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New drug aims to stop transplant complications in blood cancer patients

NCT ID NCT07349771

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
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 Results may not be published yet.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 This study
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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Sep 04, 2026 · Updated 2 times

Summary

This phase II trial tests whether adding the drug axatilimab to standard care can prevent graft-versus-host disease (GVHD) in 72 blood cancer patients receiving a stem cell transplant. GVHD occurs when donor cells attack the patient's body, causing serious symptoms. Axatilimab is a monoclonal antibody that may block this immune attack. The study compares GVHD-free survival at one year between those getting axatilimab plus standard therapy and those getting a placebo plus standard therapy.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
axatilimab
What this could lead to
If it works, this could lead to a new way to prevent graft-versus-host disease after stem cell transplants, improving outcomes for blood cancer patients.
What could go wrong
This is an early phase II trial with only 72 participants, so results may not apply broadly. The drug may not work better than current treatments or could cause unexpected side effects.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Expected to start

Jun 2026

An estimate. Start dates often move.

Expected to finish

Jun 2033

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: * Age ≥ 18 years * Patients with a history of a hematologic malignancy with a planned myeloablative conditioning (MAC) peripheral blood allogeneic HCT * 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 human leukocyte antigen (HLA)-matched sibling or unrelated donor according to transplant center's guidelines (for selection of appropriate donor) * Karnofsky performance status ≥ 60 * Total bilirubin ≤ 1.5 x upper limit of normal (ULN) or total bilirubin ≤ 3.0 x the ULN in the presence of Gilbert's syndrome (obtained ≤ 14 days prior to registration/randomization). 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 * Alanine aminotransferase (ALT) and aspartate transaminase (AST) ≤ 2.5 x ULN (obtained ≤ 14 days prior to registration/randomization) * Calculated creatinine clearance ≥ 30 mL/min using the Cockcroft-Gault formula (obtained ≤ 14 days prior to registration/randomization) * 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 * 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 * Provide written informed consent * 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: * Pregnant persons * Nursing persons * 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: * Patients receiving a cord blood transplant * Patients undergoing a T-cell depleted allogeneic transplantation (using ex vivo CD34 selection, or with serotherapy \[antithymocyte globulin or alemtuzumab\]) * Patients undergoing a second allogeneic transplant (after a previous allogeneic transplant) * Pre-planned treatment with JAK1/JAK2 inhibitor after transplant * Previous exposure to axatilimab * Currently participating in any other interventional study * Receiving an investigational treatment ≤ 28 days prior to registration/randomization * Major surgery ≤ 3 weeks prior to registration/randomization * Chemotherapy ≤ 2 weeks prior to registration/randomization unless chemotherapy is part of the pre-transplant conditioning * Radiation therapy ≤ 2 weeks prior to registration/randomization unless radiation is part of the pre-transplant conditioning * Planned use of donor lymphocyte infusion (DLI) therapy * 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 GVHD-free survival (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 * Patients with a history of hepatitis B or C are allowed if hepatitis B virus (HBV) deoxyribonucleic acid (DNA) or hepatitis C virus (HCV) RNA are undetectable * Any known infection with human immunodeficiency virus (HIV), human T-lymphotropic virus-1 (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 principal investigator (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 chronic lymphocytic leukemia \[CLL\], or acute leukemia from myelodysplastic syndrome \[MDS\]), the original hematological disorder is not considered an exclusion. Cancer treated with curative intent \< 3 years previously will not be allowed unless approved by 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.

Contacts and locations

Locations

  • Mayo Clinic in Arizona

    Scottsdale, Arizona, 85259, United States

  • Mayo Clinic in Florida

    Jacksonville, Florida, 32224-9980, United States

  • Mayo Clinic in Rochester

    Rochester, Minnesota, 55905, United States

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