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    Summary
    EudraCT Number:2022-001773-31
    Sponsor's Protocol Code Number:CVAY736O12301
    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:2022-11-18
    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 number2022-001773-31
    A.3Full title of the trial
    A phase 3, randomized, double-blind, study to assess efficacy and safety of ianalumab (VAY736) versus placebo in warm autoimmune hemolytic anemia (wAIHA) patients who failed at least one line of treatment (VAYHIA)
    Studio di fase 3, randomizzato, in doppio cieco, per valutare l’efficacia e la sicurezza d’impiego di ianalumab (VAY736) versus placebo nei pazienti con anemia emolitica autoimmune calda (wAIHA) che hanno fallito almeno una linea di trattamento (VAYHIA)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of efficacy and safety of ianalumab in previously treated patients with warm autoimmune haemolytic aneamia
    Studio di fase 3, randomizzato, in doppio cieco con ianalumab (VAY736) versus placebo in pazienti con anemia emolitica autoimmune calda (wAIHA) che hanno fallito almeno una linea di trattamento (VAYHIA)
    A.3.2Name or abbreviated title of the trial where available
    VAYHIA
    -
    A.4.1Sponsor's protocol code numberCVAY736O12301
    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 SponsorNOVARTIS PHARMA AG
    B.1.3.4CountrySwitzerland
    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 supportNovartis Pharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNOVARTIS FARMA S.p.A.
    B.5.2Functional name of contact pointRegulatory Affairs
    B.5.3 Address:
    B.5.3.1Street AddressViale Luigi Sturzo 43
    B.5.3.2Town/ cityMilano
    B.5.3.3Post code20154
    B.5.3.4CountryItaly
    B.5.4Telephone number00393427666361
    B.5.5Fax number0039029659066
    B.5.6E-mailinfo.studiclinici@novartis.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 nameIanalumab
    D.3.2Product code [VAY736]
    D.3.4Pharmaceutical form Concentrate for solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNIanalumab
    D.3.9.2Current sponsor codeVAY736
    D.3.9.4EV Substance CodeSUB193896
    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 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 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 placeboConcentrate for solution for injection/infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    warm autoimmune haemolytic anaemia (wAIHA)
    -
    E.1.1.1Medical condition in easily understood language
    warm autoimmune haemolytic anaemia (wAIHA)
    -
    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 25.0
    E.1.2Level LLT
    E.1.2Classification code 10047822
    E.1.2Term Warm type haemolytic anaemia
    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 demonstrate that either dose of ianalumab induces durable hemoglobin response compared to placebo, in patients with wAIHA who
    failed at least one previous line of treatment
    -
    E.2.2Secondary objectives of the trial
    Key secondary objective:
    1- To demonstrate that either dose of ianalumab maintains durable hemoglobin response, that is sustained beyond the end of the treatment period, compared to placebo

    Other secondary objectives:
    2- To assess the time to durable response / response / complete response in each treatment group
    3- To assess the quality of response in each treatment group (response, complete response, hemoglobin levels).
    4- To assess the need for rescue treatments in each treatment group
    5- To assess the safety profile of ianalumab
    6- To characterize the pharmacokinetics (PK) of ianalumab
    7- To assess B-cell levels at each treatment group
    8- To assess immunoglobulin levels at each treatment group
    9- To assess the immunogenicity of ianalumab
    10- To assess the quality of life (QoL) in each treatment group
    -
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - 18 years and older at time of signing consent
    - Patients with primary or secondary wAIHA, as previously documented by a positive direct antiglobulin test (DAT) specific for anti-IgG or anti-IgA, who had an insufficient response to, or relapsed after at least one line of treatment, including patients with steroid resistance, dependence or intolerance
    - Haemoglobin concentration at screening <10g/dL, associated with presence of symptoms related to anemia
    - The dose of supportive care must be stable for at least 4 weeks prior to randomisation into the study

