A Multicenter, Open-label, Phase IIb Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia (LUMINA 2)
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.
• 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.