Actively Recruiting
Phase 1b/2 Study of Axatilimab (SNDX-6352) with or without Azacitidine for Advanced Phase Myeloproliferative Neoplasms, MPN/MDS Overlap, or High-Risk Chronic Myelomonocytic Leukemia
Led by Uma Borate · Updated on 2026-03-13
52
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
Sponsors
U
Uma Borate
Lead Sponsor
I
Incyte Corporation
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying advanced phase myeloproliferative neoplasms (MPN), myeloproliferative neoplasm/myelodysplastic syndrome (MPN/MDS) overlap, and high-risk chronic myelomonocytic leukemia (CMML) in this phase Ib/II trial. The trial aims to find the best dose of axatilimab and to evaluate the effectiveness of axatilimab alone or combined with azacitidine in treating these conditions. Axatilimab is an antibody designed to block a protein involved in cancer cell growth, while azacitidine is a medication that slows cancer cell growth. The study evaluates safety, response rates, quality of life, symptom relief, and biological changes during treatment. In phase I, patients receive axatilimab intravenously over 30 minutes on days 1 and 15 of each 28-day cycle. Patients showing clinical improvement may then enter phase II. In phase II, patients receive axatilimab on days 1 and 15 plus azacitidine given intravenously or by injection on days 1 to 7 of each cycle. Treatment continues for up to 4 cycles initially, with possible extension to 24 cycles if patients respond. Throughout both phases, patients undergo bone marrow biopsies, aspirations, and blood sample collections to monitor disease and treatment effects. Participants will be closely monitored with assessments including blood tests, bone marrow samples, and quality of life questionnaires. Researchers track outcomes such as dose limiting toxicities, overall response rates, transfusion needs, hospital time, and side effects. After treatment ends, patients have follow-up visits at 30 days and then every 6 months for up to 24 months. The study aims to understand treatment safety and how well it manages symptoms and disease progression over time.
CONDITIONS
Brief 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
Eligibility Criteria
You may qualify if you...
- Signed informed consent before participation
- Age 18 years or older at consent
- Morphologically confirmed diagnosis of advanced phase MPN, MPN/MDS overlap, or high-risk CMML based on 2016 WHO classification
- Phase 1b: relapsed or refractory disease, Phase 2: newly diagnosed patients
- Specific diagnoses include intermediate-2 or high-risk CMML, atypical chronic myelocytic leukemia, MDS/MPN unclassified, myeloproliferative neoplasm accelerated phase, and MDS/MPN with ring sideroblasts and thrombocytosis or SF3B1 mutation
- Not suitable for immediate myeloablative or intensive chemotherapy
- ECOG performance status of 0, 1, or 2
- Liver enzymes (AST and ALT) no more than 3 times the upper limit of normal
- Total bilirubin no more than 1.5 times the upper limit of normal except Gilbert syndrome
- Estimated kidney function (eGFR) 30 mL/min/1.73m² or higher
- Ability to communicate and comply with study procedures
- Women of childbearing potential and men must use adequate contraception from 14 days before study entry until 90 days after last follow-up visit
You will not qualify if you...
- Previous chemotherapy or antineoplastic treatment for MPN or MDS/MPN overlap except up to 2 cycles of hypomethylating agents
- Diagnosis of acute myeloid leukemia including acute promyelocytic leukemia and extra-medullary AML
- Candidates for myeloablative or intensive chemotherapy or who decline such treatment
- History of organ transplant or allogenic hematopoietic stem cell transplant
- Prior malignancy unless adequately treated and not requiring ongoing anticancer therapy
- Known allergy or sensitivity to axatilimab components
- History of acute or chronic pancreatitis
- History of myositis
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Repeated 28-day cycles until disease progression or unacceptable toxicity
Participants receive axatilimab intravenously over 30 minutes on days 1 and 15 of each 28-day cycle. Treatment continues in the absence of disease progression or unacceptable toxicity. Participants undergo bone marrow biopsy, aspiration, and blood sample collection throughout this phase.
2 visits per 28-day cycle (in-person)
Duration - Up to 24 cycles of 28 days each in the absence of disease progression or unacceptable toxicity
Participants receive axatilimab intravenously on days 1 and 15 and azacitidine intravenously or subcutaneously on days 1-7 of each 28-day cycle. Treatment is given for up to 4 cycles initially. Participants who achieve partial response or better may continue for up to 24 total cycles. Those who achieve less than partial response receive 2 additional cycles and may continue if response improves. Bone marrow biopsy, aspiration, and blood sample collection continue throughout this phase.
Approximately 4 to 24 cycles with multiple visits per cycle (in-person)
Duration - Up to 24 months after last dose
After completion of study treatment, participants are followed up to monitor safety and health status. Follow-up visits include assessments at 30 days after last dose and every 6 months for up to 24 months.
1 visit at 30 days and visits every 6 months for up to 24 months (in-person)
Trial Site Locations
Total: 1 location
1
Ohio State University Comprehensive Cancer Center
Columbus, Ohio, United States, 43210
Actively Recruiting
Research Team
T
The Ohio State University Comprehensive Cancer Center
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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