Clinical trial • Phase III • Immunology|Rare Disease
RILZABRUTINIB for Immune thrombocytopenia (ITP)|Autoimmune thrombocytopenia|Primary immune thrombocytopenia
Phase III trial of RILZABRUTINIB for Immune thrombocytopenia (ITP)|Autoimmune thrombocytopenia|Primary immune thrombocytopenia. open-label.
Overview
- Trial Therapeutic Area
- Immunology|Rare Disease
- Trial Disease
- Immune thrombocytopenia (ITP)|Autoimmune thrombocytopenia|Primary immune thrombocytopenia
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 13-06-2025
- First CTIS Authorization Date
- 07-10-2025
Trial design
open-label Phase III trial in France, Spain, Hungary and others.
- Open Label
- Yes
- Target Sample Size
- 41
Eligibility
Recruits 41 Participants must be capable of giving signed informed consent as described in Appendix 1 Section 10.1 of the protocol; this includes compliance with requirements listed in the ICF and protocol. In countries where the legal age of majority is above 18 years, a specific ICF must also be signed by the participant’s legally authorized representative (Appendix 1 Section 10.1.3). The trial excludes individuals institutionalized by legal order or prisoners (E 27) and employees or immediate family of study site staff (E 29)..
- Pregnancy Exclusion
- E 26. Participant during pregnancy or nursing.
- Vulnerable Population
- Participants must be capable of giving signed informed consent as described in Appendix 1 Section 10.1 of the protocol; this includes compliance with requirements listed in the ICF and protocol. In countries where the legal age of majority is above 18 years, a specific ICF must also be signed by the participant’s legally authorized representative (Appendix 1 Section 10.1.3). The trial excludes individuals institutionalized by legal order or prisoners (E 27) and employees or immediate family of study site staff (E 29).
Inclusion criteria
- {"criterion_text":"- I 01. Participant must be 18 or above years of age inclusive, at the time of signing the informed consent.\n- I 10. Capable of giving signed informed consent as described in Appendix 1 Section 10.1 of the protocol which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. In countries where legal age of majority is above 18 years, a specific ICF must also be signed by the participant’s legally authorized representative (Appendix 1 Section 10.1.3).\n- I 02. Male and female participants with a documented diagnosis of primary ITP in the medical history.\n- I 03. Participants with Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower.\n- I 04. Participant received at least one course of first-line therapy per international guidelines or local standard of care (eg, oral or parenteral CS, IVIg, anti-D). Note: Participants may have received multiple courses of first-line therapy, either sequentially or in combination.\n- I 05. History of platelet response while on-treatment with first-line therapy.\n- I 06. Participant has loss of response, relapse, or steroid dependency.\n- I 07. Please see study protocol.\n- I 08. All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.\n- I 09. Adequate hematologic, hepatic, and renal function (hemoglobin >9 g/dL within 1 week prior to Study Day 1), absolute neutrophil count ≥1500/μL,, AST and ALT ≤1.5×upper limit of normal (ULN), albumin ≥3 g/dL, total bilirubin ≤1.5×ULN (unless the participant has documented Gilbert syndrome), estimated glomerular filtration rate (GFR) >50mL/min/1.73m² (CKD-EPI 2021 Creatinine Equation [Race-Free]), International normalized ratio (INR) and activated partial thromboplastin time (APTT) values are within 20% of the normal ranges."}
Exclusion criteria
- {"criterion_text":"- E 01. Participants with diagnosis of secondary ITP.\n- E 18. Participant received subsequent/advanced treatment for the treatment of ITP or was splenectomized before Study Day 1\n- E 19. Participant received drugs known to potentially improve thrombocytopenia to treat other conditions within 5 times the elimination half-life of the drug or within 14 days of Study Day 1, whichever is longer, or the participant is planned to receive treatment with drugs known to potentially improve thrombocytopenia for any indication during the course of the study.\n- E 10. HIV infection.\n- E 20. Please see study protocol.\n- E 21. Please see study protocol.\n- E 22. Please see study protocol.\n- E 23. Please see study protocol.\n- E 24. Please see study protocol.\n- E 25. Received any investigational drug within 6 months or 5 half-lives, whichever is longer, or is participating in another clinical trial.\n- E 26. Participant during pregnancy or nursing.\n- E 02. Participants with a diagnosis of Evans syndrome.\n- E 27. Individuals accommodated in an institution because of regulatory or legal order; prisoners or participants who are legally institutionalized.\n- E 28. Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.\n- E 11. A history of active or latent tuberculosis (TB) (unless the participant has completed a full course of anti-tuberculosis therapy or it is documented by a specialist that the participant has been adequately treated and can begin treatment with an immunosuppressive agent). A positive test for TB (QuantiFERON-TB-Gold [QFT] or equivalent) performed at the screening visit or within the 3 months prior to screening.