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    Summary
    EudraCT Number:2017-003652-22
    Sponsor's Protocol Code Number:INCB50465-206
    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:2021-01-15
    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 number2017-003652-22
    A.3Full title of the trial
    A Phase 2, Open-Label Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia
    Studio di fase II, in aperto, di INCB050465 in soggetti con anemia emolitica autoimmune
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2, Open-Label Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia (AIHA)
    Studio di INCB050465 in pazienti con anemia emolitica autoimmune
    A.3.2Name or abbreviated title of the trial where available
    A Phase 2, Open-Label Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia (AIHA)
    Studio di fase II, in aperto, di INCB050465 in soggetti con anemia emolitica autoimmune (AIHA)
    A.4.1Sponsor's protocol code numberINCB50465-206
    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 SponsorINCYTE CORPORATION
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportIncyte Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIncyte Corporation
    B.5.2Functional name of contact pointMedical Information
    B.5.3 Address:
    B.5.3.1Street Address1801 Augustine Cut-Off
    B.5.3.2Town/ cityWilmington
    B.5.3.3Post codeDE 19803
    B.5.3.4CountryUnited States
    B.5.4Telephone number0013024985663
    B.5.5Fax number0013024252734
    B.5.6E-mailglobalmedinfo@incyte.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameINCB050465 2.5 mg
    D.3.2Product code [INCB050465 2.5 mg]
    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.3.9.2Current sponsor codeINCB050465
    D.3.9.4EV Substance CodeSUB183790
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3
    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 nameINCB050465 1 mg
    D.3.2Product code [INCB050465 1 mg]
    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.3.9.2Current sponsor codeINCB050465
    D.3.9.4EV Substance CodeSUB183790
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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
    Autoimmune Hemolytic Anemia (AIHA), immunohemolytic anemia, autoimmune hemolytic anemia, immune complex hemolytic anemia.
    Warm AIHA, Cold AIHA
    Anemia Emolitica Autoimmune (AIHA), anemia immunoemolitica, anemia emolitica autoimmune, anemia emolitica del complesso immunitario. AIHA calda, AIHA fredda
    E.1.1.1Medical condition in easily understood language
    Autoimmune Hemolytic Anemia (AIHA)
    Anemia Emolitica Autoimmune (AIHA)
    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 LLT
    E.1.2Classification code 10003825
    E.1.2Term Autoimmune hemolytic anemia
    E.1.2System Organ Class 100000004851
    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 INCB050465 in the treatment of participants with AIHA.
    • Proportion of participants attaining a CR (defined as hemoglobin > 12 g/dL not attributed to transfusion effect and the normalization of hemolytic markers) at any visit from Week 6 to Week 12.
    • Proportion of participants attaining a PR (defined as hemoglobin 10-12 g/dL or = 2 g/dL increase from baseline not attributed to transfusion effect and the normalization of hemolytic markers) at any visit from Week 6 to Week 12.

    To evaluate the safety of INCB050465 administered as repeat doses in participants with AIHA.
    • Safety and tolerability will be assessed by monitoring AEs, measuring vital signs and ECGs, and conducting clinical laboratory blood and urine sample assessments.
    Valutare l’efficacia di INCB050465 nel trattamento dei partecipanti con AIHA.
    • Proporzione di partecipanti che otterranno una risposta completa (CR) (definita come segue: emoglobina > 12 g/dL non attribuibile all’effetto di trasfusioni e la normalizzazione dei marker emolitici) in qualsiasi visita dalla Settimana 6 alla Settimana 12.
    • Proporzione di partecipanti che otterranno una risposta parziale (PR) (definita come segue: emoglobina 10-12 g/dL o aumento di = 2 g/dL dalla baseline non attribuibile all’effetto di trasfusioni e la normalizzazione dei marker emolitici) in qualsiasi visita dalla Settimana 6 alla Settimana 12.

    Valutare la sicurezza di INCB050465 somministrato in dosaggi ripetuti in soggetti con AIHA.
    • La sicurezza e la tollerabilità verranno valutate monitorando gli eventi avversi (AE), misurando i segni vitali e con ECG, conducendo valutazioni in laboratorio su campioni di sangue e di urine.
    E.2.2Secondary objectives of the trial
    To further evaluate the efficacy of INCB050465 in the treatment of participants with AIHA.
    • Proportion of participants attaining a CR during postbaseline visits.
    • Proportion of participants attaining a PR during postbaseline visits.
    • Proportion of participants attaining a = 2 g/dL increase in hemoglobin from baseline.
    • Mean, change, and percentage change from baseline of hemoglobin.
    • Proportion of participants requiring transfusions.
