rilzabrutinib: Pharmaceutical form: Tablet Route of administration: Oral
Study summary
All participants will receive rilzabrutinib orally. The screening period is up to 28 days, followed by a treatment period of 24 weeks for Part A. Participants who complete Part A and are deemed eligible for Part B can continue in the Core Part B period followed by an Extended Part B period for up to 253 weeks.
There will be a 7-day safety follow-up period after receiving the last dose of study medication either in Part A (for those not eligible for Part B or early terminated) or Part B. In addition, each participant will be asked to attend an EOT-Core Part B visit when the last participant completes 52 weeks in Core Part B. The Extended Part B period will last for up to 253 weeks.
The study is currently in the Part B Extension segment.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* Male and female patients with a confirmed diagnosis of primary wAIHA or systemic lupus erythematosus (SLE)-associated wAIHA (without other SLE-related manifestations apart from cutaneous and musculoskeletal manifestations)
* Participants who have previously failed to maintain a sustained response after treatment with corticosteroids.
* Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower.
* Up-to-date vaccination status as per local guidelines.
* Body mass index (BMI) \>17.5 and \<40 kg/m2
* All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
Core Part B
* Evidence of treatment efficacy to rilzabrutinib as defined by achieving overall response during Part A.
* Completion of Part A treatment period (24 weeks). Extended Part B
* Completion of Core Part B period.
Exclusion Criteria:
* 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.
* 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.
* Secondary wAIHA from any cause including drugs, 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.
* Myelodysplastic syndrome.
* Uncontrolled or active HBV infection: Patients with positive HBsAg and/or HBV DNA.
* HIV infection.
* Concurrent treatment with other experimental drugs or participation in another clinical trial with any investigational drug within 30 days or 5 half-lives, whichever is greater, prior to treatment start.
* Known allergy to any of the study medications, their analogues, or excipients in the various formulations of any agent.
Part B only
* Participants who receive any therapy during Part A known to be active in wAIHA.
* Presence of unacceptable side effect(s) or toxicity associated with rilzabrutinib such that there is an unfavorable risk-benefit assessment for continued treatment with rilzabrutinib in the opinion of the Investigator and/or Sponsor.
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Primary outcome measure(s)
Part A: Proportion of participants with overall hemoglobin response — By Week 24 in Part A Response is defined as an increase in hemoglobin (Hb) by ≥2 g/dL from baseline and an absence of transfusion in the last 7 days, without biochemical resolution of hemolysis at the time response is achieved and an absence of rescue medications during the last 4 weeks.
Complete Response is defined as hemoglobin ≥11 g/dL (women) or ≥12 g/dL (men), no evidence of hemolysis (normal bilirubin, lactate dehydrogenase (LDH) , haptoglobin, and reticulocytes), and absence of transfusion in the last 7 days and an absence of rescue medications during the last 4 weeks.
Part B: Proportion of participants who maintain durable response achieved during Part A or achieve a durable response during Part B and have a hemoglobin response — By Week 50 in Part B Durable response (Part B) is defined as Hb level ≥10 g/dL with an increase from baseline (Part A) of ≥2 g/dL on three consecutive scheduled visits during Week 24 to Week 50; with absence of transfusion and no rescue medication during the period of 3 consecutive visits and for at least 7 days (transfusions) and 4 weeks (rescue medication) prior to the first consecutive visit.
Trial sites (27)
Facility
City
Region
Status
The Oncology Institute of Hope & Innovation Site Number : 8400006
Cerritos
California
TOI Clinical Research LLC_ Cerritos_Investigational Site Number: 8400006
Cerritos
California
TOI Clinical Research LLC_Glendale_Investigational Site Number: 8400006
Glendale
California
TOI Clinical Research LLC_Long Beach_Investigational Site Number: 8400006
Long Beach
California
University of Southern California_Investigational Site Number: 8400009
Los Angeles
California
TOI Clinical Research LLC_Santa Ana_Investigational Site Number: 8400006
Santa Ana
California
The Lundquist Institute_Investigational Site Number: 8400005
Torrance
California
TOI Clinical Research LLC_ Whittier_Investigational Site Number: 8400006
Whittier
California
Georgetown University Hospital_Investigational Site Number: 8400003
Washington D.C.
District of Columbia
Oncology & Hematology Associates of West Broward_Investigational Site Number: 8400002
Tamarac
Florida
Massachusetts General Hospital_Investigational Site Number: 8400001
Boston
Massachusetts
Hanush-Krankenhaus_Investigational Site Number: 0400001
Vienna
Austria
Peking Union Medical College Hospital_Investigational Site Number: 1560002
Beijing
China
Institute of hematology&blood diseases hospital_Investigational Site Number: 1560003
Tianjin
China
Odense Universitetshospital Hæmatologisk Forskningsenhed_Investigational Site Number: 2080001
Odense
Denmark
Klinik für Hämatologie und Stammzellentransplantation_Investigational Site Number: 2760001
Essen
Germany
Fejer Varmegyei Szent Gyorgy Egyetemi Oktato Korhaz_Investigational Site Number: 3480001
Székesfehérvár
Hungary
Ospedale Giuseppe Moscati_Investigational Site Number: 3800002
Avellino
Italy
Istituto Romagnolo per lo Studio dei Tumori "Dino Amadori"_Investigational Site Number: 3800003
Meldola
Italy
Ospedale Maggiore Policlinico_Investigational Site Number: 3800001
Milan
Italy
Hospital Universitario de Cruces_Investigational Site Number: 7240004
Barakaldo
Spain
Hospital Clinic de Barcelona_Investigational Site Number: 7240001
Barcelona
Spain
Hospital Universitario La Paz_Investigational Site Number: 7240003
Madrid
Spain
Hospital Universitario Virgen del Rocío_Investigational Site Number: 7240002
Seville
Spain
Leeds Teaching Hospitals NHS Trust_Investigational Site Number: 8260001
Leeds
United Kingdom
Barts Health NHS Trust_Investigational Site Number: 8260005
London
United Kingdom
Imperial College Healthcare NHS Trust_Investigational Site Number: 8260002
London
United Kingdom
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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