\n- E 29. Participants are employees of the clinical study site or other individuals directly involved in the conduct of the study, or immediate family members of such individuals (in conjunction with section 1.61 of the International Council for Harmonization Good Clinical Practice [GCP] Ordinance E6).\n- E 30. Sensitivity to any of the study intervention, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study.\n- E 12. Participants with uncontrolled or active HBV infection: Participants with positive HBsAg and/or HBV DNA.\n- E 13. Participants with active HCV infection: positive HCV-RNA and positive anti-HCV.\n- E 14. Current drug or alcohol abuse.\n- E 15. Refractory nausea and vomiting, malabsorption, external biliary shunt, significant bowel resection, or any other condition that would preclude adequate study drug absorption.\n- E 16. Participants who have undergone major surgery within 28 days prior to Study Day 1 or have planned surgery in the time frame of the study.\n- E 03. Participants with history of myelodysplastic syndrome\n- E 17. Please see study protocol.\n- E 04. Participant with history of or current life-threatening bleeding (eg., a bleed that results in hemodynamic instability or respiratory compromise) due to ITP.\n- E 05. 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.\n- E 06. Participants with history of solid organ transplant.\n- E 07. Participants with history of coagulation or bleeding disorders, including genetic conditions, other than ITP.\n- E 08. Please see the study protocol.\n- E 09. Positive COVID-19 molecular test (if COVID-19 testing required per local guidelines to be determined for each site)."}
Endpoints
Primary endpoints
- {"endpoint_text":"- The main study endpoint is to check the durable platelet response ie, to see how many participants can keep their platelet count at a safe level.","definition_or_measurement_approach":"The durable platelet response is the percentage of participants who can maintain a platelet count of at least 50,000 /μL (or between 30,000/μL and 50,000/μL and at least double their starting count) for at least half of their scheduled platelet checks every 2 weeks and for at least 4 valid visits in the last 12 weeks of the study, without needing rescue treatment."}
- {"endpoint_text":"- The durable platelet response is the percentage of participants who can maintain a platelet count of at least 50,000 /μL (or between 30,000/μL and 50,000/μL and at least double their starting count) for at least half of their scheduled platelet checks every 2 weeks and for at least 4 valid visits in the last 12 weeks of the study, without needing rescue treatment.","definition_or_measurement_approach":"Durable platelet response defined as above: percentage of participants meeting the platelet count criteria (≥50,000/μL or 30,000–50,000/μL with ≥2× baseline) for at least half of scheduled biweekly platelet checks and at least 4 valid visits in the last 12 study weeks, without rescue treatment."}
Secondary endpoints
- {"endpoint_text":"- 1.\tOverall platelet response: The percentage of participants who can achieve 2 platelet counts, at least 5 days apart, of at least 50,000/μL (or between 30,000/μL and 50,000/μL but at least double their starting count) without needing rescue treatment in the 4 weeks before the first high platelet count.","definition_or_measurement_approach":"Percentage of participants achieving two platelet counts ≥50,000/μL (or 30,000–50,000/μL with ≥2× baseline) at least 5 days apart, without rescue treatment in the 4 weeks prior to first high count."}
- {"endpoint_text":"- 2.\tDuration of platelet response: The total number and proportion of weeks where participants maintain platelet counts of at least 50,000/μL (or between 30,000/μL and 50,000/μL but at least double their starting count) without needing rescue treatment in the 4 weeks before the high platelet count in participants who achieve a response.","definition_or_measurement_approach":"Total number and proportion of weeks with platelet counts meeting response criteria (≥50,000/μL or 30,000–50,000/μL with ≥2× baseline) without rescue treatment in the 4 weeks before the high count, among responders."}
- {"endpoint_text":"- 3.\tBleeding: The change in immune thrombocytopenia bleeding scale score from the start of the study to the end of Week 28.","definition_or_measurement_approach":"Change from baseline to end of Week 28 in the immune thrombocytopenia bleeding scale (IBLS) score."}
- {"endpoint_text":"- 4.\tCorticosteroid-sparing effect: The percentage of participants who can stop or reduce their corticosteroid dose by at least 50% or to less than 5 mg/day (prednisone equivalent) from the start of the study by the end of Week 28","definition_or_measurement_approach":"Percentage of participants who discontinue or reduce corticosteroid dose by ≥50% or to <5 mg/day prednisone-equivalent from baseline to Week 28."}
Recruitment
- Planned Sample Size
- 41
- Recruitment Window Months
- 36
- Consent Approach
- Participants must provide signed informed consent as described in Appendix 1 Section 10.1 of the protocol. The ICF includes compliance requirements and restrictions; in countries where the legal age of majority is above 18 years a specific ICF must also be signed by the participant’s legally authorized representative (Appendix 1 Section 10.1.3). Subject information and ICF documents are provided (multiple L1/L2 ICF documents and language-specific versions are present in the dossier, including English, Spanish, French, Polish, Italian, Czech, German, Hungarian).
