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Phase I/II Clinical Trial of Axatilimab, a CSF1R Monoclonal Antibody, in Combination With Ruxolitinib as Therapy for Patients With Myelofibrosis (MF) and Chronic Myelomonocytic Leukemia (CMML) | Clinical Trials | Clareo Health

RECRUITINGPHASE1/PHASE2

Phase I/II Clinical Trial of Axatilimab, a CSF1R Monoclonal Antibody, in Combination With Ruxolitinib as Therapy for Patients With Myelofibrosis (MF) and Chronic Myelomonocytic Leukemia (CMML)

NCT07128381•M.D. Anderson Cancer Center

View on ClinicalTrials.gov

Summary

* To find the recommended dose of axatilimab given alone and in combination with ruxolitinib in patients with MF and CMML. * To learn if axatilimab given in combination with ruxolitinib can help to control MF and CMML.

Detailed Description

Primary Objectives: * Phase 1 dose escalation: to determine safety, tolerability and Maximum Tolerated Dose (MTD) and efficacy of axatilimab alone and in combination with ruxolitinib for patients with MF and CMML * Phase 2 dose expansion: to determine the overall response rate (ORR) of axatilimab and ruxolitinib in patients with MF and CMML. * Incidence of AEs, MTD and changes in clinical laboratory values. * Measures of efficacy in CMML: overall response rate (ORR) defined as sum of CR + complete cytogenetic remission + partial remission + marrow response + clinical benefit according to the IWG 2015 MDS/MPN response criteria37. * Measures of efficacy in MF: objective response which is defined as CR (complete remission) + PR (partial remission) + CI (clinical improvement) after 6 cycles of treatment. It will be categorized according to the International Working Group (IWG) consensus criteria for myelofibrosis26 Secondary Objectives: CMML: * To determine other efficacy outcomes such as duration of response, leukemia-free survival (LFS), progression-free survival (PFS) and overall survival (OS). * To evaluate symptom burden improvement assessed by Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF TSS). * To evaluate changes in clonal composition and VAF of identified mutations with therapy * To measure changes in cytokine profile and monocyte populations in peripheral blood and bone marrow. MF: * To explore time to response and duration of response * To explore changes in bone marrow fibrosis * To evaluate symptom burden improvement assessed by Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF TSS) (Appendix 1). * To explore changes in dynamic of cytogenetics and molecular mutations: JAK2V617F, CALR, MPL (or other relevant molecular markers) in terms of allele burden or changes in cytogenetic abnormalities * To measure changes in serum and bone marrow cytokine profile pre- and post-therapy

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Age .18 years as MF and CMML are very rare diseases in the pediatric population.
•Diagnosis of MF by WHO or ICC and:
•1. Phase 1 dose escalation (Cohort A and B): at least 1 prior therapy for MF or with suboptimal response after at least 3 months of therapy with a JAK inhibitor.
•2. Phase 2 dose expansion:
•i. Relapsed cohort (Cohort C): Patients with at least 1 prior MF therapy. or ii. JAKi-naive cohort (Cohort D): patients with newly diagnosed and treatment naive MF with intermediate .1 risk by DIPSS-plus (Appendix 2)
•Diagnosis of CMML refractory to treatment with hydroxyurea (for patients with proliferative CMML defined as WBC.13x109/L) or at least 4 cycles of treatment with hypomethylating agent, with relapse/progression after any number of cycles of hypomethylating agent therapy or who are intolerant of treatment with either therapy. Patients may have received prior therapy with other investigational agents or cytotoxic regimens.
•ECOG performance status ≤2
•Adequate hepatic function with total bilirubin \</=3 x ULN, AST or A LT \</= 3xULN unless related to disease involvement.
•Serum creatinine clearance \>30mL/min and no end/stage renal disease (using Cockcroft-Gault).
•Prior hydroxyurea for control of leukocytosis or use of hematopoietic growth factors (eg, G-CSF, GM-CSF, procrit, aranesp, thrombopoietin) is allowed at any time prior to cycle 1 day 1 of therapy.
+10 more criteria

Exclusion Criteria

•Patients who are currently receiving treatment for a malignancy (not including basal cell carcinoma, nonmelanoma skin cancer, cervical carcinoma in situ, early stage breast cancer or localized prostate cancer treated with hormone therapy). Patients with history of other cancers should be free of disease for at least 2 years prior to the Screening Visit or not requiring active treatment at the time of enrollment.
•Patients who are receiving any other investigational agents or with prior CSF1-R inhibitor therapy.
•Active, uncontrolled bacterial, fungal, parasitic, or viral infection. Infections are considered controlled if appropriate therapy has been initiated and, at the time of screening, there is no evidence of infection worsening, such as hemodynamic instability attributable to sepsis, new symptoms, worsening physical signs, or radiographic findings attributable to infection.
•Patients with evidence of active or latent tuberculosis. Prior to enrollment, patients should be evaluated for tuberculosis risk factors, and those at higher risk should be tested for latent infection. Risk factors include, but are not limited to, prior residence in or travel to countries with a high prevalence of tuberculosis, close contact with a person with active tuberculosis, and a history of active or latent tuberculosis where an adequate course of treatment cannot be confirmed.
•Patients who have a known malabsorption syndrome or other condition that may impair absorption of study medication (e.g., gastrectomy).
•History of acute or chronic pancreatitis.
•Active symptomatic myositis.
•Platelet count \<50x109/L prior to enrollment and treatment initiation (for ruxolitinib combination cohorts only) except if related to either treatment for MF or CMML or treatment with cytotoxic therapy for any other reason.
•Pregnant women are excluded from this study because axatilimab and ruxolitinib are agents with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with these agents, breastfeeding should be discontinued if the mother is treated on study. These potential risks may also apply to other agents used in this study.
•Patients with reproductive potential who are unwilling to following contraception requirements (including condom use for males with sexual partners, and for females: prescription oral contraceptives \[birth control pills\], contraceptive injections, intrauterine devices \[IUD\], doublebarrier method \[spermicidal jelly or foam with condoms or diaphragm\], contraceptive patch, or surgical sterilization) throughout the study.
+7 more criteria

Locations (1)

The University of Texas M. D. Anderson Cancer Center

Houston, Texas, United States

Key Dates

Start Date

January 2, 2026

Primary Completion Date

July 31, 2031

Completion Date

July 31, 2033

Last Updated

January 5, 2026

Enrollment

ESTIMATED participants

Conditions

Myelofibrosis (MF)Chronic Myelomonocytic Leukemia (CMML)

Interventions

Axatilimab (SNDX-6352)

DRUG

Ruxolitinib

DRUG

Contacts

Naveen Pemmaraju, MD

CONTACT

(713) 792-4956 npemmaraju@mdanderson.org

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: January 5, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Phase I/II Clinical Trial of Axatilimab, a CSF1R Monoclonal Antibody, in Combination With Ruxolitinib as Therapy for Patients With Myelofibrosis (MF) and Chronic Myelomonocytic Leukemia (CMML)

Inclusion Criteria

Exclusion Criteria

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