Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43801   clinical trials with a EudraCT protocol, of which   7258   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools

    < Back to search results

    Print Download

    EudraCT Number:2022-001773-31
    Sponsor's Protocol Code Number:CVAY736O12301
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-10-11
    Trial results
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    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)
    Estudio de fase III, aleatorizado y doble ciego en el que se evalúan la eficacia y la seguridad de ianalumab (VAY736) frente a placebo en pacientes con anemia hemolítica autoinmune por anticuerpos calientes (AHAIc) en los que haya fracasado al menos una línea de tratamiento (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
    Estudio de eficacia y seguridad de ianalumab en pacientes previamente tratados con anemia hemolítica autoinmune por anticuerpos calientes
    A.3.2Name or abbreviated title of the trial where available
    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 Farmacéutica, S.A.
    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.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Farmacéutica, S.A.
    B.5.2Functional name of contact pointTrial Monitoring Organization (TMo)
    B.5.3 Address:
    B.5.3.1Street AddressGran Via de les Corts Catalanes, 764
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08013
    B.5.4Telephone number+3490 0353036
    B.5.5Fax number+3493 2479903
    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 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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. on Advanced therapies (CAT) has issued a classification for this product No
    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 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)
    Anemia hemolítica autoinmune por anticuerpos calientes (AHAIc)
    E.1.1.1Medical condition in easily understood language
    warm autoimmune haemolytic anaemia (wAIHA)
    Anemia hemolítica autoinmune por anticuerpos calientes (AHAIc)
    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
    Demostrar que cualquiera de las dosis de ianalumab induce una respuesta duradera de hemoglobina (Hb) en comparación con placebo en los pacientes con AHAIc en los que haya fracasado al menos una línea de tratamiento.
    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
    Objetivo secundario principal:
    1- Demostrar que cualquiera de las dosis de ianalumab mantiene una respuesta de hemoglobina duradera, que se mantiene más allá del final del periodo de tratamiento, en comparación con placebo.
    Otros objetivos secundarios:
    2- Evaluar el tiempo hasta la respuesta duradera/respuesta/respuesta completa en cada grupo de tratamiento.
    3- Evaluar la calidad de la respuesta en cada grupo de tratamiento (respuesta, respuesta completa, niveles de hemoglobina)
    4- Evaluar la necesidad de utilizar tratamientos de rescate en cada grupo de tratamiento.
    5- Evaluar el perfil de seguridad de ianalumab.
    6- Caracterizar la farmacocinética (PK) de ianalumab.
    7- Evaluar los niveles de células B en cada grupo de tratamiento.
    8- Evaluar los niveles de inmunoglobulina en cada grupo de tratamiento.
    9- Evaluar la inmunogenicidad frente a ianalumab.
    10- Evaluar la calidad de vida (QoL) en cada grupo de tratamiento.
    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
    - 18 años de edad o más el día de la firma del consentimiento.
    - Participantes con AHAIc primaria o secundaria (anteriormente documentado por un resultado positivo en la prueba de antiglobulina directa [PAD] específica para anti-IgG o anti-IgA), que hayan presentado una respuesta insuficiente o hayan recaído después de al menos una línea de tratamiento, incluidos los pacientes con resistencia, dependencia o intolerancia a los esteroides.
    - Concentración de hemoglobina en la selección <10 g/dl, asociada a la presencia de síntomas relacionados con la anemia.
    - La dosis del tratamiento de soporte debe ser estable durante al menos 4 semanas antes de la aleatorización.

