Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia (wAIHA)

Running, not enrolling · Phase 2

Conditions studied: Warm Autoimmune Hemolytic Anemia (wAIHA)

In brief

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.

Key facts

Study ID

NCT05002777

Run by

Sanofi

People needed

22

Starts

2021-12-07

Expected to finish

2029-12-31

Last updated by the study team

2026-06-05

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• The Oncology Institute of Hope & Innovation Site Number : 8400006 — Cerritos, California, United States
• TOI Clinical Research LLC_ Cerritos_Investigational Site Number: 8400006 — Cerritos, California, United States
• TOI Clinical Research LLC_Glendale_Investigational Site Number: 8400006 — Glendale, California, United States
• TOI Clinical Research LLC_Long Beach_Investigational Site Number: 8400006 — Long Beach, California, United States
• University of Southern California_Investigational Site Number: 8400009 — Los Angeles, California, United States
• TOI Clinical Research LLC_Santa Ana_Investigational Site Number: 8400006 — Santa Ana, California, United States
• The Lundquist Institute_Investigational Site Number: 8400005 — Torrance, California, United States
• TOI Clinical Research LLC_ Whittier_Investigational Site Number: 8400006 — Whittier, California, United States
• Georgetown University Hospital_Investigational Site Number: 8400003 — Washington D.C., District of Columbia, United States
• Oncology & Hematology Associates of West Broward_Investigational Site Number: 8400002 — Tamarac, Florida, United States
• Massachusetts General Hospital_Investigational Site Number: 8400001 — Boston, Massachusetts, United States
• 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

Full record on ClinicalTrials.gov

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