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    Summary
    EudraCT Number:2021-002844-66
    Sponsor's Protocol Code Number:INCB50465-309
    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:2021-09-23
    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 number2021-002844-66
    A.3Full title of the trial
    A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of Parsaclisib in Participants with Primary Warm Autoimmune Hemolytic Anemia
    Studio di fase 3, randomizzato, in doppio cieco, controllato con placebo, sull’efficacia e sicurezza di Parsaclisib in partecipanti con anemia emolitica autoimmune calda primaria
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of the Efficacy and Safety of Parsaclisib in Participants with Primary Warm Autoimmune Hemolytic Anemia
    Studio sull’efficacia e sicurezza di Parsaclisib in partecipanti con anemia emolitica autoimmune calda primaria
    A.3.2Name or abbreviated title of the trial where available
    NA
    NA
    A.4.1Sponsor's protocol code numberINCB50465-309
    A.5.4Other Identifiers
    Name:INDNumber:147,208
    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.5.6E-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 nameParsaclisib (1 mg)
    D.3.2Product code [INCB050465]
    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.3.9.3Other descriptive nameINCB050465 HYDROCHLORIDE
    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.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 nameParsaclisib (2.5 mg)
    D.3.2Product code [INCB050465]
    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.3.9.3Other descriptive nameINCB050465 HYDROCHLORIDE
    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 number2
    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: 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 nametrimethoprim-sulfamethoxazole
    D.3.2Product code [trimethoprim-sulfamethoxazole]
    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 INNTRIMETOPRIM + SULFAMETOSSAZOLO *
    D.3.9.1CAS number 8064-90-2
    D.3.9.2Current sponsor codeNA
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number160
    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 namepentamidine
    D.3.2Product code [pentamidine]
    D.3.4Pharmaceutical form Powder for nebuliser solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPInhalation use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPENTAMIDINA ISETIONATO
    D.3.9.1CAS number 140-64-7
    D.3.9.2Current sponsor codeNA
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number300
    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 nameatovaquone
    D.3.2Product code [atovaquone]
    D.3.4Pharmaceutical form Oral suspension
    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 INNATOVAQUONE
    D.3.9.1CAS number 95233-18-4
    D.3.9.2Current sponsor codeNA
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Primary Warm Autoimmune Hemolytic Anemia
    Anemia emolitica autoimmune calda primaria
    E.1.1.1Medical condition in easily understood language
    Autoimmune disorder characterized by the premature destruction of healthy red blood cells
    Disturbo autoimmune caratterizzato dalla distruzione prematura dei globuli rossi sani
    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 parsaclisib in the treatment of participants with wAIHA.
    Valutare l’efficacia di parsaclisib nel trattamento di partecipanti affetti da wAIHA.
    E.2.2Secondary objectives of the trial
    Key secondary objective:
    •To further evaluate parsaclisib in the treatment of participants with wAIHA.
    Other secondary objectives:
    •To further evaluate the efficacy of parsaclisib in the treatment of participants with wAIHA.
    •To evaluate the safety and tolerability of parsaclisib in participants with wAIHA.
    Exploratory objectives:
    •To further evaluate the efficacy of parsaclisib.
    •To evaluate the participant's quality of life and other PROs.
    •To characterize serum biomarkers and/or leukocyte profiles in participants with wAIHA treated with parsaclisib.
    •To evaluate PK of parsaclisib in participants with wAIHA.
    Obiettivo secondario principale:
    •Valutare ulteriormente parsaclisib nel trattamento di partecipanti affetti da wAIHA.
    Altri obiettivi secondari:
    •Valutare ulteriormente l'efficacia di parsaclisib nel trattamento di partecipanti affetti da wAIHA
    •Valutare la sicurezza e la tollerabilità di parsaclisib nel trattamento di partecipanti affetti da wAIHA
    Obiettivi esplorativi:
    •Valutare ulteriormente l'efficacia di parsaclisib.
    •Valutare la qualità della vita del partecipante e altri PRO.
    •Caratterizzare i biomarcatori sierici e/o i profili leucocitari in partecipanti affetti da wAIHA trattati con parsaclisib.
