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
| NCT ID | NCT06523556 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Uma Borate |
| Condition | Atypical Chronic Myeloid Leukemia |
| Study Type | INTERVENTIONAL |
| Enrollment | 52 participants |
| Start Date | 2024-08-02 |
| Primary Completion | 2027-10-31 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 52 participants in total. It began in 2024-08-02 with a primary completion date of 2027-10-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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.
Eligibility Criteria
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) * 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: * Previous treatment for MPN or MDS/MPN overlap with chemotherapy or other antineoplastic agents including lenalidomide and hypomethylating agent (HMAs) such as decitabine or azacitidine or INQOVI (oral decitabine) (patients who had up to 2 cycles of hypomethylating agents \[HMAs\] can be included). However, previous treatment with hydroxyurea and/or ruxolitinib is permitted * 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
Contact & Investigator
The Ohio State University Comprehensive Cancer Center
✉ OSUCCCClinicaltrials@osumc.edu📞 800-293-5066
Uma M Borate, MD
PRINCIPAL INVESTIGATOR
Ohio State University Comprehensive Cancer Center
Frequently Asked Questions
Who can join the NCT06523556 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Atypical Chronic Myeloid Leukemia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06523556 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06523556 currently recruiting?
Yes, NCT06523556 is actively recruiting participants. Contact the research team at OSUCCCClinicaltrials@osumc.edu for enrollment information.
Where is the NCT06523556 trial being conducted?
This trial is being conducted at Columbus, United States.
Who is sponsoring the NCT06523556 clinical trial?
NCT06523556 is sponsored by Uma Borate. The principal investigator is Uma M Borate, MD at Ohio State University Comprehensive Cancer Center. The trial plans to enroll 52 participants.
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