Clinical trial • Phase II • Haematology
IANALUMAB for Immune thrombocytopenia | Warm autoimmune hemolytic anemia
Phase II trial of IANALUMAB for Immune thrombocytopenia | Warm autoimmune hemolytic anemia. None/Not specified-controlled. 34 participants.
Overview
- Trial Therapeutic Area
- Haematology
- Trial Disease
- Immune thrombocytopenia | Warm autoimmune hemolytic anemia
- Trial Stage
- Phase II
- Drug Modality
- Monoclonal antibody
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 20-08-2025
- First CTIS Authorization Date
- 05-12-2025
Trial design
None/Not specified-controlled Phase II trial across 20 sites in Belgium, Czechia, France and others.
- Comparator
- None/Not specified
- Target Sample Size
- 34
Eligibility
Recruits 34 Vulnerable population selected. Participants must provide signed informed consent prior to participation and must be aged 18 years or older. No assent procedures for minors are described in the available record..
- Vulnerable Population
- Vulnerable population selected. Participants must provide signed informed consent prior to participation and must be aged 18 years or older. No assent procedures for minors are described in the available record.
Inclusion criteria
- {"criterion_text":"- Signed informed consent obtained prior to participation in the study"}
- {"criterion_text":"- Male and female participants aged 18 years and older on the day of signing informed consent"}
- {"criterion_text":"- Primary ITP patients: 3. Previously enrolled and treated either with ianalumab/placebo in addition to first-line corticosteroids on protocol CVAY736I12301 or with ianalumab/placebo in addition to eltrombopag in the second line on protocol CVAY736Q12301, and who experienced treatment failure (TF) by parent trial definition ≥ 2 years after the last infusion of ianalumab/placebo"}
- {"criterion_text":"- Rescue medication and/or bridging therapy (See Section 6.6.3 and Section 6.6.4 for further details) are allowed to be started within the 28 days prior to screening; platelet count results obtained prior to the start of the therapy must be used to assess eligibility and have to be collected within 30 days prior to screening"}
- {"criterion_text":"- for primary or secondary wAIHA patients: Previously documented by a positive direct antiglobulin test (DAT) specific for anti-IgG or anti-IgA, previously enrolled and treated with ianalumab/placebo in blinded cohort or placebo followed by crossover to open label ianalumab in protocol CVAY736O12301, having experienced durable response lasting beyond 2 years from the last infusion of ianalumab/placebo in blinded cohorts or a durable response beyond week 20 from last dose of first course of ianalumab in the crossover arm"}
- {"criterion_text":"- for primary or secondary wAIHA patients: Relapsed wAIHA with hemoglobin concentration ≥5 g/dL and <10 g/dL and presence of symptoms related to anemia during screening or within 14 days before screening window or within 28 days before screening window if rescue medication/bridging therapy has been initiated"}
- {"criterion_text":"- Rescue medication and/or bridging therapy (see Section 6.6.3 and Section 6.6.4 for further details) are allowed to be started during the screening and within 28 days prior to screening; hemoglobin level result for eligibility assessment needs to be obtained prior to the start of the treatment within 30 days prior to screening"}
- {"criterion_text":"- Supportive care (see Section 6.6.3 for further details) is allowed in the case the participant received it in the parent trial when the relapse occurred and has remained stable at least 4 weeks prior screening"}
Exclusion criteria
- {"criterion_text":"- Evans syndrome or any cytopenia other than thrombocytopenia (for ITP participants) or anemia (for wAIHA participants), except for grade 1 anemia due to blood loss or iron deficiency"}
- {"criterion_text":"- Secondary wAIHA with BM involvement for wAIHA patients"}
- {"criterion_text":"- Current life-threatening bleeding or history of life-threatening bleeding due to thrombocytopenia"}
- {"criterion_text":"- Therapy for ITP or wAIHA other than ianalumab/placebo, bridging therapies and supportive care prior to the beginning of the screening window"}
