Actively Recruiting
Phase I/II Trial of Axatilimab and Ruxolitinib for Myelofibrosis and Chronic Myelomonocytic Leukemia
Led by M.D. Anderson Cancer Center · Updated on 2026-01-05
66
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
Sponsors
M
M.D. Anderson Cancer Center
Lead Sponsor
I
Incyte, Inc
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying axatilimab, a CSF1R monoclonal antibody, alone and combined with ruxolitinib to find the best dose and effects for patients with myelofibrosis (MF) and chronic myelomonocytic leukemia (CMML). The study aims to assess safety, side effects, and how well these treatments work in controlling these blood cancers. This trial includes a Phase 1 dose escalation to find the maximum tolerated dose and a Phase 2 dose expansion to evaluate overall response rates and symptom improvements. Participants will receive axatilimab alone or with ruxolitinib taken orally. The treatments are self-administered about 12 hours apart, without regard to food. The study monitors different groups including those with prior therapy or newly diagnosed MF patients, as well as patients with CMML who have relapsed or are intolerant to previous treatments. The trial evaluates responses using established criteria for both MF and CMML. During the trial, participants will have regular safety assessments and clinical laboratory tests to monitor adverse events and treatment effects. Researchers will measure outcomes such as symptom burden, blood and bone marrow changes, and survival rates. The study will continue through an average of one year, with ongoing evaluation of safety and effectiveness. Participants must be willing and able to follow study procedures and provide informed consent.
CONDITIONS
Brief Title
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older
- Diagnosis of myelofibrosis (MF) by WHO or ICC criteria
- For Phase 1: at least one prior therapy for MF or suboptimal response after at least 3 months of JAK inhibitor therapy
- For Phase 2: either relapsed MF with prior therapy or newly diagnosed, treatment-naive MF with intermediate-1 risk by DIPSS-plus
- Diagnosis of CMML refractory to hydroxyurea or hypomethylating agent treatment or intolerant of these therapies
- ECOG performance status 0 to 2
- Adequate liver function with bilirubin and AST/ALT levels ≤3 times the upper limit of normal unless disease-related
- Kidney function with creatinine clearance >30 mL/min and no end-stage renal disease
- Prior use of hydroxyurea or hematopoietic growth factors allowed before study start
- Signed informed consent document
- Patients with prior or concurrent malignancies that do not interfere with study assessments
- Women of childbearing potential must agree to use effective contraception during study participation
You will not qualify if you...
- Currently receiving treatment for another malignancy except certain skin, cervical, breast, or prostate cancers
- Receiving other investigational agents or prior CSF1-R inhibitor therapy
- Active uncontrolled infections including bacterial, fungal, parasitic, or viral
- Active or latent tuberculosis without confirmed adequate treatment
- Known malabsorption syndrome or conditions impairing medication absorption
- History of acute or chronic pancreatitis
- Active symptomatic myositis
- Platelet count below 50x10^9/L before treatment start for combination cohorts unless related to disease or therapy
- Pregnant or breastfeeding women
- Unwillingness to follow contraception requirements if of reproductive potential
- Positive pregnancy test at screening for females of reproductive potential
- Use of other investigational agents, chemotherapy, radiotherapy, or immunotherapy within 7 days before therapy start
- History of chronic hepatitis B or C
- History of HIV infection
- New York Heart Association Class III or IV heart failure or left ventricular ejection fraction below 50%
- Recent myocardial infarction, thromboembolic disease, unstable angina, or severe ventricular arrhythmias
- Psychiatric illness or social situations limiting compliance with study requirements
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 - Up to approximately 1 year
Participants receive treatment with axatilimab alone or in combination with ruxolitinib. They will self-administer the study drugs approximately 12 hours apart, taken orally without regard to food.
Regular visits to monitor safety and response during treatment
Trial Site Locations
Total: 1 location
1
The University of Texas M. D. Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
N
Naveen Pemmaraju, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here