Clinical trial • Phase III • Immunology|Rare Disease

RILZABRUTINIB for Warm autoimmune haemolytic anaemia|Autoimmune haemolytic anaemia

Phase III trial of RILZABRUTINIB for Warm autoimmune haemolytic anaemia|Autoimmune haemolytic anaemia.

Overview

Trial Therapeutic Area
Immunology|Rare Disease
Trial Disease
Warm autoimmune haemolytic anaemia|Autoimmune haemolytic anaemia
Trial Stage
Phase III
Drug Modality
Small molecule
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
17-03-2025
First CTIS Authorization Date
07-07-2025

Trial design

Randomised, open-label, placebo (sar444671 placebo - matched placebo for test); active comparator: rilzabrutinib (oral film-coated tablet, sar444671). dose and dosing schedule not specified in the available ctis record.-controlled Phase III trial in Denmark, Germany, Italy and others.

Randomised
Yes
Open Label
Yes
Comparator
Placebo (Sar444671 placebo - matched placebo for test); Active comparator: Rilzabrutinib (oral film-coated tablet, SAR444671). Dose and dosing schedule not specified in the available CTIS record.
Target Sample Size
75
Trial Duration For Participant
224

Eligibility

Recruits 75 Vulnerable population selected (populationOfTrialSubjects.isVulnerablePopulationSelected = true). Participant informed consent documents (L1 redacted SIS-ICF and country-specific versions) and partner/pregnancy and caregiver information/ICF documents are listed in the trial documents (country-specific L1/L2 files). Assent handling is not specified in the available record; participants are adults (public title: participants 18 years of age and older)..

Vulnerable Population
Vulnerable population selected (populationOfTrialSubjects.isVulnerablePopulationSelected = true). Participant informed consent documents (L1 redacted SIS-ICF and country-specific versions) and partner/pregnancy and caregiver information/ICF documents are listed in the trial documents (country-specific L1/L2 files). Assent handling is not specified in the available record; participants are adults (public title: participants 18 years of age and older).

Inclusion criteria

  • {"criterion_text":"- Male and female participants with a documented (confirmed) diagnosis of primary wAIHA for at least 3 months."}
  • {"criterion_text":"- Participants who have previously failed to maintain a sustained response after treatment with CS (CS-resistance [defined as failure to obtain hemoglobin response within 3 weeks on at least 1 mg/kg or 60 mg prednisone or equivalent per day], CS-dependent wAIHA [defined as need to continue on prednisone or equivalent at a dose of >10 mg/day to maintain a response]), or are intolerant or ineligible to CS (defined as with contraindications, pre-existing medical conditions or CS-related complications that may render CS intolerant or ineligible per the best clinical judgement of the investigators)."}
  • {"criterion_text":"- Participants with Eastern Cooperative Oncology Group (ECOG) performance status Grade 2 or lower."}
  • {"criterion_text":"- Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies."}

Exclusion criteria

  • {"criterion_text":"- Participants with clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the participant or compromise the quality of the data derived from his or her participation in the study as determined by the Investigator."}
  • {"criterion_text":"- Concurrent treatment with other experimental/investigational drugs within 30 days or 5 half-lives, whichever is longer, prior to treatment start. Participants who previously received treatment with BTK inhibitors for wAIHA before Day 1 (randomization) are not eligible."}
  • {"criterion_text":"- Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study."}
  • {"criterion_text":"- Participants with medical history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for the past 3 years."}
  • {"criterion_text":"- Participants with symptomatic herpes zoster within 3 months prior to screening."}
  • {"criterion_text":"- Participants with secondary wAIHA from any cause including drugs, Evans Syndrome, lymphoproliferative disorders (low count monoclonal B-cell lymphocytosis is allowed), infectious or autoimmune disease, or active hematologic malignancies. Participants with positive antinuclear antibodies but without a definitive diagnosis of an autoimmune disease are allowed."}
  • {"criterion_text":"- Participants with history of myelodysplastic syndrome."}
  • {"criterion_text":"- Participants with uncontrolled or active HBV infection or Active HCV infection."}
  • {"criterion_text":"- HIV infection."}
  • {"criterion_text":"- Participants with history of solid organ transplant."}
  • {"criterion_text":"- Participants with a history of active or latent tuberculosis (TB)."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Proportion of participants achieving DHR. DHR is defined as an increase of Hb by ≥2 g/dL from baseline on at least two thirds of evaluable scheduled visits between Week 12 and Week 24 (inclusive) in the PAP","definition_or_measurement_approach":"DHR is defined as an increase of Hb by ≥2 g/dL from baseline on at least two thirds of evaluable scheduled visits between Week 12 and Week 24 (inclusive) in the PAP."}

