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Axatilimab with or without Azacitidine for Myeloproliferative Neoplasms

This study is testing axatilimab, with or without azacitidine, for people with advanced myeloproliferative neoplasms (MPN), MPN/myelodysplastic syndrome (MDS) overlap, or high-risk chronic myelomonocytic leukemia (CMML). Axatilimab is an antibody that may slow cancer growth by blocking a protein on white blood cells. Azacitidine is a medication that works by stopping or slowing the growth of cancer cells. The study aims to find the best dose of axatilimab and see how well the treatments work. Researchers will also look at side effects and quality of life. You may be able to join if you are 18 or older and have a confirmed diagnosis of one of these conditions.

Study design
This is a Phase Ib/II interventional study planning to enroll 52 participants. It is designed to determine the recommended dose and overall response rate of the treatments.
What's involved
You would undergo blood sample collection, bone marrow biopsies, and complete surveys. The study will measure dose-limiting toxicities for up to 42 days after the first dose.
Compensation
Not stated in the trial record.
Follow-up
The overall response rate will be measured for up to 5 years after treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06523556

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

Recruiting
PHASE1Ages 18+InterventionalTreatment
Uma Borate
~49 participants
Updated 2026-08-27 on ClinicalTrials.gov
What's tested:AxatilimabAzacitidineBiospecimen CollectionBone Marrow Aspiration and BiopsySurvey Administration

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of dose limiting toxicities
Measured over Up to 42 days after the first dose of study medication
+1 more outcome measured

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

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Atypical Chronic Myeloid LeukemiaChronic Myelomonocytic LeukemiaEssential ThrombocythemiaMyelodysplastic/Myeloproliferative NeoplasmPolycythemia VeraPrimary Myelofibrosis

Where it's being run

1 sites across 1 states
Ohio1

Study leadership

  • Uma M Borate, MD · PRINCIPAL_INVESTIGATOR · Ohio State University Comprehensive Cancer Center

Who to contact

The Ohio State University Comprehensive Cancer Center
[email protected]
800-293-5066
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Signed informed consent must be obtained prior to participation in the study
Age ≥ 18 years at the date of signing the informed consent form (ICF)
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:
Chronic myelomonocytic leukemia (CMML), classified as intermediate-2, OR high-risk per the CMML Specific Prognostic Scoring System (CPSS) Molecular Model
Atypical chronic myelocytic leukemia (aCML)
MDS/MPN unclassified (MDS/MPN-U)
Myeloproliferative neoplasm accelerated phase (MPN-AP)
MPN-AP requires a previous diagnosis of polycythemia vera (PV), essential thrombocythemia (ET), or primary myelofibrosis (PMF) with intermediate-2 or high risk disease according to International Prostate Symptom Score (IPSS) as well as progression on or failure to respond to at least one line of therapy.
Myelodysplastic syndrome/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T) or MDS/MPN with SF3B1 mutation and thrombocytosis (MDS/MPN-SF3B1-T).
Not suitable for immediate myeloablative/intensive chemotherapy based on investigator assessment of age, comorbidities, local guidelines, institutional practice (any or all of these)
PHASE Ib (RELAPSE \[R\]/ REFRACTORY \[R\]): Relapse/refractory patients who have received at least two cycles of disease directed therapy (prior therapies can include hypomethylating agents \[HMAs\], HMA combination therapies, and other disease directed therapies)
PHASE II (NEWLY DIAGNOSED PHASE): Newly diagnosed patients without prior treatment, including intensive induction chemotherapy. However, previous treatment with hydroxyurea, ruxolitinib, and/or up to 2 cycles of HMAs (decitabine, azacitidine, oral decitabine \[INQOVI\]) is permitted
Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 × upper limit of normal (ULN)
Total bilirubin ≤ 1.5 × ULN (except in the setting of isolated Gilbert syndrome)
Estimated glomerular filtration rate (eGFR) ≥ 30 mL/min/1.73m\^2 (estimation based on Modification of Diet in Renal Disease \[MDRD\] formula, by local laboratory)
Patient is able to communicate with the investigator and has the ability to comply with the requirements of the study procedures
Women of childbearing potential and men, if not surgically sterilized, should use adequate contraception from 14 days prior to study entry and until 90 days after the last follow-up visit. Adequate contraception is defined as using hormonal contraceptives or an intrauterine device combined with at least 1 of the following forms of contraception: a diaphragm or cervical cap, or a condom

Exclusion

Diagnosis of acute myeloid leukemia (AML) including acute promyelocytic leukemia and extra-medullary AML based on WHO 2016 classification (Arber et al 2016)
Patients who are candidates for myeloablative or intensive chemotherapy treatment or who do not provide consent for this treatment
History of organ transplant or allogenic hematopoietic stem cell transplant
Participants with prior malignancy, except:
Participants with history of adequately treated malignancy for which no anticancer systemic therapy (namely chemotherapy, radiotherapy or surgery) is ongoing or required during the course of the study.
Participants who are receiving adjuvant therapy such as hormone therapy are eligible. However, participants who developed therapy related neoplasms are not eligible
Previous known allergy/sensitivity to components of axatilimab
History of acute or chronic pancreatitis
History of myositis

What this trial measures

  • Incidence of dose limiting toxicitiesUp to 42 days after the first dose of study medication

    Includes hematologic and non-hematologic toxicities

  • Overall response rateUp to 5 years

    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.