Clinical trial • Phase III • Haematology
Ianalumab (IANALUMAB) for Warm autoimmune haemolytic anaemia
Phase III trial of Ianalumab (IANALUMAB) for Warm autoimmune haemolytic anaemia.
Overview
- Trial Therapeutic Area
- Haematology
- Trial Disease
- Warm autoimmune haemolytic anaemia
- Trial Stage
- Phase III
- Drug Modality
- Monoclonal antibody|Other
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 10-06-2024
- First CTIS Authorization Date
- 12-07-2024
Trial design
Randomised, ianalumab (vay736) versus placebo (placebo to vay736 150 mg/1 ml concentrate for solution for infusion). investigational doses include 3 mg/kg and 9 mg/kg iv (concentrate for solution for infusion); specific dosing schedule not specified in the provided record.-controlled Phase III trial in Germany, Hungary, Spain and others.
- Randomised
- Yes
- Comparator
- Ianalumab (VAY736) versus placebo (Placebo to VAY736 150 mg/1 mL concentrate for solution for infusion). Investigational doses include 3 mg/kg and 9 mg/kg IV (concentrate for solution for infusion); specific dosing schedule not specified in the provided record.
- Target Sample Size
- 60
Eligibility
Recruits 60 Vulnerable population flag is selected. All participants must provide written informed consent prior to any screening assessments (ICF required); participants must be adults aged ≥18 years. Pregnancy-related follow-up ICFs are included in submitted documents (documents titled 'Follow up for pregnant participant', and 'Pregnancy Follow up Parent Legal Guardian'), indicating specific procedures for pregnant participants. No assent procedures for minors are provided (minors are excluded by age)..
- Vulnerable Population
- Vulnerable population flag is selected. All participants must provide written informed consent prior to any screening assessments (ICF required); participants must be adults aged ≥18 years. Pregnancy-related follow-up ICFs are included in submitted documents (documents titled 'Follow up for pregnant participant', and 'Pregnancy Follow up Parent Legal Guardian'), indicating specific procedures for pregnant participants. No assent procedures for minors are provided (minors are excluded by age).
Inclusion criteria
- {"criterion_text":"- Written informed consent form (ICF) must be obtained prior to any screening assessments"}
- {"criterion_text":"- Male or female participants aged 18 years and older on the day of signing the ICF"}
- {"criterion_text":"- Participants with primary or secondary wAIHA (previously documented by 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 corticosteroid resistance, dependence, or intolerance"}
- {"criterion_text":"- Hemoglobin concentration ≥5 g/dL and <10 g/dL and presence of symptoms related to anemia at Screening and Week 1."}
- {"criterion_text":"- The dose of supportive care must be stable for at least 4 weeks prior randomization."}
Exclusion criteria
- {"criterion_text":"- Patients with wAIHA secondary to hematologic disease involving bone marrow (e.g., chronic lymphocytic leukemia (CLL)) or another disease requiring prohibited medication. Of note, the patients with autoimmune diseases like lupus nephritis (LN), systemic lupus erythematosus (SLE), Primary Sjögren’s Syndrome (pSS) or autoimmune hepatitis (AIH) after wash-out from the treatments are allowed"}
- {"criterion_text":"- Prior use of B-cell depleting therapy: • within 12 weeks prior randomization, or • no hematologic response to the last course of B-cell depleting therapy, irrespective of time of administration"}
- {"criterion_text":"- Active viral, bacterial, or other infections (including active or latent tuberculosis or SARSCoV- 2) requiring systemic treatment at the time of screening or history of recurrent clinically significant infection"}
- {"criterion_text":"- Known history of primary or secondary immunodeficiency, or patients that are Human Immunodeficiency Virus (HIV), Hepatitis C Virus (HCV), Hepatitis B surface Antigen (HBsAg)/ Hepatitis B core antibody (HBcAb)-positive. Refer to Section 5.2 exclusion criterion #7b for exemptions applicable for patients who are HBsAg negative and HBcAb positive."}
- {"criterion_text":"- Live or live-attenuated vaccination within 4 weeks before randomization"}
Endpoints
Primary endpoints
- {"endpoint_text":"- 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","definition_or_measurement_approach":"Durable response defined as hemoglobin (Hb) ≥10 g/dL and ≥2 g/dL increase from baseline for at least 8 consecutive weeks between Week 9 and Week 25, assessed in the absence of rescue or prohibited treatment."}
Secondary endpoints