Secondary endpoints

  • {"endpoint_text":"- Proportion of participants achieving overall Hb response (response [R] or complete response [CR]) by Week 24 of treatment in the PAP","definition_or_measurement_approach":"Proportion of participants achieving overall Hb response (response [R] or complete response [CR]) by Week 24 in the PAP."}
  • {"endpoint_text":"- The time taken (days) to achieve the first Hb increase by ≥2 g/dL from baseline during the PAP in the absence of transfusion in the previous 14 days and in the absence of rescue medication in the previous 6 weeks","definition_or_measurement_approach":"Time (days) from baseline to first Hb increase ≥2 g/dL during the PAP, provided no transfusion in prior 14 days and no rescue medication in prior 6 weeks."}
  • {"endpoint_text":"- Change from baseline in fatigue total score as measured by Functional Assessment of Chronic Illness Therapy (FACIT) – fatigue at Week 24","definition_or_measurement_approach":"Change from baseline in FACIT-Fatigue total score at Week 24."}
  • {"endpoint_text":"- Change from baseline in levels of LDH at Week 24","definition_or_measurement_approach":"Change from baseline in LDH level at Week 24."}
  • {"endpoint_text":"- Proportion of participants requiring use of rescue therapy after Week 4 of treatment during the PAP","definition_or_measurement_approach":"Proportion of participants who require rescue therapy after Week 4 during the PAP period."}
  • {"endpoint_text":"- Change from baseline in dyspnea severity score as measured by FACIT-Dyspnea at Week 24","definition_or_measurement_approach":"Change from baseline in FACIT-Dyspnea score at Week 24."}
  • {"endpoint_text":"- Incidence of treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (SAEs), treatment-emergent adverse events of special interest (AESIs), as well as clinical laboratory evaluations, vital sign, physical exam, and electrocardiograms (ECG)","definition_or_measurement_approach":"Incidence and description of TEAEs, SAEs, AESIs, and safety assessments including labs, vital signs, physical exam and ECGs as recorded during the study."}

Recruitment

Digital Remote Recruitment
Yes
Planned Sample Size
75
Recruitment Window Months
51
Consent Approach
Informed consent obtained from participants (adult participants 18 years and older). Country-specific redacted subject information and informed consent forms (L1 redacted SIS-ICF) and other subject information materials (L2) are provided in multiple language versions (examples in documents list include English, Danish (da), German (de), Italian (it), Spanish (es), Swedish (sv), Polish (pl), Hungarian (hu), Czech (cs), Dutch (nl), Greek (el) and other local variants). Caregiver and partner/pregnancy information and ICFs are included where applicable (country-specific L1/L2 partner-pregnancy and caregiver documents). Assent is not applicable (adult-only population).

Methods

  • Site-based recruitment via participating hospitals/clinics (multiple site-specific recruitment documents and local site information documents are provided for each country).
  • Printed materials: patient brochures, flyers, posters and participant letters (documents named K2-recruitment-material-patient-brochure-*, -flyer-*, -poster-*, -participant-letter-* present in country-specific versions).
  • Healthcare-professional outreach: 'dr-to-dr' materials (documents named K2-recruitment-material-dr-to-dr-*).
  • Website/digital presence: Sanofi studies webpage materials (documents named K2-recruitment-material-sanofi-studies-webpage-*).
  • Social media / digital advertising: social media post assets (document 'K2-recruitment-material-social-media-post-it' present).
  • Local/ad placement: advertisements and recruitment advertisements (K2 recruitment advertisement/advertisement-nl referenced).
  • Study materials for site use (flipcharts, UYS booklets) and other localized recruitment aids (documents named K2-recruitment-material-uys-book-*, K2-recuitment-material-flipchart-*).