    •Valutare la farmacocinetica di parsaclisib nei partecipanti affetti da wAIHA.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives

    Life quality
    Version: 1
    Date: 27/05/2021
    Title: Qualitative Exit Interview sub-study
    Objectives: Data collected will be used to illustrate how improvements in fatigue due to AIHA have an effect of the participant's life and treatment satisfaction to generate evidence of perceived meaningful improvement in fatigue symptoms or impacts.

    Qualita' della vita
    Versione: 1
    Data: 27/05/2021
    Titolo: Qualitative Exit Interview sub-study
    Obiettivi: I dati raccolti verranno utilizzati per illustrare come i miglioramenti della fatica causata dall'AIHA abbiano un effetto sulla vita del partecipante e sulla soddisfazione del trattamento per generare prove di un miglioramento significativo percepito nei sintomi o negli impatti della fatica.
    E.3Principal inclusion criteria
    1. Ability to comprehend and willingness to sign a written ICF for the study.
    2. Men or women, age = 18 years at the time of signing the ICF.
    3. Diagnosis of primary wAIHA based on the presence of hemolytic anemia and serological evidence of anti-erythrocyte antibodies, detectable by the DAT positive for IgG only or IgG plus C3d.
    Note: Prior documentation of DAT testing is permitted.
    4. Participants who were inadequately controlled with, were intolerant to, or have a contraindication to other therapies. There is no limit to the number of prior treatment regimens.
    5. Hemoglobin = 7 to < 10 g/dL with symptoms of anemia as assessed by the investigator at screening (Hgb as determined by local laboratory).
    6. FACIT-F score = 43 at screening.
    7. Willingness to avoid pregnancy or fathering children based on the criteria below.
    a. Male participants with reproductive potential must agree to take appropriate precautions to avoid fathering children (with 99% certainty) from screening through 90 days after the last dose of study drug and must refrain from donating sperm during this period. Permitted methods in that are at least 99% effective preventing
    pregnancy should be communicated to the participants and their understanding confirmed.
    b. Female participants who are WOCBP must have a negative serum pregnancy test at screening and a negative urine pregnancy test before the first dose on Day 1 and must agree to take appropriate precautions to avoid pregnancy (with 99% certainty) from screening through 90 days after the last dose of study drug and must refrain from donating oocytes during this period. Permitted methods in that are at least 99% effective preventing pregnancy should be communicated to the participants and their understanding confirmed.
    b. A female participant not considered to be of childbearing potential is eligible.
    Note: This criterion does not apply to women of nonchildbearing potential (i.e., surgically sterile with a hysterectomy and/or bilateral oophorectomy OR postmenopausal, defined as amenorrhea at least 12 months before screening, confirmed by FSH levels at screening).
    8. Willingness to receive PJP prophylaxis during the study period from Day 1 through at least 2 to 6 months after the last dose of study drug.
    1. Capacità di comprendere e volontà di firmare un modulo di consenso informato scritto per lo studio.
    2. Uomini o donne di età = 18 anni al momento in cui viene firmato il consenso informato.
    Nota: per il Giappone, uomini e donne di età = 20 anni.
    3. Diagnosi di wAIHA primaria, basata sulla presenza di anemia emolitica e sull’evidenza sierologica di anticorpi anti-eritrociti, rilevabile mediante DAT positivo per le sole IgG o per le IgG+C3d.
    Nota: È ammessa eventuale documentazione precedente riguardante l’analisi DAT.
    4. Aver manifestato un controllo inadeguato della malattia con altre terapie, essere intolleranti o avere controindicazioni alle stesse. Non vi è un limite al numero di regimi di trattamento precedenti.
    5. Valori di emoglobina = 7 e < 10 g/dl con anemia sintomatica rilevata dallo sperimentatore allo screening (Hgb determinato da un laboratorio locale).
    6. Un punteggio FACIT-F = 43 allo screening.
    7. Essere disposti ad adottare misure contraccettive in base ai criteri indicati di seguito.
    a. I partecipanti di sesso maschile in età fertile devono acconsentire ad adottare misure contraccettive appropriate (con almeno il 99% di efficacia) per evitare un concepimento, dallo screening fino a 90 giorni dopo l’ultima dose del farmaco in studio, e devono astenersi dal donare lo sperma durante questo periodo. Le misure contraccettive consentite, ovvero con almeno il 99% di efficacia nella prevenzione di una gravidanza (vedere l’Appendice A), devono essere comunicate ai partecipanti, i quali devono confermare di averle comprese.
    b. Le partecipanti di sesso femminile in età fertile devono presentare un test di gravidanza sul siero negativo allo screening e un test di gravidanza sulle urine negativo prima di ricevere la prima dose il Giorno 1, devono acconsentire ad adottare le misure contraccettive adeguate (con il 99% di efficacia) per evitare una gravidanza, a partire dallo screening e fino a 90 giorni dopo l’ultima dose del farmaco in studio, e devono astenersi dal donare gli ovociti durante questo periodo. Le misure contraccettive approvate, con almeno il 99% di efficacia nella prevenzione di una gravidanza (vedere l’Appendice A), devono essere comunicate alle partecipanti, i quali devono confermare di averle comprese.
    b. Sono idonee le partecipanti di sesso femminile non considerate fertili secondo le definizioni contenute nell’Appendice A.
    Nota: tale criterio non si applica alle donne non fertili (ovvero chirurgicamente sterili a seguito di isterectomia e/o ovariectomia bilaterale OPPURE in postmenopausa, definita come almeno 12 mesi di amenorrea prima dello screening, confermata dai livelli di FSH allo screening).
    8. Disponibilità a ricevere la profilassi per PJP durante il periodo dello studio, dal Giorno 1 fino ad almeno 2-6 mesi dopo l’ultima dose del farmaco in studio.
    E.4Principal exclusion criteria
    1. Women currently pregnant or breastfeeding or participants expecting to conceive or father children within the projected duration of the study, starting with the screening visit through 90 days from the date of last dose of study drug.
    2. A diagnosis of other types of AIHA; CAD, cold agglutinin syndrome, mixed-type AIHA or paroxysmal cold hemoglobinuria.
    3. Warm AIHA suspected to be secondary to a lymphoproliferative malignancy or secondary to an autoimmune disease (eg, systemic lupus erythematosus, Castleman's disease, Sjögren's syndrome, or other autoimmune diseases).
    4. A splenectomy less than 3 months before randomization.
    5. Concurrent conditions or history of other diseases:
    a. History or clinical manifestations of significant unstable metabolic, hepatic, renal, hematological, pulmonary, cardiovascular, gastrointestinal, urological, neurological, or psychiatric disorders.
    b. Current or previous malignancy within 5 years of study entry, except basal or squamous cell skin cancer with removal considered to be curative, superficial bladder cancer, prostate intraepithelial neoplasm, carcinoma in situ of the cervix, or other noninvasive or indolent malignancy without sponsor approval.
    c. Clinically significant cardiac disease, including unstable angina, acute myocardial infarction, and/or cardiac conduction issues within 6 months of Day 1 visit.
    d. Current New York Heart Association Class II to IV congestive heart failure or uncontrolled arrhythmia.
    6. Anti-phospholipid antibodies positive or elevated anti-streptolysin antibodies as assessed by the investigator as a clinical risk for thrombosis.
    7. Hepatitis B (HBV) or hepatitis C (HCV) infection: Participants who are positive for the hepatitis B surface antibody or hepatitis B core antibody will be eligible if they are negative for HBV-DNA; these participants should be considered for prophylactic antiviral therapy. Participants who are positive for the anti-HCV antibody will be eligible
    if they are negative for HCV-RNA.
    8. Known HIV infection or positivity on immunoassay.
    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 or < 480 milliseconds for females.
    1. Donne incinte o in fase di allattamento o partecipanti maschi e femmine che abbiano intenzione di concepire un figlio o di rimanere incinte nell’arco di tempo previsto per lo studio, a cominciare dallo screening e fino ai 90 giorni dopo la data dell’ultima dose del farmaco in studio.
    2. Diagnosi di altri tipi di AIHA, CAD (malattia da agglutinine fredde), AIHA di tipo misto o emoglobinuria parossistica a frigore.
    3. Avere una wAIHA che si sospetta essere secondaria a una neoplasia linfoproliferativa o secondaria a una patologia autoimmune (ad es. lupus eritematoso sistemico, malattia di Castleman, sindrome di Sjögren o altre malattie autoimmuni).