    Otros criterios de inclusión definidos por protocolo pueden aplicar
    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
    - Pacientes con AHAIc secundaria a enfermedad hematológica que afecte a la médula ósea (por ejemplo, LLC) u otra enfermedad inmunológica que requiera tratamientos inmunosupresores que no están permitidos en este estudio.
    - Presencia de otras formas de AHAI (por anticuerpos fríos o mixtas), síndrome de Evans u otras citopenias.
    - Uso anterior de terapia de depleción de células B (e.j. rituximab) durante las 12 semanas anteriores a la aleatorización.
    - Neutrófilos: <1000/mm3
    - Creatinina sérica >1.5 por encima del límite superior de la normalidad
    - Infecciones víricas, bacterianas u otras infecciones activas (incluida la tuberculosis o SARS-CoV-2) que requieren tratamiento sistémico en el momento de la selección, o antecedentes de infecciones clínicamente significativas recurrentes (por ejemplo infecciones bacterianas con organismos encapsulados).
    - Positividad para virus de la hepatitis C, antígeno de superficie de la hepatitis B (HBsAg) o anticuerpo del núcleo de la hepatitis B (HBcAb).
    - Antecedentes conocidos de inmunodeficiencia primaria o secundaria, o un resultado de test positivo al virus de la inmunodeficiencia humana (HIV).
    - Vacunas vivas o atenuadas durante las 4 semanas anteriores a la aleatorización.
    - Historia de esplenectomía

    Otros criterios de exclusión definidos por protocolo pueden aplicar
    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
    Variable binaria que indica si un participante logra una respuesta duradera (Hb>=10 g/dL y aumento >=2 g/dL desde la basal) durante un periodo de al menos 8 semanas, entre la S9 y la S25, sin tratamiento de rescate o prohibido.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Randomisation until W25
    Aleatorización hasta S25
    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
    1- Duración de la respuesta
    2- Tiempo desde la aleatorización hasta la consecución de una respuesta duradera, hasta la primera respuesta, hasta la primera respuesta completa.
    3- Tasa de respuesta, tasa de respuesta completa y nivel de hemoglobina.
    4- Número y proporción de participantes que reciben tratamiento de rescate en general y por tipo de tratamiento de rescate; Número de cada tipo de tratamiento de rescate estandarizado por tiempo;
    Cambio respecto a la basal en el número de transfusiones estandarizado por tiempo.
    5- Frecuencia de acontecimientos adversos (AA) y otros parámetros de seguridad
    6-Parámetros de PKs para Ianalumab (i) Área bajo la curva calculada hasta el final de un intervalo de dosificación, Área bajo la curva desde el tiempo cero hasta la última concentración cuantificable (ii) Ratio de acumulación, (iii) Concentración plasmática máxima (pico) del fámarco, Tiempo para alcanzar la concentración plasmática máxima (pico) del fármaco
    7- Cambio respecto a la basal en la frecuencia y número absoluto de recuentos de células B CD19+; Tiempo hasta la primera recuperación de células B, definido como >=80 % del valor basal o >=50 células/μl.
    8- Cambio respecto a la basal en los niveles de inmunoglobulinas.
    9- Incidencia y títulos de anticuerpos antifármaco (AAF) en suero a lo largo del tiempo.
    10-Cambio desde basal en las puntuaciones de los 8 dominios y en las puntuaciones de resumen (PCS, MCS) de cuestionario SF-36; Cambio desde basal la puntuación total del cuestionario PROMIS-fatiga-13a.
    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)
    1-5: Aleatorización hasta la finalización del estudio (hasta 39 meses después de la aleatorización del último paciente)
    6i) Después de la primera dosis de tratamiento y después de la última dosis.
    6ii) Primera dosis hasta la última dosis del tratamiento del estudio
    6iii) Después de la primera dosis de tratamiento del estudio y después de la última dosis.
    7: Aleatorización hasta la finalización del estudio (hasta 39 meses después de la aleatorización del último paciente)
    8: Aleatorización a 2 años después de la última dosis
    9: Primera dosis del tratamiento del estudio (antes de la dosis) hasta 20 semanas después de la última dosis
    10: Aleatorización hasta la finalización del estudio (hasta 39 meses después de la aleatorización del último paciente)
    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
    Resultados comunicados por el paciente, Inmunogenicidad
    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. 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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 No
    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
    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
    última visita último paciente
    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 months10
    E.8.9.1In the Member State concerned days26
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 28
    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)
    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 Decision2023-02-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-11-21
    P. End of Trial
    P.End of Trial StatusOngoing
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands