Phase 3 Study to Evaluate Oral Rilzabrutinib Versus Placebo in Adults With Warm Autoimmune Hemolytic Anemia

What this Trial Is About

Researchers are evaluating oral rilzabrutinib in a Phase 3 study involving adults aged 18 and older with primary warm autoimmune hemolytic anemia wAIHA. The study aims to compare the drugs effectiveness, safety, pharmacokinetics, and pharmacodynamics to a placebo in achieving a durable hemoglobin response. Approximately 90 participants will take part, with a focus on those who have a confirmed diagnosis of primary wAIHA and meet specific criteria related to corticosteroid treatment history and health status. Participants will first undergo a 4-week screening period. Eligible individuals will then be randomly assigned in a 21 ratio to receive either oral rilzabrutinib or a matching placebo twice daily during the primary analysis period lasting up to 24 weeks. Those who complete this phase will enter an open-label period for 28 weeks, during which all receive rilzabrutinib. Following this, participants who show an increase in hemoglobin during the last 8 weeks of the open-label period may continue in a long-term extension phase lasting up to 52 weeks. A safety follow-up will occur 2 weeks after treatment ends or is stopped. During the study, participants will have scheduled visits to assess hemoglobin levels, fatigue, dyspnea, and other health indicators using questionnaires and laboratory tests. Researchers will monitor adverse events, vital signs, physical exams, and electrocardiograms throughout the study and extension periods. The primary outcome is the proportion of participants achieving a durable hemoglobin response, defined as an increase of at least 2 gdL from baseline in most visits between weeks 12 and 24. Secondary outcomes include overall hemoglobin response, fatigue changes, and need for rescue therapy. Total study duration varies by participant based on their progression through the study phases.

A Study to Investigate the Efficacy, Safety, and Pharmacokinetics of Oral Rilzabrutinib Compared With Placebo in Participants 18 Years of Age and Older With Warm Autoimmune Hemolytic Anemia