A Phase 1/2 interventional study of Axatilimab and Azacitidine in Atypical Chronic Myeloid Leukemia, Chronic Myelomonocytic Leukemia and Myelodysplastic/Myeloproliferative Neoplasm, sponsored by Uma Borate. Recruiting at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-08-27.
Sponsored by Uma Borate · Phase 1/2, Interventional, and Treatment
This phase Ib/II trial tests the best dose of axatilimab and effectiveness of axatilimab with or without azacitidine for the treatment of patients with advanced phase myeloproliferative neoplasms (MPN), myeloproliferative neoplasm/myelodysplastic syndrome (MPN/MDS) overlap or high risk chronic myelomonocytic leukemia (CMML). Axatilimab is an antibody that is cloned from a single white blood cell that is known to be able to recognize cancer cells and block a protein on the surface of the white blood cells that may be involved in cancer cell growth. By blocking the proteins, this may slow or halt the growth of the cancer. Azacitidine is in a class of medications called antimetabolites. It works by stopping or slowing the growth of cancer cells. Giving axatilimab with or without azacitidine may be safe and effective in treating patients with advanced phase MPN, MPN/MDS overlap or high risk CMML.
PRIMARY OBJECTIVES:
I. To determine the recommended phase 2 dose (RP2D) of axatilimab in relapsed or refractory patients with advanced phase MPN, MPN/MDS overlap or high-risk CMML.
II. To evaluate the overall response rate of axatilimab + azacitidine (AZA) in newly diagnosed patients with advanced phase MPN, MPN/MDS overlap or high-risk CMML using Savona response criteria.
SECONDARY OBJECTIVES:
I. Determine the safety and tolerability of axatilimab + azacitidine combination therapy in those with newly diagnosed advanced phase MPN, MPN/MDS overlap or high-risk CMML.
II. Determine the quality of life in patients receiving axatilimab + AZA using a Modified Myeloproliferative Neoplasm Symptom Assessment Form (MPNSAF).
III. Determine the effect of axatilimab + azacitidine on symptom relief which will be measured by transfusion burden (platelet and/or packed red blood cell), time in hospital, and immune related side effects specifically reactivation of tuberculosis (TB), infusion-related reactions, hepatotoxicity, pneumonia, pyrexia, sepsis, shortness of breath (SBO), hemoptysis, periorbital edema, fatigue, and pancreatitis.
EXPLORATORY OBJECTIVES:
I. To assess the pharmacokinetics of axatilimab in combination with AZA. II. To describe the prevalence and trajectories of patients' physical activity during treatment.
III. To perform detailed correlative studies related to genetic, biochemical, and immunologic changes that occur with axatilimab in combination with AZA.
OUTLINE: This is a phase I, dose-escalation study of axatilimab followed by a phase II study.
PHASE I: Patients receive axatilimab intravenously (IV) over 30 minutes on days 1 and 15 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. At time of phase Ib completion, patients with clinical improvement may transition to phase II. Patients undergo bone marrow biopsy and aspiration and blood sample collection throughout the study.
PHASE II: Patients receive axatilimab IV over 30 minutes on days 1 and 15 and azacitidine IV over 10-40 minutes or subcutaneously (SC) on days 1-7 of each cycle. Cycles repeat every 28 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity. Patients who achieve partial response (PR) or better may continue for up to 24 total cycles. Patients who achieve less than PR receive 2 additional cycles and, if PR or better is achieved, may complete up to 24 total cycles. Patients undergo bone marrow biopsy and aspiration and blood sample collection throughout the study.
After completion of study treatment, patients are followed up at 30 days and then every 6 months for up to 24 months after last dose of study medication.
44 studies on the registry are indexed under Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative; 8 are open to participants now.
This study's planned enrollment of 49 is above the median of 35 across 37 interventional studies indexed under Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative.
Browse Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative studies →Uma Borate is the lead sponsor of 7 studies on the registry; 4 are open to participants now.
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Morphologically confirmed diagnosis of the following based on 2016 World Health Organization (WHO) classification (Arber et al 2016): Phase 1b, patients with relapsed or refractory of any of the following; phase 2, patients with newly diagnosed of any of the following:
Exclusion Criteria:
Participants with prior malignancy, except:
Patients receive axatilimab IV over 30 minutes on days 1 and 15 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. At time of phase Ib completion, patients with clinical improvement may transition to phase II. Patients undergo bone marrow biopsy and aspiration and blood sample collection throughout the study.
Biological: Axatilimab · Procedure: Biospecimen Collection · Procedure: Bone Marrow Aspiration and Biopsy
Patients receive axatilimab IV over 30 minutes on days 1 and 15 and azacitidine IV over 10-40 minutes or SC on days 1-7 of each cycle. Cycles repeat every 28 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity. Patients who achieve PR or better may continue for up to 24 total cycles. Patients who achieve less than PR receive 2 additional cycles and, if PR or better is achieved, may complete up to 24 total cycles. Patients undergo bone marrow biopsy and aspiration and blood sample collection throughout the study.
Biological: Axatilimab · Drug: Azacitidine · Procedure: Biospecimen Collection · Procedure: Bone Marrow Aspiration and Biopsy · Other: Survey Administration
Given IV
Also known as: Anti-M-CSFR Monoclonal Antibody SNDX-6352, SNDX 6352, SNDX-6352, SNDX6352, UCB6352
Given IV or SC
Also known as: 5 AZC, 5-AC, 5-Azacitidine, 5-Azacytidine, 5-AZC, Azacytidine, Azacytidine, 5-, Ladakamycin, Mylosar, U-18496, Vidaza
Undergo blood sample collection
Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection
Undergo bone marrow biopsy and aspiration
Ancillary study
Incidence of dose limiting toxicities
Includes hematologic and non-hematologic toxicities
Time frame: Up to 42 days after the first dose of study medication
Overall response rate
The number of participants whose best response (according to the 2006 International Working Group response criteria) over the efficacy analysis period is a complete response or partial response will be tallied to estimate the overall response rate and provide a 95% exact confidence interval, according to the Clopper-Pearson method.
Time frame: Up to 5 years
Incidence of adverse events
Adverse events will be tabulated by toxicity grade and relationship with study medication and assessed by Common Terminology Criteria for Adverse Events version 5.0.
Time frame: Up to 30 days after end of treatment
Quality of life measured by Modified Myeloproliferative Neoplasm Symptom Assessment Form (MPNSAF)
Will be assessed in patients receiving axatilimab + azacitidine (AZA) and will be determined using a Modified Myeloproliferative Neoplasm Symptom Assessment Form (MPNSAF) score. The MPNSAF score has a range from 0 to 100 with 100 representing the highest level of symptom severity. Descriptive statistics, means and standard deviations or medians and ranges will be applied to summarize the MPNSAF score.
Time frame: Up to 5 years
Number of platelet transfusions
Will be assessed in patients receiving axatilimab + azacitidine and will be determined by descriptive statistics.
Time frame: Up to 5 years
Number of packed red blood cell transfusions
Will be assessed in patients receiving axatilimab + azacitidine and will be determined by descriptive statistics.
Time frame: Up to 5 years
Time in hospital
Will be assessed in patients receiving axatilimab + azacitidine and will be determined by descriptive statistics.
Time frame: Up to 5 years
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