- {"criterion_text":"- After primary analysis of each respective parent trial, participants whose treatment was unblinded and who received placebo only will be excluded"}
- {"criterion_text":"- ITP participants only: Participants with concurrent coagulation disorders and/or receiving anti-platelet or anti-coagulant medication except for low dose of acetylsalicylic acid (≤150 mg per day)"}
Endpoints
Primary endpoints
- {"endpoint_text":"- ITP participants: Treatment failure free (yes, no) by 12 m after start of second course of ianalumab is defined as any of: • platelet count <30G/L later than 8 weeks from start of second course • use of rescue treatment later than 8 weeks from second course • start of new ITP treatment • death • inability to taper TPO-RA by week 24","definition_or_measurement_approach":"Defined as any of the listed events occurring by 12 months after the start of the second course: platelet count <30 G/L later than 8 weeks from start of second course; use of rescue treatment later than 8 weeks from second course; start of new ITP treatment; death; inability to taper TPO-RA by week 24."}
- {"endpoint_text":"- For wAIHA participants Durable response (Hb ≥10 g/dL and ≥2 g/dL increase from baseline) for a period of at least 8 consecutive weeks, between W9 and W25 in the absence of rescue or prohibited treatment prior to that durable response achievement.","definition_or_measurement_approach":"Durable response defined as hemoglobin ≥10 g/dL and ≥2 g/dL increase from baseline sustained for at least 8 consecutive weeks between Week 9 and Week 25, without rescue or prohibited treatment prior to achieving the durable response."}
Secondary endpoints
- {"endpoint_text":"- Primary ITP participants only: • Response rate and complete response rate","definition_or_measurement_approach":"Measures of response and complete response rate in primary ITP participants (no further definition provided in the extracted record)."}
- {"endpoint_text":"- Primary ITP participants only: • Number and proportion of participants receiving rescue treatment and/or new ITP therapy","definition_or_measurement_approach":"Count and proportion of participants receiving rescue treatment and/or initiating new ITP therapy."}
- {"endpoint_text":"- wAIHA participants only: • Response rate and complete response rate","definition_or_measurement_approach":"Measures of response and complete response rate in wAIHA participants (no further definition provided in the extracted record)."}
- {"endpoint_text":"- wAIHA participants only: • Number and proportion of participants who received rescue treatment and/or new wAIHA therapy","definition_or_measurement_approach":"Count and proportion of participants receiving rescue treatment and/or initiating new wAIHA therapy (overall and by type of treatment)."}
- {"endpoint_text":"- for all participants: Frequency of adverse events and other safety parameters","definition_or_measurement_approach":"Frequency (incidence) of adverse events and other safety parameters; includes number/proportion of severe infections and other safety measures as listed in secondary endpoints."}
- {"endpoint_text":"- for all participants: Number of severe infections and proportion of participants with severe infection","definition_or_measurement_approach":"Count and proportion of participants with severe infections."}
- {"endpoint_text":"- for all participants: Ianalumab concentration in serum","definition_or_measurement_approach":"Measurement of serum concentration of ianalumab (pharmacokinetic assessment)."}
- {"endpoint_text":"- for all participants: Incidence and titer of anti-ianalumab antibodies in serum (ADA assay) over time","definition_or_measurement_approach":"Incidence and titres of anti-ianalumab antibodies measured in serum over time using ADA assay (immunogenicity assessment)."}
Recruitment
- Planned Sample Size
- 34
- Recruitment Window Months
- 57
- Consent Approach
- Signed informed consent required from participants; participants must be aged 18 years or older. Subject information and informed consent forms for adults are provided in multiple languages and country-specific versions (examples in the submitted documents include English, Dutch, French, Spanish, Bulgarian, Czech, German, Hungarian, Italian, Romanian). A 'Follow up for pregnant participant' ICF is included in some country packs. No assent for minors is described.