    • Proportion of participants who achieve normalization of hemolytic markers. (Hemolysis markers: hemoglobin, haptoglobin, LDH, reticulocyte count, total bilirubin, and direct/indirect bilirubin.)
    • Change of daily usage of prednisone.
    • FACIT-F sub-scale assessment.
    • Proporzione dei partecipanti che otterranno una risposta completa (CR) durante le visite post baseline.
    • Proporzione dei partecipanti che otterranno una risposta parziale (PR) durante le visite post baseline.
    • Proporzione dei partecipanti che otterranno un aumento = 2 g/dL dell’emoglobina, rispetto alla baseline.
    • Variazione media e variazione della percentuale dalla baseline dell’emoglobina.
    • Proporzione di partecipanti che necessitano trasfusioni.
    • Proporzione di partecipanti che raggiunge una normalizzazione dei marker emolitici. (Marker dell’emolisi: emoglobina, aptoglobina, LDH, conto reticolocitario, bilirubina totale, bilirubina diretta e indiretta).
    • Variazione dell’utilizzo giornaliero del prednisone.
    • Valutazione dei sottoscala del questionario FACIT-F.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    A participant who meets all of the following criteria may be included in the study:
    1. Men or women, aged 18 years or older.
    2. Diagnosis of AIHA based on the presence of hemolytic anemia and serological evidence of anti-erythrocyte antibodies, detectable by the DAT as follows:
    a. Warm: DAT positive for IgG only or IgG plus C3d
    b. Cold (CAD): DAT positive for C3d only, with cold agglutinins of I specificity
    c. Mixed: DAT positive for IgG and C3d, with coexistence of warm autoantibodies and high titer cold agglutinins
    3. Participants who have disease progression after treatment with standard therapies that are known to confer clinical benefit, or who are intolerant to treatment. There is no limit to the number of prior treatment regimens.
    4. Hemoglobin 7 to 10 g/dL.
    5. No evidence of a lymphoproliferative malignancy or other autoimmune-related underlying conditions (eg, systemic lupus erythematosus, Castleman's disease, Sjögren's syndrome, or other autoimmune diseases).
    6. ECOG performance status score of 0 to 2.
    7. Willingness to avoid pregnancy or fathering children based on the criteria below:
    a. Woman of nonchildbearing potential (ie, surgically sterile with a hysterectomy and/or bilateral oophorectomy OR postmenopausal, defined by last menstrual period > 12 months before screening and confirmed by FSH).
    b. Woman of childbearing potential who has a negative serum pregnancy test at screening and who agrees to take appropriate precautions to avoid pregnancy (with at least 99% certainty) from screening through at least 93 days after the last dose of study drug. Permitted methods that are at least 99% effective in preventing pregnancy should be communicated to the participant and her understanding confirmed.
    c. Man who agrees to take appropriate precautions to avoid fathering children (with at least 99% certainty) from screening through at least 93 days after the last dose of study drug. Permitted methods that are at least 99% effective in preventing pregnancy should be communicated to the participant and his understanding confirmed.
    8. Able to comprehend and willing to sign an ICF.
    9. Willingness to receive PJP prophylaxis during the study period.

    Potrà essere incluso nello studio chiunque presenti i seguenti requisiti:
    1. Uomo o donna di età uguale o superiore a 18 anni.
    2. Diagnosi di AIHA, basata sulla presenza di anemia emolitica e di evidenze sierologiche di anticorpi anti-eritrocitari, rilevabili mediante DAT, come segue:
    a. Calda: DAT positiva solo per IgG o per IgG e C3d.
    b. Fredda (CAD): DAT positiva solo per C3d, con agglutinine a freddo di specificità I.
    c. Mista: DAT positiva per IgG e C3d, con coesistenza di anticorpi a caldo e alta concentrazione di agglutinine a freddo.
    3. Partecipanti che presentano una progressione della malattia dopo il trattamento con terapie standard che sono note per il beneficio apportato, o che risultano intolleranti al trattamento. Non esiste un limite ai regimi di trattamento precedenti.
    4. Emoglobina da 7 a 10 g/dL.
    5. Partecipanti che non presentano evidenze di una neoplasia linfoproliferativa o di altre patologie sottostanti di tipo autoimmune (ad es. lupus eritematoso sistemico, malattia di Castleman, sindrome di Sjögren o altre malattie autoimmuni).