Geography
- Total Number Of Sites
- 42
- Total Number Of Participants
- 41
France
- Earliest CTIS Part Ii Submission Date
- 19-09-2025
- Latest Decision Or Authorization Date
- 10-10-2025
- Processing Time Days
- 21
- Number Of Sites
- 4
- Number Of Participants
- 5
Sites
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- 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 Bordeaux
- Department Name
- Medecine interne et maladies infectieuses
- Principal Investigator Name
- Jean-Francois VIALLARD
- Principal Investigator Email
- Jean-francois.viallard@chu-bordeaux.fr
- Contact Person Name
- Jean-Francois VIALLARD
- Contact Person Email
- Jean-francois.viallard@chu-bordeaux.fr
- Site Name
- Centre Hospitalier Universitaire De Dijon
- Department Name
- Médecine interne et immunologie clinique
- Principal Investigator Name
- Sylvain AUDIA
- Principal Investigator Email
- Sylvain.audia@chu-dijon.fr
- Contact Person Name
- Sylvain AUDIA
- Contact Person Email
- Sylvain.audia@chu-dijon.fr
- Site Name
- Centre Hospitalier Universitaire De Toulouse
- Department Name
- Médecine interne
- Principal Investigator Name
- Guillaume MOULIS
- Principal Investigator Email
- moulis.g@chu-toulouse.fr
- Contact Person Name
- Guillaume MOULIS
- Contact Person Email
- moulis.g@chu-toulouse.fr
Spain
- Earliest CTIS Part Ii Submission Date
- 22-09-2025
- Latest Decision Or Authorization Date
- 09-10-2025
- Processing Time Days
- 17
- Number Of Sites
- 9
- Number Of Participants
- 11
Sites
- Site Name
- Hospital Universitario De Canarias
- Department Name
- Hematology
- Principal Investigator Name
- Sunil Lakhwani
- Principal Investigator Email
- sunillakhwani@hotmail.com
- Contact Person Name
- Sunil Lakhwani
- Contact Person Email
- sunillakhwani@hotmail.com
- Site Name
- Hospital Universitario La Paz
- Department Name
- Hematology
- Principal Investigator Name
- Maria Teresa Alvarez Roman
- Principal Investigator Email
- talvarez.ensayos@gmail.com
- Contact Person Name
- Maria Teresa Alvarez Roman
- Contact Person Email
- talvarez.ensayos@gmail.com
- Site Name
- Hospital Del Mar
- Department Name
- Hematology
- Principal Investigator Name
- Blanca Sanchez Gonzalez
- Principal Investigator Email
- 97894@hospitaldelmar.cat
- Contact Person Name
- Blanca Sanchez Gonzalez
- Contact Person Email
- 97894@hospitaldelmar.cat
- Site Name
- Hospital General Universitario Morales Meseguer
- Department Name
- Hematology
- 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
- University Hospital Virgen Del Rocio S.L.
