sicurezza e la tollerabilità; Parametri clinici e di laboratorio standard ed eventi avversi.
    5) Tasso di risposta complessiva al Giorno 169, tempo alla R o alla CR, tempo alla perdita di R o CR, percentuale di partecipanti che richiede terapia di salvataggio (qualsiasi terapia diretta a wAIHA diversa da prednisone o trasfusione) o splenectomia; Tasso di risposta complessiva al Giorno 169, tempo alla R o alla CR, tempo alla perdita di R o CR (perdita di R definita come emoglobina <10 g/dl in due visite consecutive ad almeno 7 giorni l’una dall’altra e inizio di nuovo trattamento per l’anemia o aumento della dose di steroidi; la perdita di CR è definita come emoglobina <11 g/dl (donne) o <12 g/dl (uomini) in due visite consecutive ad almeno 7 giorni di distanza, la percentuale di partecipanti che richiede terapia di salvataggio (qualsiasi terapia diretta a wAIHA diversa dal prednisone o trasfusione) o splenectomia.
    6) Punteggio della scala FACIT-affaticamento
    Parti A (tutte le Coorti) e B:
    7) Variazione rispetto al basale in LDH, aptoglobina, reticolociti e bilirubina totale.
    8) Parametri PK dopo somministrazioni sottocutanee; compresa la massima concentrazione ricevuta dopo iniezione (Cmax) e l’area sotto la curva della concentrazione plasmatica vs la curva del tempo calcolata sull’intervallo di dosaggio T (336 h) (AUC 0-2 settimane).
    9) Incidenza e titolazione (se pertinente) degli anticorpi anti-isatuximab.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Part A (Cohorts 2 and 3 Only):
    1)Through Day 85
    2)-9) Through Day 169
    Parte A (solo Coorti 2 e 3):
    1) Fino al Giorno 85
    2)-9) Fino al Giorno 169
    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 Yes
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Phase I/II study
    Studio di fase I/II
    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 Yes
    E.8.1.7.1Other trial design description
    In aperto
    Open-label
    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 EEA9
    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
    United States
    Belgium
    France
    Germany
    Hungary
    Italy
    Netherlands
    United Kingdom
    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
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days28
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months2
    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: 17
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 6
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 19
    F.4.2.2In the whole clinical trial 23
    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 Decision2021-06-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-01-12
    P. End of Trial
    P.End of Trial StatusOngoing
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