    6. Punteggio sulla scala ECOG da 0 a 2.
    7. Desiderio di non intraprendere una gravidanza o una paternità, in base ai criteri seguenti.
    a. Donne potenzialmente non fertili (ad es. donne chirurgicamente sterili per isterectomia e/o ooforectomia O post menopausa, definita con ultima mestruazione > 12 mesi prima dello screening e confermata da FSH).
    b. Donne potenzialmente fertili che sono risultate negative al test di gravidanza nel siero al momento dello screening e che sono disponibili a prendere precauzioni per evitare la gravidanza (precauzioni efficaci almeno al 99%), dallo screening in poi e per almeno i 93 giorni seguenti l’ultima somministrazione del farmaco. I sopracitati metodi efficaci almeno al 99% nella prevenzione della gravidanza dovranno essere comunicati alla partecipante che dovrà dar prova della propria comprensione.
    c. Uomini disponibili a prendere precauzioni per evitare di concepire figli (precauzioni efficaci almeno al 99%), dallo screening in poi e per almeno i 93 giorni seguenti l’ultima somministrazione del farmaco. I sopracitati metodi efficaci almeno al 99% nella prevenzione della gravidanza dovranno essere comunicati al partecipante che dovrà dar prova della propria comprensione.
    8. Capacità di comprendere e di firmare un modulo di consenso informato.
    9. Disponibilità a ricevere la profilassi per la PJP durante il periodo dello studio.
    E.4Principal exclusion criteria
    1.Pregnant or breastfeeding women
    2.Concurrent conditions and history of other diseases:
    a.History or clinical manifes of significant unstable metabolic,hepatic,renal,hematological,pulmonary,cardiovas,gastroint,urolog,neurolog or psychiatric disorders
    b.Current or previous malignancy within 5years of study entry,except basal or squamous cell skin cancer with removal considered to be curative,superficial bladder cancer,prostate intraepithel neoplasm,carcinoma in situ of the cervix,or other noninvasive or indolent malignancy without sponsor approval
    c.Clinic significant cardiac disease,including unstable angina,acute myocardial infarction,and or cardiac conduction issues within 6months of the date of study drug administration
    d.Current NewYorkHeartAssociationClass II to IV congestive heart failure or uncontrolled arrhythmia
    3.Inadequate hematologic function defined:
    a.ANC <1.5×109/L
    b.Platelet count <100 ×109/L
    4.Severely impaired liver function(Child-Pugh Class C)or ALTorAST >= 2×ULN on repeated assessment
    5.Impaired renal function with estimated creatinine clearance less than45mL/min
    6.Anti-phospholipid antibodies positive or elevated anti-streptolysin antibodies
    7.Positive serology test results for hepatitis B surface antibody or core antibody, or hepatitis C virus antibody with detectable RNA at screening, consistent with active or chronic infection
    8.Known HIV infection or positivity on immunoassay. Note: HIV screening test is optional for participants enrolled in the United States, but participants with known HIV infection enrolled in the United States will be excluded
    9.History or presence of an abnormal ECG, that in the investigator’s opinion is clinically meaningful. Participants with screening QTc interval> 470 milliseconds for males and > 480 milliseconds for females (corrected by Fridericia) are excluded. In the event that a single QTcF is >470 milliseconds for males or >480 milliseconds for females, the participant may enroll if the average QTcF for 3 ECGs is <470 milliseconds for males and <480 milliseconds for females
    10.Use of the following medications:
    a.Treatment with rituximab within 3 months of the baseline visit
    b.Use of immunosuppressive therapy within 28 days of the baseline visit. Immunosuppressive therapy includes,but is not limited to, cyclosporine A, tacrolimus, or high-dose corticosteroids. Participants receiving corticosteroids must be at a dose level min= 20mg/day(prednisone or equivalent corticosteroid dose)within 14days of the baseline visit
    c.Chronic or current active infectious disease requiring systemic antibiotics,antifungal,or antiviral treatment or exposure to a live vaccine within 28 days of the baselineV
    d.Use or expected use during the study of any prohibited medications, including potent CYP3A4 inhibitors or inducers within 14 days or 5 half-lives (whichever is longer)before the baselineV.
