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
NCT Number: NCT06523556
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE1|PHASE2
Conditions: Atypical Chronic Myeloid Leukemia|Chronic Myelomonocytic Leukemia|Myelodysplastic/Myeloproliferative Neoplasm|Recurrent Myelodysplastic/Myeloproliferative Neoplasm|Recurrent Myeloproliferative Neoplasm|Refractory Chronic Myelomonocytic Leukemia|Refractory Myelodysplastic/Myeloproliferative Neoplasm|Refractory Myeloproliferative Neoplasm
Interventions: BIOLOGICAL: Axatilimab|DRUG: Azacitidine|PROCEDURE: Biospecimen Collection|PROCEDURE: Bone Marrow Aspiration and Biopsy|OTHER: Survey Administration
Sponsor: Uma Borate
Collaborators: Incyte Corporation
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 52
Study Design: Allocation: NON_RANDOMIZED|Intervention Model: CROSSOVER|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-09-30
Completion Date: 2026-12-31
Locations: Ohio State University Comprehensive Cancer Center, Columbus, Ohio, 43210, United States

Summary:
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 Outcome Measures: Incidence of dose limiting toxicities, Includes hematologic and non-hematologic toxicities, 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., Up to 5 years
Secondary Outcome Measures: 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., 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., Up to 5 years|Number of platelet transfusions, Will be assessed in patients receiving axatilimab + azacitidine and will be determined by descriptive statistics., 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., Up to 5 years|Time in hospital, Will be assessed in patients receiving axatilimab + azacitidine and will be determined by descriptive statistics., Up to 5 years