- Department Name
- Hematology
- Principal Investigator Name
- Maria Eva Mingot Castellano
- Principal Investigator Email
- mariae.mingot.sspa@juntadeandalucia.es
- Contact Person Name
- Maria Eva Mingot Castellano
- Contact Person Email
- mariae.mingot.sspa@juntadeandalucia.es
- Site Name
- Hospital General Universitario Gregorio Maranon
- Department Name
- Hematology
- Principal Investigator Name
- Cristina Pascual Izquierdo
- Principal Investigator Email
- cpascuali@salud.madrid.org
- Contact Person Name
- Cristina Pascual Izquierdo
- Contact Person Email
- cpascuali@salud.madrid.org
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- Hematology
- Principal Investigator Name
- David Valcarcel Ferreiras
- Principal Investigator Email
- dvalcarcel@vhio.net
- Contact Person Name
- David Valcarcel Ferreiras
- Contact Person Email
- dvalcarcel@vhio.net
- Site Name
- Hospital Universitario De Salamanca
- Department Name
- Hematology
- Principal Investigator Name
- Jose Ramon Gonzalez Porras
- Principal Investigator Email
- jrgp@usal.es
- Contact Person Name
- Jose Ramon Gonzalez Porras
- Contact Person Email
- jrgp@usal.es
- Site Name
- Hospital Universitario De Burgos
- Department Name
- Hematology
- Principal Investigator Name
- Tomas Jose Gonzalez Lopez
- Principal Investigator Email
- tjgonzalez@saludcastillayleon.es
- Contact Person Name
- Tomas Jose Gonzalez Lopez
- Contact Person Email
- tjgonzalez@saludcastillayleon.es
Hungary
- Earliest CTIS Part Ii Submission Date
- 25-09-2025
- Latest Decision Or Authorization Date
- 15-05-2026
- Processing Time Days
- 232
- Number Of Sites
- 3
- Number Of Participants
- 2
Sites
- Site Name
- University Of Debrecen
- Department Name
- Haematology
- Principal Investigator Name
- Árpád Illés
- Principal Investigator Email
- illes.arpad@med.unideb.hu
- Contact Person Name
- Árpád Illés
- Contact Person Email
- illes.arpad@med.unideb.hu
- Site Name
- Somogy Varmegyei Kaposi Mor Oktato Korhaz
- Department Name
- Haematology
- Principal Investigator Name
- Peter Rajnics
- Principal Investigator Email
- rajnicsp@hotmail.com
- Contact Person Name
- Peter Rajnics
- Contact Person Email
- rajnicsp@hotmail.com
- Site Name
- Semmelweis University
- Department Name
- Department of Internal Medicine and Haematology
- Principal Investigator Name
- Zsolt Gyorgy Nagy
- Principal Investigator Email
- nagy.zsolt@med.semmelweis-univ.hu
- Contact Person Name
- Zsolt Gyorgy Nagy
- Contact Person Email
- nagy.zsolt@med.semmelweis-univ.hu
Italy
- Earliest CTIS Part Ii Submission Date
- 29-08-2025
- Latest Decision Or Authorization Date
- 12-05-2026
- Processing Time Days
- 256
- Number Of Sites
- 10
- Number Of Participants
- 8
Sites
- Site Name
- Azienda Unita Locale Socio Sanitaria N 8 Berica
- Department Name
- UOC 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
- Azienda Ospedaliero-Universitaria Policlinico Umberto I
- Department Name
- UOC Hematology
- Principal Investigator Name
- Cristina Santoro
- Principal Investigator Email
- santoro@bce.uniroma1.it
- Contact Person Name
- Cristina Santoro
- Contact Person Email
- santoro@bce.uniroma1.it
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- Hematology
- 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
- UOC Hematology
- Principal Investigator Name
- Francesca Palandri
- Principal Investigator Email
- francesca.palandri@unibo.it
- Contact Person Name
- Francesca Palandri
- Contact Person Email
- francesca.palandri@unibo.it
- Site Name
- Azienda Ospedaliera Universitaria Federico II Di Napoli
- Department Name
- Hematology and Bone Marrow Transplant
- Principal Investigator Name
- Fabrizio Pane
- Principal Investigator Email
- fabrizio.pane@unina.it
- Contact Person Name
- Fabrizio Pane
- Contact Person Email
- fabrizio.pane@unina.it
- Site Name
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
- Department Name
- SC Ematologia
- Principal Investigator Name
- Bruno Fattizzo
- Principal Investigator Email
- bruno.fattizzo@policlinico.mi.it
- Contact Person Name
- Bruno Fattizzo
- Contact Person Email
- bruno.fattizzo@policlinico.mi.it
- Site Name
- ASST Grande Ospedale Metropolitano Niguarda
- Department Name
- Dipartimento di Ematologia, Oncologia e Medicina Molecolare
- Principal Investigator Name
- Monica Carpenedo
- Principal Investigator Email
- monica.carpenedo@ospedaleniguarda.it
- Contact Person Name
- Monica Carpenedo
- Contact Person Email
- monica.carpenedo@ospedaleniguarda.it
- Site Name
- Azienda Sanitaria Universitaria Giuliano Isontina
- Department Name
- UOC Ematologia
- 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
- Istituto Romagnolo Per Lo Studio Dei Tumori Dino Amadori IRST S.r.l.