Geography
- Total Number Of Sites
- 20
- Total Number Of Participants
- 65
Belgium
- Earliest CTIS Part Ii Submission Date
- 31-10-2025
- Latest Decision Or Authorization Date
- 08-12-2025
- Processing Time Days
- 38
- Number Of Sites
- 1
- Number Of Participants
- 8
Sites
- Site Name
- Algemeen Ziekenhuis Delta
- Department Name
- 3001: Hematology
- Principal Investigator Name
- Lien Deleu
- Principal Investigator Email
- Lien.deleu@azdelta.be
- Contact Person Name
- Lien Deleu
- Contact Person Email
- Lien.deleu@azdelta.be
Czechia
- Earliest CTIS Part Ii Submission Date
- 05-11-2025
- Latest Decision Or Authorization Date
- 09-12-2025
- Processing Time Days
- 34
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- Fakultni Nemocnice Ostrava
- Department Name
- 3101: Klinika hematoonkologie FNO a LF OU
- Principal Investigator Name
- Jaromir Gumulec
- Principal Investigator Email
- jaromir.gumulec@fno.cz
- Contact Person Name
- Jaromir Gumulec
- Contact Person Email
- jaromir.gumulec@fno.cz
- Site Name
- Fakultni Nemocnice Kralovske Vinohrady
- Department Name
- 3102: Hematologicka klinika
- Principal Investigator Name
- Olga Cerna
- Principal Investigator Email
- olga.cerna@fnkv.cz
- Contact Person Name
- Olga Cerna
- Contact Person Email
- olga.cerna@fnkv.cz
France
- Earliest CTIS Part Ii Submission Date
- 20-11-2025
- Latest Decision Or Authorization Date
- 11-03-2026
- Processing Time Days
- 111
- Number Of Sites
- 2
- Number Of Participants
- 10
Sites
- Site Name
- Centre Hospitalier Universitaire De Caen Normandie
- Department Name
- 3152 : 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
- 3153 : Médecine interne
- Principal Investigator Name
- Louis Terriou
- Principal Investigator Email
- Louis.terriou@chru-lille.fr
- Contact Person Name
- Louis Terriou
- Contact Person Email
- Louis.terriou@chru-lille.fr
Germany
- Earliest CTIS Part Ii Submission Date
- 06-11-2025
- Latest Decision Or Authorization Date
- 11-03-2026
- Processing Time Days
- 125
- Number Of Sites
- 1
- Number Of Participants
- 11
Sites
- Site Name
- Universitaetsklinikum Jena KöR
- Department Name
- 3201 : Klinik für Innere Medizin II Abteilung Hämatologie und Internistische Onkologie
- Principal Investigator Name
- Thomas Stauch
- Principal Investigator Email
- Thomas.Stauch@med.uni-jena.de
- Contact Person Name
- Thomas Stauch
- Contact Person Email
- Thomas.Stauch@med.uni-jena.de
Hungary
- Earliest CTIS Part Ii Submission Date
- 11-09-2025
- Latest Decision Or Authorization Date
- 10-12-2025
- Processing Time Days
- 90
- Number Of Sites
- 1
- Number Of Participants
- 6
Sites
- Site Name
- University Of Debrecen
- Department Name
- 3251: Belgyogyaszati Klinika
- Principal Investigator Name
- Arpad Iles
- Principal Investigator Email
- illes.arpad@med.unideb.hu
- Contact Person Name
- Arpad Iles
- Contact Person Email
- illes.arpad@med.unideb.hu
Italy
- Earliest CTIS Part Ii Submission Date
- 24-11-2025
- Latest Decision Or Authorization Date
- 09-12-2025
- Processing Time Days
- 15
- Number Of Sites
- 6
- Number Of Participants
- 10
Sites
- Site Name
- Azienda Unita Locale Socio Sanitaria N 8 Berica
- Department Name
- 3351; U.O.C. Ematologia
- Principal Investigator Name
- Giuseppe Carli
- Principal Investigator Email
- g.carli@aulss8.veneto.it
- Contact Person Name
- Giuseppe Carli
- Contact Person Email
- g.carli@aulss8.veneto.it
- Site Name
- ASST Fatebenefratelli Sacco
- Department Name
- 3356; U.O.C. Ematologia e Medicina Trasfusionale
- Principal Investigator Name
- Alessio Di Prima
- Principal Investigator Email
- diprima.alessio@asst-fbf-sacco.it
- Contact Person Name
- Alessio Di Prima
- Contact Person Email
- diprima.alessio@asst-fbf-sacco.it
- Site Name
- Azienda Ospedaliero Universitaria Careggi
- Department Name
- 3352; Dipartimento Ematologia
- Principal Investigator Name
- Valentina Carrai
- Principal Investigator Email
- carraiv@aou-careggi.toscana.it
- Contact Person Name
- Valentina Carrai
- Contact Person Email
- carraiv@aou-careggi.toscana.it
- Site Name
- Azienda Sanitaria Universitaria Giuliano Isontina
- Department Name
- 3353; U.C.O. Ematologia - Dipartimento di Oncologia
- Principal Investigator Name
- Francesco Zaja
- Principal Investigator Email
- francesco.zaja@asugi.sanita.fvg.it
- Contact Person Name
- Francesco Zaja
- Contact Person Email
- francesco.zaja@asugi.sanita.fvg.it
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- 3355; U.O.C. Servizio e DH di Ematologia
- Principal Investigator Name
- Elena Rossi
- Principal Investigator Email
- elena.rossi@unicatt.it
