Recruiting
Phase 1
Phase 2

Axatilimab & Azacitidine

Sponsor:

Uma Borate

Code:

NCT06523556

Conditions

Atypical Chronic Myeloid Leukemia

Chronic Myelomonocytic Leukemia

Myelodysplastic/Myeloproliferative Neoplasm

Recurrent Myelodysplastic/Myeloproliferative Neoplasm

Recurrent Myeloproliferative Neoplasm

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Axatilimab

Azacitidine

Biospecimen Collection

Bone Marrow Aspiration and Biopsy

Survey Administration

Study Details

Brief 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.

Conditions

Atypical Chronic Myeloid Leukemia

Chronic Myelomonocytic Leukemia

Myelodysplastic/Myeloproliferative Neoplasm

Recurrent Myelodysplastic/Myeloproliferative Neoplasm

Recurrent Myeloproliferative Neoplasm

Study ID

NCT06523556

Start date

Aug 2, 2024

Status verified date

Aug, 2026

Completion date

Oct 31, 2028

Anticipated

Primary completion date

Oct 31, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 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 Criteria:

  • 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

Study Design

Enrollment

49 participants

Anticipated

Allocation

Non randomized

Intervention Model

Crossover

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Phase I (Axatilimab)

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.

experimental: Phase II (Axatilimab and azacitidine)

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.

Interventions

Axatilimab

Given IV

Azacitidine

Given IV or SC

Biospecimen Collection

Undergo blood sample collection

Bone Marrow Aspiration and Biopsy

Undergo bone marrow biopsy and aspiration

Survey Administration

Ancillary study

Primary outcome measure

  • Incidence of dose limiting toxicities [ Time Frame: Up to 42 days after the first dose of study medication ]
  • Overall response rate [ Time Frame: Up to 5 years ]

Central Contacts and Locations

Central contacts

The Ohio State University Comprehensive Cancer Center

800-293-5066OSUCCCClinicaltrials@osumc.edu

Locations

Ohio State University Comprehensive Cancer Center

Recruiting

Columbus, Ohio, United States, 43210

Contacts

Uma M. Borate

614-293-3316Uma.Borate@osumc.edu

Principal Investigator:

Uma M. Borate

More Information

Sponsor

Uma Borate

Last update posted

Aug 27, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Uma Borate on 2026-08-27.