This is a multinational, open label, single arm study that will evaluate the impact of early multi-immune modulation with rilzabrutinib in adult ITP patients who failed first-line treatment. The study includes a screening period (up to 8 weeks), a primary analysis period (up to 28 weeks), a long-term extension period for selected participants (28 weeks) and a 24-week follow-up period only for eligible participants.
Male or female participants aged 18 years and older with a documented diagnosis of primary ITP in the medical history
Participant received at least one course of first-line therapy and had a history of response while on treatment
Participant has loss of response, relapse, or steroid dependency
Participants with Secondary ITP
Participants with Evans syndrome or history of myelodysplastic syndrome
Participants with history of lymphoma, leukemia, or any malignancy within the past 5 years except for non-melanoma skin malignancy.
Participants with history of solid organ transplant
Single group
rilzabrutinib
DrugPharmaceutical form:Tablet-Route of administration:Oral
Durable platelet response
Defined as the percentage of participants able to achieve platelet counts ≥50 000/μL (or ≥30 000/μL and \<50 000/μL and at least double from baseline) for ≥50% of 6 biweekly scheduled platelet measurements and at least 4 non-missing, biweekly visits during the last 12 weeks of the primary analysis period (PAP) in absence of rescue therapy
Overall platelet response
Percentage of participants able to achieve 2 platelet counts at least 5 days apart of ≥50 000/μL (or ≥30 000/μL and \<50 000/μL and at least double from baseline) without rescue therapy in the 4 weeks prior to the first elevated platelet count
Duration of platelet response
Cumulative number and proportion of non-missing weeks with platelet counts ≥50 000/μL (or ≥30 000/μL and \<50 000/μL and at least double from baseline) in absence of rescue therapy in the 4 weeks prior to the elevated platelet count in participants who achieve a response (single platelet count)
Change from baseline in the immune thrombocytopenia bleeding scale (IBLS) score at the end of week 28
Percentage of participants able to discontinue or reduce CS dose by at least 50% or to <5 mg/day (prednisone equivalent) from baseline at the end of week 28