    e.Current treatment or treatment within 30days or 5half-lives (whichever is longer) before the baselineV with another investigat medication,or current enrollment in another investigational drug protocol
    f.Use of any prohibited medications within 14days or 5half-lives(whichever is longer)before the baselineV
    11.Known hypersensitivity or severe reaction to INCB050465 or its excipients
    12.Unable to swallow oral medication,malabsorption syndrome,disease significantly affecting gastroint function,total resection of the stomach or small bowel, ulcerat colitis,symptomatic inflamm bowel disease,or partial or complete bowel obstruction
    13.Current alcohol or drug use that will interfere with the participant's ability to comply with the dose regimen and study evaluations
    14.Participants who are unable or unlikely to comply with the dose regimen and study evaluations
    15.Any condition that would interfere with full participation in the study, including adminis of study drug and attending required study visits;pose a significant risk to the participant;or interfere with interpretation of study data
    1.Donne incinte o allattam
    2.Condiz concorrenti e preced malatt
    a.Preced malat o manifestaz clin metab significativ instabile o malat epat,ren,emat,polmon,cardiov,gastroint,urolog,neurolog o psichiatr
    b.Patol maligna presente o passata(5 anni precedenti iniziostudio)eccezione per cancro pelle basale o squamoso,con rimoz curativa,cancro vescica superfic,neoplasia prostatica intraepiteliale,carcinoma in situ cervice o altre patol maligne non invasive o indolenti,senza l’approvaz sponsor
    c.Malat cardiov clinicam significative,tra cui angina instabile,infarto miocardico acuto e/o patologie a carico sist conduz del cuore nei 6 mesi preced data della somministr farmaco studio
    d.Insuffic cardiaca congestizia classe II alla IV secondo classif attuale della NewYorkHeartAssociation o aritmia incontroll
    3.Funz ematiche inadeguate,definite come segue(analisi del lab locale):
    a.ANC<1.5×109/L.
    b.Conta piastr<100×109/L
    4.Funz epat seriam comprom(Classe C classificaz di Child-Pugh)o ALT o ASTmag =2×ULN in ripetute valutaz
    5.Funz ren compromessa con clearance della creatinina stimato infer a 4mL/min
    6.Positiv ad Ab antifosfolipidi o elevati Ab antistreptolisina
    7.Infezione da ep B(HBV) o ep C(HCV): i partecipanti + per anticorpo di sup dell’epB o per l’antic anti-core dell’epB saranno idonei se sono neg per HBV-DNA; per tali partecip andrebbe considerata una terapia di profilassi antivirale. I partecip + per l'antic anti-HCV saranno idonei se sono neg per HCV-RNA.
    8.Infez da HIV conclam o risultato positivo al dosaggio immunolog. il test screening HIV è facoltat per partecip statunitensi; i partecip con HIV conclam arruol negli USA verranno esclusi.
    9.Anome nell’elettrocardiogramma,passate o presenti,che lo Sperim valuti come clinicam signific.Esclusi i partecip con interv QTc di screening>470millisec per gli uomini e >480 millisec per le donne (con correz di Fridericia). Nel caso in cui un singolo QTcF sia>470 millisec per gli uomini o>480 millisec per le donne, i partecip potranno essere arruol se il QTcF medio in 3ECG è<470 millisec per gli uomini e < 480 millisec per le donne.
    10.Uso dei seguenti farmaci:
    a.Trattam con rituximab 3 mesi preced Vbasale.
    b.Terapia immunosopp nei 28 giorni preced visita basale.Terapia immunosopp include ma non si limita a:ciclosporina A,tacrolimus o corticosteroidi ad alto dosaggio.I partecipanti che assumono corticoster devono rientrare nel dosaggio giorn min=20mg/die(prednisone o dosaggio equiv di corticost) nei 14 giorni preced la Vbasale
    c.Malat infett croniche o ricorrenti che richiedano antibiot sistemici,antimicotici o trattam antivirali o esposiz a vaccini con microrganismi vivi entro i 28gg preced la Vbasale.
    d.Utilizzo presente o previsto di farmaci proibiti,inclusi potenti inibitori o induttori del CYP3A4,nei 14gg o nelle 5 emivite (il periodo che risulti più lungo)precedenti la Vbasale.
    e.Trat in corso o tratt avvenuto nei 30gg o 5 emivite preced (il periodo che risulti più lungo) la visita basale, con altri farmaci oggetto di studio, o arruolamento in altri protocolli di studio farmacologico.
    f.Uso di farmaci proibiti nei 14gg o 5 emivite precedenti(il periodo che risulti più lungo)la Vbasale
    11.Ipersensitività nota o grave reaz a INCB050465 o ai suoi eccipienti
    12.Incapacità di assumere il farmaco per via orale, sindr da malass,malattie che interessano in maniera significativa le funz gastrointe, resez gastrica o resez intestino tenue,colite ulcerosa,malat infiamm croniche intesti, ostruz intest completa o parziale
    13.Uso alcool o droghe che interferirebbero con abilità di attenersi al dosaggio giornaliero e alle valutaz clinic
    14.Partecipanti ch non sono in grado di attenersi al dosag giornaliero e alle valutaz clin o che è improbabile che vi si attengano
    15.Qualsiasi condiz che:potrebbe interferire con piena partecipaz allo studio,inclusa la somm farmaco studio e le visite richieste;potrebbe rappresentare un rischio per partecipante;potrebbe interferire con l’interpretaz dei dati
    E.5 End points
    E.5.1Primary end point(s)
    • Proportion of participants attaining a CR (defined as hemoglobin >12 g/dL not attributed to transfusion effect and the normalization of hemolytic markers) at any visit from Week 6 to Week 12.
