A multi-center, open-label, single-arm study to evaluate the efficacy and safety of oral rilzabrutinib in adults with immune thrombocytopenia (ITP) who failed first-line treatment

immune thrombocytopenia (ITP)

The main study endpoint is to check the durable platelet response ie, to see how many participants can keep their platelet count at a safe level., The durable platelet response is the percentage of participants who can maintain a platelet count of at least 50,000/μL (or between 30,000/μL and 50,000/μL and at least double their starting count) for at least half of their scheduled platelet checks every 2 weeks and for at least 4 valid visits in the last 12 weeks of the study, without needing emergency treatment.

Overall platelet response: The percentage of participants who can achieve 2 platelet counts, at least 5 days apart, of at least 50,000/μL (or between 30,000/μL and 50,000/μL but at least double their starting count) without needing emergency treatment in the 4 weeks before the first high platelet count., Duration of platelet response: The total number and proportion of weeks where participants maintain platelet counts of at least 50,000/μL (or between 30,000/μL and 50,000/μL but at least double their starting count) without needing emergency treatment in the 4 weeks before the high platelet count in participants who achieve a response., Bleeding: The change in immune thrombocytopenia bleeding scale score from the start of the study to the end of Week 28., Corticosteroid-sparing effect: The percentage of participants who can stop or reduce their corticosteroid dose by 50% or to less than 5 mg from the start of the study by the end of Week 28.

Austria, Czechia, France, Germany, Hungary, Italy, Poland, Spain