New drug combo aims to shrink Hard-to-Treat blood cancers
NCT ID NCT06523556
First seen Jun 26, 2026 · Last updated Aug 28, 2026 · Updated 3 times
Summary
This study tests a new antibody called axatilimab, given with or without the chemotherapy drug azacitidine, for people with advanced myeloproliferative neoplasms (MPN), MPN/MDS overlap, or high-risk chronic myelomonocytic leukemia (CMML). The goal is to find the safest dose and see if the combination can shrink tumors. About 52 adults will take part in this early-phase trial.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- axatilimab and azacitidine
- What this could lead to
- If it works, this could offer a new treatment option for people with advanced blood cancers that are hard to treat.
- What could go wrong
- This is an early-phase trial with only 52 participants, so results may not apply to everyone. Side effects from the drugs could be serious.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 49 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Aug 2024
- Expected to finish
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Oct 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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) * PHASE Ib (RELAPSE \[R\]/ REFRACTORY \[R\]): Relapse/refractory patients who have received at least two cycles of disease directed therapy (prior therapies can include hypomethylating agents \[HMAs\], HMA combination therapies, and other disease directed therapies) * PHASE II (NEWLY DIAGNOSED PHASE): Newly diagnosed patients without prior treatment, including intensive induction chemotherapy. However, previous treatment with hydroxyurea, ruxolitinib, and/or up to 2 cycles of HMAs (decitabine, azacitidine, oral decitabine \[INQOVI\]) is permitted * 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: * 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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The study's own enquiry address
This study publishes an address for enquiries. See it below .
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Show contact details
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Genom att skicka in godkänner du våra Användarvillkor
Study contacts
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Contact
Email: •••••@•••••
Locations
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Ohio State University Comprehensive Cancer Center
RECRUITINGColumbus, Ohio, 43210, United States
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