Actively Recruiting

Phase 1
Phase 2
Age: 18Years +
All Genders
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

Led by Uma Borate · Updated on 2026-03-13

52

Participants Needed

1

Research Sites

221 weeks

Total Duration

On this page

Sponsors

U

Uma Borate

Lead Sponsor

I

Incyte Corporation

Collaborating Sponsor

AI-Summary

What this Trial Is About

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

Official Title

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

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Signed informed consent before joining the study
  • Age 18 years or older at consent
  • Confirmed diagnosis of relapsed/refractory or newly diagnosed advanced phase MPN, MPN/MDS overlap, or high-risk CMML as defined by WHO 2016
  • Specific conditions include CMML (intermediate-2 or high-risk), atypical chronic myelocytic leukemia, MDS/MPN unclassified, myeloproliferative neoplasm accelerated phase with prior PV, ET, or PMF and intermediate-2/high risk, MDS/MPN with ring sideroblasts and thrombocytosis or with SF3B1 mutation
  • Not suitable for immediate intensive chemotherapy based on age, health, or local guidelines
  • ECOG performance status of 0, 1, or 2
  • Liver enzymes (AST, ALT) no more than 3 times upper normal limit
  • Total bilirubin no more than 1.5 times upper normal limit (except Gilbert syndrome)
  • Kidney function (eGFR) at least 30 mL/min/1.73m²
  • Able to communicate and comply with study requirements
  • Women and men of childbearing potential must use effective contraception from 14 days before study entry until 90 days after last follow-up
Not Eligible

You will not qualify if you...

  • Prior treatment for MPN or MDS/MPN overlap with chemotherapy or antineoplastic agents except up to 2 cycles of hypomethylating agents; prior hydroxyurea or ruxolitinib allowed
  • Diagnosis of acute myeloid leukemia including acute promyelocytic leukemia or extramedullary AML
  • Candidates for intensive chemotherapy or unwilling to undergo it
  • History of organ or stem cell transplant
  • Previous malignancy except adequately treated cancers without ongoing therapy or therapy-related neoplasms
  • Known allergy to axatilimab components
  • History of acute or chronic pancreatitis
  • History of myositis

AI-Screening

AI-Powered Screening

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Trial Site Locations

Total: 1 location

1

Ohio State University Comprehensive Cancer Center

Columbus, Ohio, United States, 43210

Actively Recruiting

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Research Team

T

The Ohio State University Comprehensive Cancer Center

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

CROSSOVER

Primary Purpose

TREATMENT

Number of Arms

2

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