- {"endpoint_text":"- Duration of response","definition_or_measurement_approach":"Duration of response as recorded for responders (no further detailed measurement approach provided in the record)."}
- {"endpoint_text":"- Time from randomization to achievement of durable response, to first response, to first complete response","definition_or_measurement_approach":"Time-to-event measures (time from randomization to durable response / first response / first complete response)."}
- {"endpoint_text":"- Response rate, complete response rate and hemoglobin level","definition_or_measurement_approach":"Proportions of participants achieving response and complete response; hemoglobin levels measured over time."}
- {"endpoint_text":"- Number and proportion of participants that receive rescue treatment overall and by type of rescue treatment","definition_or_measurement_approach":"Counts and proportions of participants receiving rescue treatments overall and stratified by rescue treatment type."}
- {"endpoint_text":"- Time-standardized numbers of each type of rescue treatment","definition_or_measurement_approach":"Time-standardized counts of each type of rescue treatment (method not further specified)."}
- {"endpoint_text":"- Change from baseline in time-standardized number of transfusions","definition_or_measurement_approach":"Change from baseline in transfusion counts standardized by time (specific calculation not detailed)."}
- {"endpoint_text":"- Frequency of AEs and other safety parameters","definition_or_measurement_approach":"Incidence/frequency counts of adverse events and other safety parameters."}
- {"endpoint_text":"- Ianalumab concentration in serum and PK parameters after the first and last dose","definition_or_measurement_approach":"Serum concentrations of ianalumab and derived pharmacokinetic parameters following first and last dose."}
- {"endpoint_text":"- B-cell levels: • 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","definition_or_measurement_approach":"Change from baseline in CD19+ B-cell frequency and absolute counts; time to B-cell recovery defined as ≥80% of baseline or ≥50 cells/μL."}
- {"endpoint_text":"- Immunoglobulins: • Change from baseline in immunoglobulin levels","definition_or_measurement_approach":"Change from baseline in immunoglobulin levels (measurement method not detailed)."}
- {"endpoint_text":"- Incidence and titer of anti-drug antibodies (ADA) in serum over time","definition_or_measurement_approach":"Incidence and titres of anti-drug antibodies measured in serum at scheduled timepoints."}
- {"endpoint_text":"- Change from baseline in dedicated Patient Reported Outcomes (PROs)","definition_or_measurement_approach":"Change from baseline in pre-specified patient-reported outcome instruments; specific PRO instruments not listed in the record."}
Recruitment
- Registry Or Advocacy Recruitment
- True, Health Literacy Media
- Digital Remote Recruitment
- True — third-party Jumo Health USA Inc. is listed with responsibility for patient recruitment and retention material; country-specific advertisement materials (K2_Advertisements) were provided in multiple languages suggesting use of digital/print advertisement channels.
- Planned Sample Size
- 60
- Recruitment Window Months
- 69
- Consent Approach
- Written informed consent required from each participant prior to any screening assessments. Participants must be adults (≥18) and provide their own consent; ICFs and related subject information sheets are provided in multiple country-specific languages (documents available in Hungarian, Romanian, French, German, Spanish, Italian, English among submitted ICF and information documents). Pregnancy follow-up ICFs are included for pregnant participants or partners.
Methods
- Country-specific recruitment arrangements documents (K1_Recruitment Arrangements) submitted for multiple countries (e.g., DE, HU, ES, IT, FR, RO) indicating use of organised country-level recruitment plans.
- Advertisements (K2_Advertisements) in country-specific languages (e.g., German, Hungarian, Spanish, Italian, French) — materials submitted for multiple countries implying use of adverts targeted at potential participants with wAIHA.
- Third-party patient recruitment and retention material provided by Jumo Health USA Inc. (duty: 'Patient recruitment and retention material').
- Patient guides/tools developed with patient organisation 'Health Literacy Media' (duty: 'Patient guides and tools').
- Quantitative in-trial interviews and patient-facing materials managed by agencies (Adelphi Values Limited and RWS Life Sciences Inc. listed with duties relating to participant interviews and PRO formatting/translation).
