Can a new drug stop the Body's attack after stem cell transplants?
NCT ID NCT07808112
First seen Sep 08, 2026 · Last updated Sep 09, 2026 · Updated 1 time
Summary
This phase II trial tests whether adding axatilimab to standard care can prevent graft-versus-host disease (GVHD), a serious complication where donor immune cells attack the recipient's body after a stem cell transplant. Adults with blood cancers receiving a myeloablative allogeneic transplant are randomly assigned to receive either standard care alone or standard care plus axatilimab. The main goal is to see if more people survive one year without severe acute or chronic GVHD.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- axatilimab, an experimental drug that blocks a protein called CSF-1R, added to standard post-transplant care
- What this could lead to
- If it works, adding axatilimab after a stem cell transplant could reduce the risk of severe graft-versus-host disease, making transplants safer and more successful.
- What could go wrong
- This is a phase II trial with only 72 participants, so results may not apply to everyone. Axatilimab may cause side effects, and it is not yet proven to improve outcomes.
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 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 72 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Nov 2026
An estimate. Start dates often move.
- Expected to finish
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Nov 2031
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: * Age ≥18 years. * Patients with a history of a hematologic malignancy with a planned myeloablative conditioning (MAC) peripheral blood allogeneic HCT. a. Note: Patients with acute leukemia must be in morphologic complete remission with or without hematologic recovery with ≤5% blasts in the bone marrow. Patients with Chronic Myelomonocytic Leukemia (CMML) must have a white blood cell (WBC) count ≤10,000 cells/µL and ≤5% blasts in the marrow. Patients with ≥5% blasts due to a regenerating marrow must contact the protocol chairs for review. Patients with a diagnosis of myelofibrosis require sponsor approval before enrolling. * Patients must be receiving an allograft from a suitable HLA-matched sibling or (7/8 or 8/8) unrelated donor according to transplant center's guidelines (for selection of appropriate donor). * The following laboratory values obtained ≤14 days prior to registration/randomization: 1. Total bilirubin ≤1.5 x ULN or total bilirubin ≤3.0 x the Upper Limit of Normal (ULN) in the presence of Gilbert's syndrome. If total bilirubin is abnormal (≥1.5 x ULN), assess direct bilirubin. NOTE: Patients with Gilbert Syndrome and elevated baseline unconjugated (indirect) bilirubin up to 3.0 mg/dL are eligible. Gilbert syndrome should be confirmed by the presence of UGT1A1\*28 variant. 2. Alanine aminotransferase (ALT) and aspartate transaminase (AST) ≤2.5 x ULN). 3. Calculated creatinine clearance ≥30 mL/min using the Cockcroft-Gault formula below: Creatinine clearance for males = (140 - age)(weight in kg) /(72)(serum creatinine in mg⁄dL) Creatinine clearance for females = (140 - age)(weight in kg) (0.85) /(72)(serum creatinine in mg⁄dL) * Negative serum pregnancy test done ≤7 days prior to registration/randomization, for persons of childbearing potential only. * Sexually active patients and their partners must use an effective method of contraception associated with a low failure rate prior to study entry and for the duration of study participation and for at least 3 months after the last dose of study drug. a. Note: The following are considered effective contraceptives: oral contraceptive pill; condom plus spermicide; diaphragm plus spermicide; patient or partner surgically sterile; patient or partner more than 12 months postmenopausal; or injectable or implantable agent/device. Male patients should refrain from sperm donation and female patients should refrain from breastfeeding throughout this period. * Ability to understand and provide written informed consent at the time of initial study enrollment. Participants who later lose decisional capacity may remain on study with consent from a legally authorized representative (LAR), as applicable. * Willingness to provide mandatory blood and bone marrow aspirate specimens for correlative research. * Willing to return to enrolling institution for follow-up (during the Active Monitoring Phase of the study). Exclusion Criteria: * Any of the following because this study involves an investigational agent whose genotoxic, mutagenic, and teratogenic effect on the developing fetus and newborn are unknown: 1. Pregnant persons. 2. Nursing persons. 3. Persons of childbearing potential, and persons able to father a child, who are unwilling to employ adequate contraception. * Any of the following current or prior therapies: 1. Patients receiving a cord blood transplant. 2. Patients undergoing a T-cell depleted allogeneic transplantation (using ex vivo CD34 selection, or with serotherapy \[antithymocyte globulin or alemtuzumab\]). 3. Patients undergoing a second allogeneic transplant (after a previous allogeneic transplant). 4. Pre-planned treatment with JAK1/JAK2 inhibitor after transplant. 5. Previous exposure to axatilimab. 6. Receiving an investigational treatment ≤28 days prior to registration/randomization. 7. Major surgery ≤3 weeks prior to registration/randomization. 8. Chemotherapy ≤2 weeks prior to registration/randomization unless chemotherapy is part of the pre-transplant conditioning. 9. Radiation therapy ≤2 weeks prior to registration/randomization unless radiation is part of the pre-transplant conditioning. 10. Planned use of Donor Lymphocyte Infusion (DLI) therapy. 11. Exception: Use of maintenance regimens as routine clinical care will be permitted but must be declared prior to registration/randomization. The trial does not restrict subsequent treatment after meeting the GFS endpoint. * Active central nervous system (CNS) involvement by malignant cells. * Leukemia involvement in the CNS ≤4 weeks of registration for patients with a history of prior CNS leukemia involvement (i.e., leukemic blasts previously detected in the cerebral spinal fluid). * History of acute or chronic pancreatitis. * History of myositis. * Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens. * Patients with active hepatitis B or C. a. Patients with a history of hepatitis B or C are allowed if HBV DNA or HCV RNA are undetectable. * Any known infection with human immunodeficiency virus (HIV), HTLV-1. * Uncontrolled bacterial, viral, or fungal infections (currently taking medication and with progression or no clinical improvement) at the time of registration/randomization. * Psychiatric illness/social situations that would limit compliance with study requirements. * Diagnosed with another malignancy (other than malignancy for which transplant was performed) ≤ 3 years of registration/randomization, unless previously treated with curative intent and approved by PI (e.g., but not limited to completely resected basal cell, squamous cell, or ductal carcinoma in situ, or low-risk prostate cancer after curative resection or on watchful waiting). In patients with transformed disease (e.g. aggressive lymphoma evolving from CLL, or acute leukemia from MDS), the original hematological disorder is not considered an exclusion. Cancer treated with curative intent \<3 years previously will not be allowed unless approved by any of the Study Chairs. * History of myocardial infarction ≤6 months, or New York Heart Association (NYHA) Class III or IV heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia.
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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 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
Locations
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Froedtert & the Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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