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

Inclusion Criteria:

• Male and female patients with a confirmed diagnosis of primary wAIHA or systemiclupus erythematosus (SLE)-associated wAIHA (without other SLE-related manifestationsapart from cutaneous and musculoskeletal manifestations)

• Participants who have previously failed to maintain a sustained response aftertreatment 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 regulationsregarding the methods of contraception for those participating in clinical studies.

Core Part B

• Evidence of treatment efficacy to rilzabrutinib as defined by achieving overallresponse 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 wouldjeopardize the safety of the participant or compromise the quality of the dataderived from his or her participation in the study as determined by theInvestigator.

• Participants with medical history of lymphoma, leukemia, or any malignancy withinthe past 5 years except for basal cell or squamous epithelial carcinomas of the skinthat 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 autoimmunedisease, or active hematologic malignancies. Participants with positive antinuclearantibodies 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 anotherclinical 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 thevarious 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 rilzabrutinibsuch that there is an unfavorable risk-benefit assessment for continued treatmentwith 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.