Geography

Total Number Of Sites
41
Total Number Of Participants
58

Denmark

Earliest CTIS Part Ii Submission Date
17-06-2025
Latest Decision Or Authorization Date
13-01-2026
Processing Time Days
210
Number Of Sites
3
Number Of Participants
4

Sites

Site Name
Region Midtjylland
Department Name
Aarhus Universitetshospital - Skejby Blodsygdomme, Klinisk Forskningsenhed
Contact Person Name
Marianne Agerlund Petersen
Contact Person Email
maragepe@rm.dk
Site Name
Odense University Hospital
Department Name
Odense University Hospital Department of Haematology X
Contact Person Name
Henrik Frederiksen
Contact Person Email
henrik.frederiksen@rsyd.dk
Site Name
Rigshospitalet
Department Name
Copenhagen Rigshospitalet Afdeling for Blodsygdomme
Contact Person Name
Andreas Glenthøj
Contact Person Email
andreas.glenthoej@regionh.dk

Germany

Earliest CTIS Part Ii Submission Date
10-06-2025
Latest Decision Or Authorization Date
14-01-2026
Processing Time Days
218
Number Of Sites
2
Number Of Participants
8

Sites

Site Name
Universitaetsklinikum Essen AöR
Department Name
Klinik für Hämatologie und Stammzellentransplantation
Contact Person Name
Alexander Roeth
Contact Person Email
alexander.roeth@uk-essen.de
Site Name
Onkologische Schwerpunktpraxis (OSP) Kurfürstendamm
Department Name
N.A.
Contact Person Name
Ingo Schwaner

Italy

Earliest CTIS Part Ii Submission Date
12-06-2025
Latest Decision Or Authorization Date
15-01-2026
Processing Time Days
217
Number Of Sites
10
Number Of Participants
15

Sites

Site Name
IRCCS Ospedale Policlinico San Martino
Department Name
S.C. Ematologia e Terapie Cellulari
Contact Person Name
Marica Laurino
Contact Person Email
marica.laurino@hsanmartino.it
Site Name
Azienda Ospedaliera Universitaria Federico II Di Napoli
Department Name
U.O.C. di Ematologia e Trapianti di midollo
Contact Person Name
Fabrizio Pane
Contact Person Email
fabrizio.pane@unina.it
Site Name
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Department Name
UOC Ematologia Milano
Contact Person Name
Bruno Fattizzo
Contact Person Email
brunofattizzo@gmail.com
Site Name
Azienda Ospedaliero Universitaria Careggi
Department Name
S.O.D. Ematologia
Contact Person Name
Valentina Carrai
Contact Person Email
carraiv@aou-careggi.toscana.it
Site Name
Azienda Ospedaliero-Universitaria Maggiore Della Carita
Department Name
Struttura semplice Innovazione clinica e terapeutica in ematologia
Contact Person Name
Andrea Patriarca
Contact Person Email
andrea.patriarca@uniupo.it
Site Name
Istituto Romagnolo Per Lo Studio Dei Tumori Dino Amadori IRST S.r.l.
Department Name
Onco-Ematologia
Contact Person Name
Alessandro Lucchesi
Contact Person Email
alessandr.lucchesi@irst.emr.it
Site Name
Azienda Sanitaria Universitaria Giuliano Isontina
Department Name
UCO Ematologia
Contact Person Name
Francesco Zaja
Site Name
Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello
Department Name
U.O.C. Ematologia
Contact Person Name
Simona Raso
Contact Person Email
simona.raso86@gmail.com
Site Name
Azienda Unita Locale Socio Sanitaria N 8 Berica
Department Name
U.O.C. Ematologia
Contact Person Name
Giuseppe Carli
Contact Person Email
g.carli@aulss8.veneto.it
Site Name
Azienda Socio Sanitaria Territoriale Dei Sette Laghi
Department Name
S.C. EMATOLOGIA
Contact Person Name
Marta Coscia

Sweden

Earliest CTIS Part Ii Submission Date
06-06-2025
Latest Decision Or Authorization Date
14-01-2026
Processing Time Days
222
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
Region Skane Skanes Universitetssjukhus
Department Name
VO Hematologi, Onkologi och Strålningsfysik
Contact Person Name
Alexandros Arvanitakis
Site Name
Karolinska University Hospital
Department Name
Karolinska Universitetssjukhuset - Huddinge Cancerstudieenheten Huddinge, M62
Contact Person Name
Cecilia Karlström