- Department Name
- Oncologia Medica
- Principal Investigator Name
- Alessandro Lucchesi
- Principal Investigator Email
- alessandro.lucchesi@irst.emr.it
- Contact Person Name
- Alessandro Lucchesi
- Contact Person Email
- alessandro.lucchesi@irst.emr.it
- Site Name
- Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
- Department Name
- S.C. Ematologia U
- Principal Investigator Name
- Alessandra Borchiellini
- Principal Investigator Email
- aborchiellini@cittadellasalute.to.it
- Contact Person Name
- Alessandra Borchiellini
- Contact Person Email
- aborchiellini@cittadellasalute.to.it
Germany
- Earliest CTIS Part Ii Submission Date
- 24-09-2025
- Latest Decision Or Authorization Date
- 12-05-2026
- Processing Time Days
- 230
- Number Of Sites
- 6
- Number Of Participants
- 6
Sites
- Site Name
- Technische Universitaet Dresden
- Department Name
- Medizinische Klinik und Poliklinik I
- Principal Investigator Name
- Karolin Trautmann Grill
- Principal Investigator Email
- Karolin.trautmann@ukdd.de
- Contact Person Name
- Karolin Trautmann Grill
- Contact Person Email
- Karolin.trautmann@ukdd.de
- Site Name
- Justus-Liebig-Universitaet Giessen
- Department Name
- Marburg UKGM
- Principal Investigator Name
- Mathias J. Rummel
- Principal Investigator Email
- Mathias.rummel@innere.med.uni-giessen.de
- Contact Person Name
- Mathias J. Rummel
- Contact Person Email
- Mathias.rummel@innere.med.uni-giessen.de
- Site Name
- Goethe University Frankfurt
- Department Name
- ZIM Med II
- Principal Investigator Name
- Wolfgang Miesbach
- Principal Investigator Email
- Wolfgang.miesbach@unimedizin-ffm.de
- Contact Person Name
- Wolfgang Miesbach
- Contact Person Email
- Wolfgang.miesbach@unimedizin-ffm.de
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Charite Universitiy Medicine Berlin
- Principal Investigator Name
- Marie Luise Huetter-Kroenke
- Principal Investigator Email
- Luise.huetter-kroenke@charite.de
- Contact Person Name
- Marie Luise Huetter-Kroenke
- Contact Person Email
- Luise.huetter-kroenke@charite.de
- Site Name
- Universitaetsklinikum Essen AöR
- Department Name
- Klinik fuer Haematologie und Stammzelltransplantation
- Principal Investigator Name
- Alexander Roeth
- Principal Investigator Email
- Alexander.roeth@uk-essen.de
- Contact Person Name
- Alexander Roeth
- Contact Person Email
- Alexander.roeth@uk-essen.de
- Site Name
- MVZ Leipzig Mitte
- Department Name
- MVZ Leipzig
- Principal Investigator Name
- Baerbel Schaedlich
- Principal Investigator Email
- Baerbel.schaedlich@doceins.de
- Contact Person Name
- Baerbel Schaedlich
- Contact Person Email
- Baerbel.schaedlich@doceins.de
Austria
- Earliest CTIS Part Ii Submission Date
- 29-09-2025
- Latest Decision Or Authorization Date
- 13-05-2026
- Processing Time Days
- 226
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Medical University Of Vienna
- Department Name
- Department of Medicine I
- Principal Investigator Name
- Johanna Gebhart
- Principal Investigator Email
- Johanna.gebhart@meduniwien.ac.at
- Contact Person Name
- Johanna Gebhart
- Contact Person Email
- Johanna.gebhart@meduniwien.ac.at
- Site Name
- Hanusch Krankenhaus Der Wiener Gebietskrankenkasse
- Department Name
- 3rd Medical Department
- Principal Investigator Name
- Michael Fillitz
- Principal Investigator Email
- michael.fillitz@oegk.at
- Contact Person Name
- Michael Fillitz
- Contact Person Email
- michael.fillitz@oegk.at
Poland
- Earliest CTIS Part Ii Submission Date
- 16-09-2025
- Latest Decision Or Authorization Date
- 15-05-2026
- Processing Time Days
- 241
- Number Of Sites
- 5
- Number Of Participants
- 4
Sites
- Site Name
- Wojewodzki Szpital Specjalistyczny Im. Janusza Korczaka W Slupsku Sp. z o.o.