    • Proportion of participants attaining a PR (defined as hemoglobin 10-12 g/dL or at least = 2 g/dL increase from baseline not attributed to transfusion effect and the normalization of hemolytic markers) at any visit from Week 6 to Week 12.
    • Proporzione di partecipanti che otterranno una risposta completa (CR) (definita come segue: emoglobina > 12 g/dL non attribuibile all’effetto di trasfusioni e la normalizzazione dei marker emolitici) in qualsiasi visita dalla Settimana 6 alla Settimana 12.
    • Proporzione di partecipanti che otterranno una risposta parziale (PR) (definita come segue: emoglobina 10-12 g/dL o aumento di = 2 g/dL dalla baseline non attribuibile all’effetto di trasfusioni e la normalizzazione dei marker emolitici) in qualsiasi visita dalla Settimana 6 alla Settimana 12.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Efficacy: from week 6 to week 12
    Safety: throughout the study
    Efficacia: dalla settimana 6 alla settimana 12
    Sicurezza: per tutto lo studio
    E.5.2Secondary end point(s)
    • Proportion of participants attaining a CR during postbaseline visits.
    • Proportion of participants attaining a PR during postbaseline visits.
    • Proportion of participants attaining a = 2 g/dL increase in hemoglobin
    from baseline.
    • Mean, change, and percentage change from baseline of hemoglobin.
    • Proportion of participants requiring transfusions.
    • Proportion of participants who achieve normalization of hemolytic
    markers. (Hemolysis markers: hemoglobin, haptoglobin, LDH,
    reticulocyte count, total bilirubin, and direct/indirect bilirubin.)
    • Change of daily usage of prednisone.
    • FACIT-F sub-scale assessment.
    • Proporzione dei partecipanti che otterranno una risposta completa (CR) durante le visite post baseline.
    • Proporzione dei partecipanti che otterranno una risposta parziale (PR) durante le visite post baseline.
    • Proporzione dei partecipanti che otterranno un aumento = 2 g/dL dell’emoglobina, rispetto alla baseline.
    • Variazione media e variazione della percentuale dalla baseline dell’emoglobina.
    • Proporzione di partecipanti che necessitano trasfusioni.
    • Proporzione di partecipanti che raggiunge una normalizzazione dei marker emolitici. (Marker dell’emolisi: emoglobina, aptoglobina, LDH, conto reticolocitario, bilirubina totale, bilirubina diretta e indiretta).
    • Variazione dell’utilizzo giornaliero del prednisone.
    • Valutazione dei sottoscala del questionario FACIT-F.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Efficacy: Throughout the study
    PK: Week 1: predose; week 2 and week 8: predose, at 1h +- 10min, at 2h
    +- 10min and at 4h +- 30 min; week 12: predose
    PD: DAT for IgG and C3b and cold agglutinin levels: at screening and
    week 12; reticulocyte count, haptoglobin, total bilirubin, direct/indirect
    bilirubin, and LDH: at screening, day 1, weeks 1, 2, 4, 6, 8 and 10;
    complement assessment (CH50, C3, and C4): at day 1 and week 12
    Efficacia: per tutto lo studio
    PK: settimana 1: predose; settimana 2 e 8: predose, a 1 ora +/- 10 minuti, a 2 ore +/- 10 min e a 4 ore +/- 30 minuti; settimana 12: predose
    PD: DAT per IgG e C3b e livelli di agglutinina a freddo: allo screening e alla settimana 12; conta reticolocitaria, aptoglobina,bilirubina totale,bilirubina diretta/indiretta, e LDH: allo screening, al giorno 1, alla settimana 1,2,4,6,8 e 10; valutazione del complemento (CH50, c3 e C4): al giorno 1 e alla settimana 12
    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) 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 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA8
    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
    Austria
    France
    Italy
    United States
    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
    The end of the study will occur when all participants have completed all applicable follow-up assessments.
    La fine dello studio avverrà quando tutti i partecipanti avranno completato tutte le
    valutazioni di follow-up applicabili.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months3
    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: 8
    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 state21
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 23
    F.4.2.2In the whole clinical trial 25
    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)
    standard of care for treatment of AIHA
    standard di cura per il trattamento della AIHA
    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 Decision2018-09-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-07-17
    P. End of Trial
    P.End of Trial StatusOngoing
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