Austria

Earliest CTIS Part Ii Submission Date
07-07-2025
Latest Decision Or Authorization Date
16-01-2026
Processing Time Days
193
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
Medical University Of Vienna
Department Name
Allgemeines Krankenhaus Medizinische Universität Wien, Clinical Pharmacology
Contact Person Name
Bernd Jilma
Contact Person Email
bernd.jilma@meduniwien.ac.at
Site Name
Hanusch Krankenhaus Der Wiener Gebietskrankenkasse
Department Name
Hanusch Hospital, 3rd Medical Department
Contact Person Name
Michael Fillitz
Contact Person Email
michael.fillitz@oegk.at

Czechia

Earliest CTIS Part Ii Submission Date
01-04-2025
Latest Decision Or Authorization Date
13-01-2026
Processing Time Days
287
Number Of Sites
2
Number Of Participants
2

Sites

Site Name
Institute Of Hematology And Blood Transfusion
Contact Person Name
Jacqueline Soukupova Maaloufova
Contact Person Email
Jacqueline.Maaloufova@uhkt.cz
Site Name
Fakultni Nemocnice Brno
Department Name
Interni hematologicka a onkologicka klinika
Contact Person Name
Libor Červinek
Contact Person Email
Cervinek.libor@fnbrno.cz

Greece

Earliest CTIS Part Ii Submission Date
01-04-2025
Latest Decision Or Authorization Date
13-01-2026
Processing Time Days
287
Number Of Sites
5
Number Of Participants
5

Sites

Site Name
Geniko Nosokomeio Thessalonikis George Papanikolaou
Department Name
Hematology Dept. BMT Unit
Contact Person Name
Ioanna Sakellari
Contact Person Email
bmt@gpapanikolaou.gr
Site Name
Laiko General Hospital Of Athens
Department Name
Department of Hematology, NKUA
Contact Person Name
Marina Siakantaris
Contact Person Email
msiaka@med.uoa.gr
Site Name
General University Hospital Of Patras
Department Name
Department of Hematology
Contact Person Name
Alexandros Spyridonidis
Contact Person Email
spyridonidis@upatras.gr
Site Name
General University Hospital Of Larissa
Department Name
Department of Hematology
Contact Person Name
Georgios Vassilopoulos
Contact Person Email
gvasilop@uth.gr
Site Name
Olympion Therapeftirio General Clinic Of Patras S.A.
Department Name
Haematology & Oncology Dept.
Contact Person Name
Argiris Symeonidis
Contact Person Email
argiris.symeonidis@yahoo.gr

Spain

Earliest CTIS Part Ii Submission Date
09-06-2025
Latest Decision Or Authorization Date
14-01-2026
Processing Time Days
219
Number Of Sites
5
Number Of Participants
8

Sites

Site Name
Hospital Universitario De Cruces
Department Name
Servicio Hematologia
Contact Person Name
Miriam Vara Pampliega
Site Name
Hospital Clinic De Barcelona
Department Name
Servicio Hematologia
Contact Person Name
Joan Cid Vidal
Contact Person Email
jcid@clinic.cat
Site Name
Hospital General Universitario Gregorio Maranon
Department Name
Servicio Hematologia
Contact Person Name
Cristina Pascual Izquierdo
Contact Person Email
crisizquierdo3@yahoo.es
Site Name
University Hospital Virgen Del Rocio S.L.
Department Name
Servicio Hematologia
Contact Person Name
Maria Eva Mingot Castellano
Contact Person Email
memingot@gmail.com
Site Name
Hospital Universitario Puerta De Hierro De Majadahonda
Department Name
Servicio Hematologia
Contact Person Name
Silvia Monsalvo
Contact Person Email
silviamonsalvo@gmail.com

Netherlands

Earliest CTIS Part Ii Submission Date
02-07-2025
Latest Decision Or Authorization Date
19-01-2026
Processing Time Days
201
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
Leids Universitair Medisch Centrum (LUMC)
Department Name
Hematology
Contact Person Name
Marit Jalink
Contact Person Email
m.jalink@lumc.nl
Site Name
Amsterdam UMC Stichting
Department Name
Hematology
Contact Person Name
Sophie Bernelot Moens

Hungary

Earliest CTIS Part Ii Submission Date
09-05-2025
Latest Decision Or Authorization Date
15-01-2026
Processing Time Days
251
Number Of Sites
3
Number Of Participants
3

