Study of Axatilimab With or Without Azacitidine to Treat Advanced Myeloproliferative Neoplasms, MPNMDS Overlap, or High-Risk Chronic Myelomonocytic Leukemia

What this Trial Is About

Researchers are studying advanced phase myeloproliferative neoplasms MPN, myeloproliferative neoplasmmyelodysplastic syndrome MPNMDS overlap, and high-risk chronic myelomonocytic leukemia CMML in this phase IbII trial. The trial aims to find the best dose of axatilimab and to evaluate the effectiveness of axatilimab alone or combined with azacitidine in treating these conditions. Axatilimab is an antibody designed to block a protein involved in cancer cell growth, while azacitidine is a medication that slows cancer cell growth. The study evaluates safety, response rates, quality of life, symptom relief, and biological changes during treatment. In phase I, patients receive axatilimab intravenously over 30 minutes on days 1 and 15 of each 28-day cycle. Patients showing clinical improvement may then enter phase II. In phase II, patients receive axatilimab on days 1 and 15 plus azacitidine given intravenously or by injection on days 1 to 7 of each cycle. Treatment continues for up to 4 cycles initially, with possible extension to 24 cycles if patients respond. Throughout both phases, patients undergo bone marrow biopsies, aspirations, and blood sample collections to monitor disease and treatment effects. Participants will be closely monitored with assessments including blood tests, bone marrow samples, and quality of life questionnaires. Researchers track outcomes such as dose limiting toxicities, overall response rates, transfusion needs, hospital time, and side effects. After treatment ends, patients have follow-up visits at 30 days and then every 6 months for up to 24 months. The study aims to understand treatment safety and how well it manages symptoms and disease progression over time.

Axatilimab With or Without Azacitidine for the Treatment of Patients With Advanced Phase Myeloproliferative Neoplasms, Myeloproliferative Neoplasm/Myelodysplastic Syndrome Overlap or High Risk Chronic Myelomonocytic Leukemia