- Contact Person Name
- Elena Rossi
- Contact Person Email
- elena.rossi@unicatt.it
- Site Name
- Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
- Department Name
- 3354; U.O.C. di Ematologia Dipartimento di Oncologia e Ematologia
- Principal Investigator Name
- Francesca Palandri
- Principal Investigator Email
- francesca.palandri@unibo.it
- Contact Person Name
- Francesca Palandri
- Contact Person Email
- francesca.palandri@unibo.it
Romania
- Earliest CTIS Part Ii Submission Date
- 20-11-2025
- Latest Decision Or Authorization Date
- 08-12-2025
- Processing Time Days
- 18
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Spitalul Clinic Coltea
- Department Name
- 3501: Hematology
- Principal Investigator Name
- Colita Andrei
- Principal Investigator Email
- andreicolita@yahoo.com
- Contact Person Name
- Colita Andrei
- Contact Person Email
- andreicolita@yahoo.com
Spain
- Earliest CTIS Part Ii Submission Date
- 26-09-2025
- Latest Decision Or Authorization Date
- 10-12-2025
- Processing Time Days
- 75
- Number Of Sites
- 5
- Number Of Participants
- 8
Sites
- Site Name
- Hospital Universitario De Salamanca
- Department Name
- 3405: Hematology
- Principal Investigator Name
- Jose Ramón González Porras
- Principal Investigator Email
- jrgp@usal.es
- Contact Person Name
- Jose Ramón González Porras
- Contact Person Email
- jrgp@usal.es
- Site Name
- Hospital General Universitario Morales Meseguer
- Department Name
- 3403: Hematology
- Principal Investigator Name
- María Luisa Lozano Almela
- Principal Investigator Email
- mllozano@um.es
- Contact Person Name
- María Luisa Lozano Almela
- Contact Person Email
- mllozano@um.es
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- 3402: Hematology
- Principal Investigator Name
- David Valcárcel Ferreiras
- Principal Investigator Email
- dvalcarcelct@vhio.net
- Contact Person Name
- David Valcárcel Ferreiras
- Contact Person Email
- dvalcarcelct@vhio.net
- Site Name
- Complexo Hospitalario Universitario De Santiago
- Department Name
- 3404: Hematology
- Principal Investigator Name
- Adrián Mosquera Orgueira
- Principal Investigator Email
- adrian.mosquera.orgeira@sergas.es
- Contact Person Name
- Adrián Mosquera Orgueira
- Contact Person Email
- adrian.mosquera.orgeira@sergas.es
- Site Name
- Hospital General Universitario Gregorio Maranon
- Department Name
- 3401: Hematology and Hemotherapy
- Principal Investigator Name
- Cristina Pascual Izquierdo
- Principal Investigator Email
- crisizquierdo3@yahoo.es
- Contact Person Name
- Cristina Pascual Izquierdo
- Contact Person Email
- crisizquierdo3@yahoo.es
Bulgaria
- Earliest CTIS Part Ii Submission Date
- 03-11-2025
- Latest Decision Or Authorization Date
- 20-02-2026
- Processing Time Days
- 109
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- Diagnostic-Consultative Center I Plovdiv EOOD
- Department Name
- 3052: N/A
- Principal Investigator Name
- Mariya Dimitrova
- Principal Investigator Email
- manasieva64@abv.bg
- Contact Person Name
- Mariya Dimitrova
- Contact Person Email
- manasieva64@abv.bg
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
- 1
- Name
- IQVIA Limited
- Responsibilities
- 1,3
- Name
- Parexel International (IRL) Limited
- Responsibilities
- 12
- Name
- Icon Clinical Research Limited
- Responsibilities
- 1
- Name
- SGS France
- Responsibilities
- 4
Third parties
- {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"1","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Illingworth Research Group Limited","duties_or_roles":"Off site research nursing implementation and management","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United States","full_name":"Jumo Health USA Inc.","duties_or_roles":"Preparation and translation of patient engagement materials","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"1,3","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"12","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Q Squared Solutions Limited","duties_or_roles":"4","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Patient reimbursement and travel support","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"1","organisation_type":"Pharmaceutical company"}
- {"country":"France","full_name":"SGS France","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- VAY736
- Active Substance
- IANALUMAB
- Modality
- Monoclonal antibody
- Routes Of Administration
- INTRAVENOUS
- Route
- INTRAVENOUS
- Authorisation Status
- prodAuthStatus:1
- Orphan Designation
- Yes
- Maximum Dose
- 9 mg/kg
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