NCT07128381

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)

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 66 (estimated)
• Sponsor: M.D. Anderson Cancer Center · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

* To find the recommended dose of axatilimab given alone and in combination with ruxolitinib in patients with MF and CMML. * To learn if axatilimab given in combination with ruxolitinib can help to control MF and CMML.

Detailed description

Primary Objectives: * Phase 1 dose escalation: to determine safety, tolerability and Maximum Tolerated Dose (MTD) and efficacy of axatilimab alone and in combination with ruxolitinib for patients with MF and CMML * Phase 2 dose expansion: to determine the overall response rate (ORR) of axatilimab and ruxolitinib in patients with MF and CMML. * Incidence of AEs, MTD and changes in clinical laboratory values. * Measures of efficacy in CMML: overall response rate (ORR) defined as sum of CR + complete cytogenetic remission + partial remission + marrow response + clinical benefit according to the IWG 2015 MDS/MPN response criteria37. * Measures of efficacy in MF: objective response which is defined as CR (complete remission) + PR (partial remission) + CI (clinical improvement) after 6 cycles of treatment. It will be categorized according to the International Working Group (IWG) consensus criteria for myelofibrosis26 Secondary Objectives: CMML: * To determine other efficacy outcomes such as duration of response, leukemia-free survival (LFS), progression-free survival (PFS) and overall survival (OS). * To evaluate symptom burden improvement assessed by Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF TSS). * To evaluate changes in clonal composition and VAF of identified mutations with therapy * To measure changes in cytokine profile and monocyte populations in peripheral blood and bone marrow. MF: * To explore time to response and duration of response * To explore changes in bone marrow fibrosis * To evaluate symptom burden improvement assessed by Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF TSS) (Appendix 1). * To explore changes in dynamic of cytogenetics and molecular mutations: JAK2V617F, CALR, MPL (or other relevant molecular markers) in terms of allele burden or changes in cytogenetic abnormalities * To measure changes in serum and bone marrow cytokine profile pre- and post-therapy

Conditions

• Myelofibrosis (MF)
• Chronic Myelomonocytic Leukemia (CMML)

Interventions

Type	Name	Description
DRUG	Axatilimab (SNDX-6352)	Taken orally
DRUG	Ruxolitinib	Taken orally

Timeline

• Start date: 2026-01-02
• Primary completion: 2031-07-31
• Completion: 2033-07-31
• First posted: 2025-08-19
• Last updated: 2026-01-05

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study