Sites

Site Name
University Of Debrecen
Department Name
Belgyogyaszati Klinika B Epulet
Contact Person Name
Árpád Illés
Contact Person Email
illes.arpad@med.unideb.hu
Site Name
Semmelweis University
Department Name
Belgyógyászati és Hematológiai Klinika
Contact Person Name
Gabor Mikala
Contact Person Email
gmikala67@gmail.com
Site Name
Del-Pesti Centrumkorhaz Orszagos Hematologiai Es Infektologiai Intezet
Department Name
Hematologiai es Ossejt-transzplantacios Osztaly
Contact Person Name
Gábor Mikala
Contact Person Email
gmikala@laszlokorhaz.hu

Poland

Earliest CTIS Part Ii Submission Date
10-06-2025
Latest Decision Or Authorization Date
19-01-2026
Processing Time Days
223
Number Of Sites
5
Number Of Participants
4

Sites

Site Name
Wojewodzkie Wielospecjalistyczne Centrum Onkologii I Traumatologii Im M.Kopernika W Lodzi
Department Name
Wojewódzki Szpital Specjalistyczny im. M.Kopernika w Łodzi
Contact Person Name
Robak Tadeusz
Contact Person Email
robaktad@csk.umed.lodz.pl
Site Name
In Vivo Sp. z o.o.
Department Name
IN VIVO
Contact Person Name
Jaroslaw Czyz
Contact Person Email
jczyz@onet.pl
Site Name
Santa Sp. z o.o.
Department Name
Santa Familia PTG Lodz
Contact Person Name
Michal Piotr Witkowski
Site Name
Mazowiecki Szpital Specjalistyczny w Radomiu
Department Name
Mazowiecki Szpital Specjalistyczny w Radomiu Oddzial Hematologii
Contact Person Name
Marzena WATEK
Contact Person Email
marzena.watek@wp.pl
Site Name
Uniwersyteckie Centrum Kliniczne
Department Name
Gdańskiego Uniwersytetu Medycznego Klinika Hematologii
Contact Person Name
Andrzej MITAL
Contact Person Email
andrzej.mital@gumed.edu.pl

Sponsor

Primary sponsor

Full Name
Sanofi-Aventis Recherche & Developpement
Organisation Type
Pharmaceutical company
Country Of Registered Address
France

Contract research organisations

Name
Endpoint Clinical Inc.
Responsibilities
code: 3
Name
MARKEN Germany GmbH
Responsibilities
code: 14
Name
Fisher Clinical Services UK Limited
Responsibilities
code: 14
Name
Labcorp Central Laboratory Services LP
Responsibilities
code: 4
Name
Labcorp Central Laboratory Services SARL
Responsibilities
code: 4
Name
Discovery Life Sciences LLC
Responsibilities
code: 4

Third parties

  • {"country":"United States","full_name":"Endpoint Clinical Inc.","duties_or_roles":"code: 3","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Labcorp Early Development Laboratories Inc.","duties_or_roles":"code: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"Germany","full_name":"MARKEN Germany GmbH","duties_or_roles":"code: 14","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"ESMS Global Limited","duties_or_roles":"Centralized 24-Hour Emergency System: eSMS (value shown)","organisation_type":"Pharmaceutical company"}
  • {"country":"Czechia","full_name":"PHOENIX lekarensky velkoobchod s.r.o.","duties_or_roles":"code: 14","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Fisher Clinical Services UK Limited","duties_or_roles":"code: 14","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"code: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Azenta US Inc.","duties_or_roles":"Bio repository (value shown)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"National Jewish Health","duties_or_roles":"code: 4","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"code: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"Spain","full_name":"Alcura Health Espana S.A.","duties_or_roles":"code: 14","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"code: 7","organisation_type":"Pharmaceutical company"}
  • {"country":"Hungary","full_name":"PetMobile Kft.","duties_or_roles":"code: 14","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Discovery Life Sciences LLC","duties_or_roles":"code: 4","organisation_type":"Laboratory/Research/Testing facility"}

Investigational products

Investigational Product Name
Rilzabrutinib
Active Substance
RILZABRUTINIB
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
prodAuthStatus: 1 (as recorded in productDictionaryInfo)
Orphan Designation
Yes
Maximum Dose
800 mg (maxDailyDoseAmount)
Investigational Product Name
Sar444671 placebo - matched placebo for test
Modality
Other

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