Geography
- Total Number Of Sites
- 21
- Total Number Of Participants
- 32
Germany
- Latest Decision Or Authorization Date
- 12-03-2026
- Number Of Sites
- 4
- Number Of Participants
- 10
Sites
- Site Name
- Gemeinschaftspraxis Haematologie Onkologie
- Department Name
- #2100 Gemeinschaftspraxis Hämatologie-Onkologie
- Principal Investigator Name
- Thomas Illmer
- Principal Investigator Email
- illmer@onkologie-dresden.net
- Contact Person Name
- Thomas Illmer
- Contact Person Email
- illmer@onkologie-dresden.net
- Site Name
- Onkologisches Studienzentrum am Raschplatz GbR
- Department Name
- #2105 Onkologisches Studienzentrum
- Principal Investigator Name
- Eyck Von der Heyde
- Principal Investigator Email
- hdheyde@onkologie-am-raschplatz.de
- Contact Person Name
- Eyck Von der Heyde
- Contact Person Email
- hdheyde@onkologie-am-raschplatz.de
- Site Name
- Justus-Liebig-Universitaet Giessen
- Department Name
- #2104 Hämatologie/ Onkologie Med. Klinik IV
- Principal Investigator Name
- Mathias Rummel
- Principal Investigator Email
- Mathias.rummel@innere.med.uni-giessen.de
- Contact Person Name
- Mathias Rummel
- Contact Person Email
- Mathias.rummel@innere.med.uni-giessen.de
- Site Name
- Universitaetsklinikum Essen AöR
- Department Name
- #2103 Hämatologie und Stammzellentransplantation
- Principal Investigator Name
- Alexander Roeth
- Principal Investigator Email
- Mathias.rummel@innere.med.uni-giessen.de
- Contact Person Name
- Alexander Roeth
- Contact Person Email
- Mathias.rummel@innere.med.uni-giessen.de
Hungary
- Latest Decision Or Authorization Date
- 12-03-2026
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- University Of Debrecen
- Department Name
- 2200:I. Belgyogyaszati Klinika
- Principal Investigator Name
- Arpad Illes
- Principal Investigator Email
- illesarpaddr@gmail.com
- Contact Person Name
- Arpad Illes
- Contact Person Email
- illesarpaddr@gmail.com
Spain
- Latest Decision Or Authorization Date
- 11-03-2026
- Number Of Sites
- 2
- Number Of Participants
- 3
Sites
- Site Name
- Hospital General Universitario Morales Meseguer
- Department Name
- #2601 Hematología
- Principal Investigator Name
- Maria Luisa Lozano Almela
- Principal Investigator Email
- mllozano@um.es
- Contact Person Name
- Maria Luisa Lozano Almela
- Contact Person Email
- mllozano@um.es
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- #2600 Hematología
- Principal Investigator Name
- David Beneitez Pastor
- Principal Investigator Email
- david.beneitez@vallhebron.cat
- Contact Person Name
- David Beneitez Pastor
- Contact Person Email
- david.beneitez@vallhebron.cat
Italy
- Latest Decision Or Authorization Date
- 09-03-2026
- Number Of Sites
- 6
- Number Of Participants
- 8
Sites
- Site Name
- Azienda Universitaria Ospedaliera Consorziale Policlinico Bari
- Department Name
- 2405:U.O Ematologia con Trapianto
- Principal Investigator Name
- Pellegrino Musto
- Principal Investigator Email
- pellegrino.musto@uniba.it
- Contact Person Name
- Pellegrino Musto
- Contact Person Email
- pellegrino.musto@uniba.it
- Site Name
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
- Department Name
- 2404:U.O.C. Ematologia
- Principal Investigator Name
- Bruno Fattizzo
- Principal Investigator Email
- bruno.fattizzo@policlinicp.mi.it
- Contact Person Name
- Bruno Fattizzo
- Contact Person Email
- bruno.fattizzo@policlinicp.mi.it
- Site Name
- Regione Del Veneto Azienda ULSS N 7 Pedemontana
- Department Name
- 2401:U.O.C. Oncoematologia
- Principal Investigator Name
- Eros Di Bona
- Principal Investigator Email
- eros.dibona@aulss7.veneto.it
- Contact Person Name
- Eros Di Bona
- Contact Person Email
- eros.dibona@aulss7.veneto.it
- Site Name
- AORN San Giuseppe Moscati Avellino
- Department Name
- 2400:U.O.C. di Ematologia e Trapianto Emopoietico
- Principal Investigator Name
- Fulvia Fanelli
- Principal Investigator Email
- fulvia.fanelli@aornmoscati.it
- Contact Person Name
- Fulvia Fanelli
- Contact Person Email
- fulvia.fanelli@aornmoscati.it
- Site Name
- Azienda Ospedaliero-Universitaria Maggiore Della Carita
- Department Name
- 2402:S.C.D.U. Ematologia
- Principal Investigator Name
- Andrea Patriarca
- Principal Investigator Email
- andrea.patriarca@uniupo.it
- Contact Person Name
- Andrea Patriarca
- Contact Person Email
- andrea.patriarca@uniupo.it
- Site Name
- ASST Fatebenefratelli Sacco
- Department Name
- 2403:U.O.C. Ematologia e Medicina Transfusionale
- Principal Investigator Name
- Alessio DiPrima
- Principal Investigator Email
- diprima.alessio@asst-fbf-sacco.it
- Contact Person Name
- Alessio DiPrima
- Contact Person Email
- diprima.alessio@asst-fbf-sacco.it
France
- Latest Decision Or Authorization Date
- 30-03-2026
- Number Of Sites
- 7
- Number Of Participants
- 4
Sites
- Site Name
- Centre Hospitalier Le Mans
- Department Name
- #2008 Hématologie
- Principal Investigator Name
- Kamel Laribi