    4. Aver subito una splenectomia meno di 3 mesi prima della randomizzazione.
    5. Essere affetti da patologie concomitanti o pregresse:
    a. Manifestazioni cliniche o pregresse di significativa instabilità metabolica, epatica, renale, ematologica, polmonare, cardiovascolare, gastrointestinale, urologica, neurologica o disturbi psichiatrici.
    b. Malignità concomitanti o pregresse nei 5 anni precedenti l’inizio dello studio, fatta eccezione per il carcinoma basocellulare o squamocellulare con rimozione considerata curativa, il carcinoma vescicale superficiale, la neoplasia prostatica intraepiteliale, il carcinoma in situ della cervice o altre malignità non invasive o indolenti senza l’approvazione del promotore.
    c. Malattie cardiovascolari clinicamente significative, tra cui angina instabile, infarto miocardico acuto e/o problemi di conduzione cardiaca nei 6 mesi precedenti la visita del Giorno 1.
    d. Attuale aritmia incontrollata o insufficienza cardiaca congestizia di grado II-IV secondo la classificazione della New York Heart Association.
    6. Avere anticorpi antifosfolipidi o anticorpi antistreptolisina elevati, considerati dallo sperimentatore come un rischio clinico di trombosi.
    7. Essere affetti da epatite B (HBV) o da epatite C (HCV): i partecipanti risultati positivi all’antigene di superficie dell’epatite B o all’antigene core dell’epatite B saranno considerati idonei purché negativi all’HBV-DNA; per tali partecipanti si dovrà considerare la terapia antivirale profilattica. I partecipanti risultati positivi agli anticorpi anti-HCV saranno considerati idonei purché negativi all’HCV-RNA.
    8. Avere un’infezione da HIV nota o essere positivi al test immunologico.
    Nota: per i partecipanti arruolati nel territorio degli Stati Uniti il test di screening per l’HIV è facoltativo. Tuttavia, saranno esclusi i partecipanti arruolati nel territorio degli Stati Uniti con infezione da HIV nota.
    9. Storia o presenza di un ECG anomalo che, a giudizio dello sperimentatore, sia clinicamente significativo. Saranno esclusi i partecipanti con un intervallo QTc allo screening > 470 millisecondi per i maschi e > 480 millisecondi per le femmine (correzione di Fridericia). Qualora un singolo QTcF fosse > 470 millisecondi per i maschi o > 480 millisecondi per le femmine, il partecipante potrà arruolarsi, purché il QTcF medio per 3 ECG sia < 470 millisecondi per i maschi o < 480 millisecondi per le femmine.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of participants attaining a durable hemoglobin response, defined as hemoglobin = 10 g/dL with an increase from baseline of
    = 2 g/dL not attributed to rescue therapy at = 3 of the 4 available visits at Week 12 and/or later during the 24-week double-blind treatment period.
    Percentuale di partecipanti che raggiungono una risposta durevole dell'emoglobina, definita come emoglobina =10 g/dl con un aumento rispetto al basale di = 2 g/dl non attribuibile alla terapia di soccorso a = 3 delle 4 visite disponibili alla Settimana 12 e/o successivamente durante il periodo di trattamento in doppio cieco di 24 settimane.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 12 and/or later during the 24-week double-blind treatment period
    Settimana 12 e/o successivamente durante il periodo di trattamento in doppio cieco di 24 settimane
    E.5.2Secondary end point(s)
    Proportion of participants with a = 3-point increase from baseline in FACIT-F score at Week 24.
    Percentuale di partecipanti con un aumento di =3 punti dal basale del punteggio FACIT-F alla Settimana 24.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 24
    Settimana 24
    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 No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    To evaluate the participant's quality of life and other PROs.
    Valutare la qualità della vita dei partecipanti e altri PRO.
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA29
    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
    Belgium
    Canada
    France
    Germany
    Israel
    Italy
    Japan
    Netherlands
    Poland
    Spain
    Ukraine
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days10
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days10
    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: 80
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 57
    F.4.2.2In the whole clinical trial 100
    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)
    The patients will continue standard of care after completing the study.
    I pazienti continueranno lo standard di cura dopo aver completato lo studio.
    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 Decision2021-11-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-10-05
    P. End of Trial
    P.End of Trial StatusOngoing
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