Phase 1 Study to Evaluate Safety and Effectiveness of Karonudib for Treating Blood Cancers Including Leukemia and Myelodysplastic Syndrome

What this Trial Is About

Researchers are evaluating the safety and tolerability of Karonudib, an oral drug, for treating patients with advanced blood cancers including Acute Myeloid Leukemia AML, Acute Lymphoblastic Leukemia ALL, Diffuse Large B-Cell Lymphoma DLBCL, Multiple Myeloma MM, high-risk Myelodysplastic Syndrome MDS, and Burkitts lymphoma. The study also aims to find a recommended dose and schedule for further development, understand how the drug is processed in the body, and look for early signs of treatment effect. Overall survival of patients will also be recorded during the trial. The study includes a dose escalation phase where Karonudib is given as an oral solution every other day in three planned dose groups. An extension phase involves Karonudib taken twice a week together with standard care treatment Idarubicin on days 1 to 3. Patients will receive the drug orally and the study is designed sequentially to evaluate safety and tolerability first before further phases. This is a Phase 1 interventional study focused on treatment. Participants will be monitored closely for safety and tolerability during the first 28-day treatment cycle, which is the primary outcome period. Researchers will collect blood and bone marrow samples or imaging to measure disease and treatment response. Additional assessments include heart function, liver and kidney tests, and pregnancy tests for women of childbearing potential. The trial lasts until April 2027 and includes repeated assessments to understand the drugs effects and patient survival.

A Study in Leukemia Patients With Karonudib