- Principal Investigator Email
- klaribi@ch-lemans.fr
- Contact Person Name
- Kamel Laribi
- Contact Person Email
- klaribi@ch-lemans.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- #2000 Médecine interne
- Principal Investigator Name
- Marc Michel
- Principal Investigator Email
- marc.michel2@aphp.fr
- Contact Person Name
- Marc Michel
- Contact Person Email
- marc.michel2@aphp.fr
- Site Name
- Centre Hospitalier Universitaire De Caen Normandie
- Department Name
- #2001 Hématologie
- Principal Investigator Name
- Stéphane Cheze
- Principal Investigator Email
- cheze-s@chu-caen.fr
- Contact Person Name
- Stéphane Cheze
- Contact Person Email
- cheze-s@chu-caen.fr
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- #2002 Médecine interne
- Principal Investigator Name
- Louis Terriou
- Principal Investigator Email
- louis.terriou@chu.lille.fr
- Contact Person Name
- Louis Terriou
- Contact Person Email
- louis.terriou@chu.lille.fr
- Site Name
- Centre Hospitalier Universitaire De Nice
- Department Name
- #2004 Médecine interne
- Principal Investigator Name
- Pierre Yves Jeandel
- Principal Investigator Email
- Jeandel.py@chu-nice.fr
- Contact Person Name
- Pierre Yves Jeandel
- Contact Person Email
- Jeandel.py@chu-nice.fr
- Site Name
- Centre Hospitalier Universitaire De Nantes
- Department Name
- #2009 Médecine interne
- Principal Investigator Name
- Julie Graveleau
- Principal Investigator Email
- Julie.graveleau@chu-nantes.fr
- Contact Person Name
- Julie Graveleau
- Contact Person Email
- Julie.graveleau@chu-nantes.fr
- Site Name
- Institut Universitaire Du Cancer Toulouse-Oncopole
- Department Name
- #2003 Hématologie
- Principal Investigator Name
- Thibault Comont
- Principal Investigator Email
- comont.thibault@iuct-oncopole.fr
- Contact Person Name
- Thibault Comont
- Contact Person Email
- comont.thibault@iuct-oncopole.fr
Sponsor
Primary sponsor
- Full Name
- Novartis Pharma AG
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Switzerland
Contract research organisations
- Name
- Syneos Health Inc.
- Responsibilities
- code:1
- Name
- Icon Clinical Research Limited
- Responsibilities
- code:1; code:2; code:7
- Name
- Parexel International (IRL) Limited
- Responsibilities
- code:12
- Name
- IQVIA Limited
- Responsibilities
- code:1
- Name
- Pharmaceutical Research Associates Group B.V.
- Responsibilities
- code:4
Third parties
- {"country":"United Kingdom","full_name":"Labcorp Early Development Laboratories Limited","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"DATAMAP-Gesellschaft fuer Datenmanagement Datenanalyse und Datenpraesentation mbH","duties_or_roles":"code:10; Independent statistical group for Data Monitoring Committee (DMC)","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Jumo Health USA Inc.","duties_or_roles":"Patient recruitment and retention material (code:15)","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"code:1","organisation_type":"Pharmaceutical company"}
- {"country":"Netherlands","full_name":"Pharmaceutical Research Associates Group B.V.","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Adelphi Values Limited","duties_or_roles":"Quantitative in-trial interviews with trial participants (code:15)","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"code:1; code:2; code:7","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"code:1","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"code:12","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Health Literacy Media","duties_or_roles":"Patient guides and tools (code:15)","organisation_type":"Patient organisation/association"}
- {"country":"United Kingdom","full_name":"Illingworth Research Group Limited","duties_or_roles":"off-site nursing service for protocol-specified visits in the long term follow up stage of the study (code:15)","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United States","full_name":"RWS Life Sciences Inc.","duties_or_roles":"PRO formatting and translation (code:15)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Icon Laboratory Services Inc.","duties_or_roles":"code:4","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Iqvia Rds Inc.","duties_or_roles":"Technical management of ePROs (code:15); code:3","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Iqvia Laboratories Limited","duties_or_roles":"code:4","organisation_type":"Non-Pharmaceutical company"}
Investigational products
- Investigational Product Name
- VAY736
- Active Substance
- Ianalumab (IANALUMAB)
- Modality
- Monoclonal antibody
- Routes Of Administration
- INTRAVENOUS
- Route
- Intravenous
- Orphan Designation
- Yes
- Starting Dose
- 3 mg/kg
- Dose Levels
- 3 mg/kg; 9 mg/kg
- Maximum Dose
- 9 mg/kg
- Investigational Product Name
- Placebo to VAY736 150 mg/1 mL concentrate for solution for infusion
- Modality
- Other
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