- Department Name
- Oddzial Hematologiczny
- Principal Investigator Name
- Wojciech Homenda
- Principal Investigator Email
- hematologia@szpital.slupsk.pl
- Contact Person Name
- Wojciech Homenda
- Contact Person Email
- hematologia@szpital.slupsk.pl
- Site Name
- Uniwersyteckie Centrum Kliniczne
- Department Name
- Klinika Hematologii i Transplantologii
- Principal Investigator Name
- Andrej Mital
- Principal Investigator Email
- amital@uck.gda.pl
- Contact Person Name
- Andrej Mital
- Contact Person Email
- amital@uck.gda.pl
- Site Name
- Wojewodzkie Wielospecjalistyczne Centrum Onkologii I Traumatologii Im M.Kopernika W Lodzi
- Department Name
- Oddział Hematologii Ogolnej i Chorob Wewnetrznych
- Principal Investigator Name
- Tadeusz Robak
- Principal Investigator Email
- robaktad@csk.umed.lodz.pl
- Contact Person Name
- Tadeusz Robak
- Contact Person Email
- robaktad@csk.umed.lodz.pl
- Site Name
- Copernicus Podmiot Leczniczy Sp. z o.o.
- Department Name
- Wojewodzkie Centrum Onkologii
- Principal Investigator Name
- Hanna Ciepluch
- Principal Investigator Email
- ciepluch@gumed.edu.pl
- Contact Person Name
- Hanna Ciepluch
- Contact Person Email
- ciepluch@gumed.edu.pl
- Site Name
- Aidport Sp. z o.o.
- Principal Investigator Name
- Michał Kwiatek
- Principal Investigator Email
- michal.kwiatek@aidport.pl
- Contact Person Name
- Michał Kwiatek
- Contact Person Email
- michal.kwiatek@aidport.pl
Czechia
- Earliest CTIS Part Ii Submission Date
- 23-09-2025
- Latest Decision Or Authorization Date
- 11-05-2026
- Processing Time Days
- 230
- Number Of Sites
- 3
- Number Of Participants
- 3
Sites
- Site Name
- Fakultni Nemocnice Ostrava
- Department Name
- Klinika hematoonkologie FNO a LF OUFN Ostrava
- Principal Investigator Name
- Ivo Demel
- Principal Investigator Email
- ivo.demel@fno.cz
- Contact Person Name
- Ivo Demel
- Contact Person Email
- ivo.demel@fno.cz
- Site Name
- Fakultni Nemocnice Brno
- Department Name
- Interni hematologicka a onkologicka klinika FN Brno
- Principal Investigator Name
- Libor Cervinek
- Principal Investigator Email
- cervinek.libor@fnbrno.cz
- Contact Person Name
- Libor Cervinek
- Contact Person Email
- cervinek.libor@fnbrno.cz
- Site Name
- Vseobecna Fakultni Nemocnice V Praze
- Department Name
- I. interni klinika - klinika hematologie
- Principal Investigator Name
- Marek Trneny
- Principal Investigator Email
- trneny@cesnet.cz
- Contact Person Name
- Marek Trneny
- Contact Person Email
- trneny@cesnet.cz
Sponsor
Primary sponsor
- Full Name
- Sanofi-Aventis Recherche & Developpement
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- France
Investigational products
- Investigational Product Name
- Rilzabrutinib
- Active Substance
- RILZABRUTINIB
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- ORAL USE
- Orphan Designation
- Yes
- Maximum Dose
- 800 mg (max daily dose amount 800 mg)
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- ALLOGENEIC ADIPOCYTE-DERIVED MESENCHYMAL STROMAL CELLS TRANSDUCED WITH A LENTIVIRAL PROVIRUS VECTOR CONTAINING THE HUMAN CXCR4 AND IL-10 GENES for Acute graft-versus-host disease (aGVHD) | Steroid-refractory acute graft-versus-host disease | Ruxolitinib-refractory acute graft-versus-host disease
- DEUCRICTIBANT for Acquired angioedema due to C1 inhibitor deficiency