    Other protocol-defined inclusion criteria may apply
    -
    E.4Principal exclusion criteria
    -wAIHA secondary to haematologic disease involving bone marrow (e.g. CLL) or other immunologic disease requiring immunosuppressant treatments that are not allowed in this study
    - Presence of other forms of AIHA (cold or intermediate forms), Evans syndrome or other cytopenias.
    - Prior use of B-cell depleting therapy (e.g., rituximab) within 12 weeks prior to randomisation
    -Neutrophils: <1000/mm3
    - Serum creatinine >1.5 x upper limit of normal (ULN)
    - Active viral, bacterial or other infections (including tuberculosis -TB or SARS-CoV-2) requiring systemic treatment at the time of screening, or history of recurrent clinically significant infections (e.g. bacterial infections with encapsulated organisms)
    - Positivity for hepatitis C virus, hepatitis B surface antigen (HBsAg), or hepatitis B core antibody (HBcAb)
    - Known history of primary or secondary immunodeficiency, or a positive human immune deficiency virus (HIV) test result
    - Live or live-attenuated vaccination within 4 weeks of randomisation
    - History of splenectomy

    Other protocol-defined exclusion criteria may apply
    -
    E.5 End points
    E.5.1Primary end point(s)
    Binary variable indicating whether a patient achieves a durable response (Hb =10 g/dL and =2 g/dL increase from baseline), for a period of at least 8 weeks, between W9 and W25, in the absence of rescue or prohibited treatment
    -
    E.5.1.1Timepoint(s) of evaluation of this end point
    Randomisation until W25
    -
    E.5.2Secondary end point(s)
    1- Duration of response
    2- Time from randomization to achievement of durable response, to first response, to first complete response
    3- Response rate, complete response rate and hemoglobin level
    4- Number and percentage of participants who received rescue; treatment overall and by type of rescue medication; Time-standardized numbers of each type of rescue treatment; Change from baseline in time-standardized number of transfusions
    5-frequency of adverse events and other safety parameters
    6- Ianalumab PK parameters – (i) AUClast, AUCtau, (ii) accumulation ratio Racc, (iii) Cmax, Tmax
    7- Change from baseline in the frequency and absolute number of CD19+B cell counts; Time to first occurrence of B Cell recovery, defined as =80% of baseline or =50 cells/µl
    8- Change from baseline in immunoglobulin levels
    9- Incidence and titer of anti-ianalumab antibodies in serum (ADA assay) over time
    10- Change from baseline in the 8 domain scores and in the summary scores (PCS, MCS) of SF-36 questionnaire; Change from baseline in the total score of PROMIS fatigue-13a questionnaire
    -
    E.5.2.1Timepoint(s) of evaluation of this end point
    1- 5: Randomization to end of study (up to 39 months after randomization of last patient)
    6i) After first dose of study treatment and after last dose
    6ii) First dose until last dose of study treatment
    6iii) After first dose of study treatment and after last dose
    7: Randomization to end of study (up to 39 months after randomization of last patient)
    8: Randomization to 2 years post last dose
    9: First dose of study treatment (pre-dose) to 20 weeks post last dose
    10: Randomization to end of study (up to 39 months after randomization of last patient)
    -
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic 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 Yes
    E.6.13.1Other scope of the trial description
    Patient Reported Outcomes, Immunogenicity
    -
    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 trial3
    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 EEA24
    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
    Argentina
    Australia
    China
    India
    Israel
    Japan
    Malaysia
    Singapore
    Taiwan
    Thailand
    United States
    France
    Romania
    Spain
    Germany
    Italy
    Hungary
    United Kingdom
    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
    -
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days7
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months1
    E.8.9.2In all countries concerned by the trial days19
    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: 48
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 42
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 24
    F.4.2.2In the whole clinical trial 90
    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
    -
    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 Decision2023-01-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-11-30
    P. End of Trial
    P.End of Trial StatusOngoing
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