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New drug combo aims to shrink Hard-to-Treat blood cancers

NCT ID NCT06523556

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 49 people

The number the study aims to enrol. It can still change while the study runs.

Started

Aug 2024

Expected to finish

Oct 2028

An estimate. End dates often move.

Lead sponsor

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

18 years and older

Sex

Anyone

Healthy volunteers

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

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.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    1 site. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. 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

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Ohio State University Comprehensive Cancer Center

    RECRUITING

    Columbus, Ohio, 43210, United States

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