Study of Axatilimab Alone or With Ruxolitinib to Treat Adults With Myelofibrosis and Chronic Myelomonocytic Leukemia

What this Trial Is About

Researchers are studying axatilimab, a CSF1R monoclonal antibody, alone and in combination with ruxolitinib to find the recommended dose and evaluate its effects in adults with myelofibrosis MF and chronic myelomonocytic leukemia CMML. This phase 12 trial aims to assess safety, tolerability, and overall response rates in these patients while monitoring adverse events and clinical changes related to disease response. Participants will receive axatilimab orally, either alone or with ruxolitinib, also taken orally. Dosing schedules include monotherapy or combination treatment administered approximately 12 hours apart, with dose escalation in phase 1 to identify the maximum tolerated dose, followed by phase 2 dose expansion to evaluate response rates. The study includes treatment of both relapsed and newly diagnosed MF patients and those with refractory CMML. During the trial, participants will undergo safety monitoring including adverse event tracking over about one year. Researchers will evaluate symptom improvements using specific symptom assessment forms, measure changes in blood and bone marrow samples, and study molecular markers. Participants will self-administer medications and attend scheduled visits for assessments, including laboratory tests and clinical evaluations, until study completion in 2033.

Phase I/II Clinical Trial of Axatilimab, a CSF1R Monoclonal Antibody, in Combination With Ruxolitinib as Therapy for Patients With Myelofibrosis (MF) and Chronic Myelomonocytic Leukemia (CMML)