Immune thrombocytopenia is an autoimmune disorder characterized by isolated thrombocytopenia due to immune-mediated platelet destruction and impaired platelet production. Patients can present with petechiae, bruising, mucosal bleeding, or severe hemorrhage in rare cases. Disease may be acute or chronic, and treatment is tailored to bleeding risk and platelet count.
Sanofi's oral BTK inhibitor rilzabrutinib received orphan drug designation in Japan for IgG4-related disease and warm autoimmune hemolytic anemia, while also securing U.S. FDA breakthrough therapy designation for the rare anemia indication.
Japan's Ministry of Health, Labour and Welfare has granted orphan drug designation to Sanofi's rilzabrutinib for IgG4-related disease, marking the third global orphan designation for this indication based on positive phase 2 study data.
International researchers have identified a specific antibody mutation that causes rare but serious blood clots after adenovirus-based COVID-19 vaccines or natural adenovirus infection, providing a roadmap for safer vaccine design.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07599280 |
Universal STAR-T Cell Injection in R/R Autoimmune Diseases. |
NOT_YET_RECRUITING | PHASE1 |
| NCT07597395 |
ATRA for Management of Primary ITP |
NOT_YET_RECRUITING | PHASE3 |
| NCT07587723 |
A Real-world Study of Treatment Patterns and Outcomes in Primary Immune Thrombocytopenia Patients Treated With Eltrombopag |
NOT_YET_RECRUITING | |
| NCT07362238 |
Daratumumab Versus Rituximab in the Management of Pediatric Primary Immune Thrombocytopenia (ITP) |
RECRUITING | PHASE2 |
| NCT07362199 |
Anti-CD38 Monoclonal Antibody Combined With Rituximab in the Management of Primary Immune Thrombocytopenia (ITP) |
RECRUITING | PHASE2 |
| NCT07315373 |
Optimal Dose of Zanubrutinib to Treat Adult Immune Thrombocytopenia |
NOT_YET_RECRUITING | PHASE1 |
| NCT07298733 |
The Value of Interleukin-1β and Interleukin-33 Genetic Expression in the Pathogenesis and Differentiation of Primary ITP and SLE-Related Thrombocytopenia |
NOT_YET_RECRUITING | |
| NCT07297563 |
Anti-CD38 Monoclonal Antibody Versus Rituximab in the Management of Primary Immune Thrombocytopenia (ITP) |
RECRUITING | PHASE2 |
| NCT07234019 |
Rituximab Combining Anti-CD38 Monoclonal Antibody Versus Rituximab in the Management of Primary Immune Thrombocytopenia (ITP) |
RECRUITING | PHASE2 |
| NCT07216079 |
Rilzabrutinib for the Adult Participants With Chronic ITP Who Have Completed Phase 3 Study in Japan |
ACTIVE_NOT_RECRUITING | PHASE3 |
