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Hematopoietic and Lymphatic System NeoplasmPhase 2NCT07349771

Axatilimab Plus Standard of Care Therapy for the Prevention of Graft Versus Host Disease Following Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Hematologic Cancer, ABRAXAS Trial

A Phase 2 study testing Allogeneic Hematopoietic Stem Cell Transplantation, Axatilimab and Biospecimen Collection in people with Hematopoietic and Lymphatic System Neoplasm, run by Mayo Clinic at 3 sites in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

This phase II trial studies how well adding axatilimab to standard of care (SOC) therapy works in preventing graft versus host disease (GVHD) following allogeneic hematopoietic stem cell transplantation (HCT) in patients with hematologic cancer. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Allogeneic Hematopoietic Stem Cell Transplantation, Axatilimab and Biospecimen Collection
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
0 participants
Where
3 sitesUnited States
Study dates
1 Jun 2026to 1 Jun 2033
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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)

Who cannot join

  • 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
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  • 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

Trial locations and contacts

3 sites in 1 country.

United States3 sites

  • Mayo Clinic in Florida

    Jacksonville, Florida, 32224-9980

  • Mayo Clinic in Rochester

    Rochester, Minnesota, 55905

  • Mayo Clinic in Arizona

    Scottsdale, Arizona, 85259

Other trials in United States

About this study

Official title: Phase II Double-Blinded Placebo-Controlled Randomized Trial of CSF-1R Inhibitor, Axatilimab for Prevention of Acute and Chronic GVHD After HLA-Matched Related and Unrelated Donor Allogeneic Stem Cell Transplantation (ABRAXAS)

This phase II trial studies how well adding axatilimab to standard of care (SOC) therapy works in preventing graft versus host disease (GVHD) following allogeneic hematopoietic stem cell transplantation (HCT) in patients with hematologic cancer. Allogeneic HCT is a procedure in which a person receives blood-forming stem cells (cells from which all blood cells develop) from a genetically similar, but not identical, donor. This is often a sister or brother, but could be an unrelated donor. Sometimes the transplanted cells from a donor can attack the body's normal cells, causing GVHD. Symptoms of GVHD can include yellowing of the skin, mucous membranes, and eyes, skin rash or blisters, dry mouth, or dry eyes. Typically, drugs such as cyclophosphamide, tacrolimus, and mycophenolate mofetil are given after the transplant to help stop GVHD from happening, but these current therapies may negatively affect patient quality of life and newer treatment strategies are needed. Axatilimab is a monoclonal antibody. A monoclonal antibody is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens), which may prevent GVHD from developing. Adding axatilimab to SOC therapy may be more effective in preventing GVHD following allogeneic HCT in patients with hematologic cancer.

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Allogeneic Hematopoietic Stem Cell TransplantationProcedure or surgery

    Also called Allogeneic, Allogeneic Hematopoietic Cell Transplantation, Allogeneic Stem Cell Transplantation, HSC

    Undergo SOC allogeneic HCT

  • AxatilimabBiological

    Also called Anti-M-CSFR Monoclonal Antibody SNDX-6352, Axatilimab-csfr, INCA 034176, INCA 34176

    Given IV

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection

    Undergo blood sample collection

  • Bone Marrow AspirationProcedure or surgery

    Undergo bone marrow aspiration

  • Computed TomographyProcedure or surgery

    Also called CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography

    Undergo CT

  • CyclophosphamideDrug

    Also called (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Asta B 518, B 518

    Given IV

  • Magnetic Resonance ImagingProcedure or surgery

    Also called Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan

    Undergo MRI

  • Mycophenolate MofetilDrug

    Also called CellCept, MMF

    Given IV or PO

  • Placebo AdministrationDrug

    Given IV

  • Positron Emission TomographyProcedure or surgery

    Also called Medical Imaging, Positron Emission Tomography, PET, PET Scan, Positron emission tomography (procedure)

    Undergo PET

  • Skin BiopsyProcedure or surgery

    Also called Biopsy of Skin

    Undergo skin biopsy

  • TacrolimusDrug

    Also called FK 506, FK-506, FK506, Fujimycin

    Given tacrolimus

  • X-Ray ImagingProcedure or surgery

    Also called Conventional X-Ray, Diagnostic Radiology, Medical Imaging, X-Ray, Plain film radiographs

    Undergo x-ray

Study groups in NCT07349771
Study groupRoleWhat they receive
Stage 1 safety run-in (SOC GVHD prophylaxis, axatilimab)ExperimentalPatients undergo SOC allogeneic HCT on day 0 and receive SOC GVHD prophylaxis consisting of cyclophosphamide IV over 1-2 hours on days +3 and +4, tacrolimus starting on day +5 and tapered through day +90 to +100, and mycophenolate mofetil IV or PO TID on days +5 to +35 in the absence of disease progression or unacceptable toxicity. Starting on day +35, patients also receive axatilimab IV over 30 minutes on day 1 of each cycle. Cycles repeat every 14 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Additionally, patients undergo x-ray or CT during screening and blood sample collection and bone marrow aspiration throughout the study. Patients may also undergo CT, MRI, or PET throughout the study and may optionally undergo skin biopsy on study.
Receives: Procedure: Allogeneic Hematopoietic Stem Cell Transplantation; Biological: Axatilimab; Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Computed Tomography; Drug: Cyclophosphamide; Procedure: Magnetic Resonance Imaging; Drug: Mycophenolate Mofetil; Procedure: Positron Emission Tomography; Procedure: Skin Biopsy; Drug: Tacrolimus; Procedure: X-Ray Imaging
Stage 2 arm I (SOC GVHD prophylaxis, axatilimab)ExperimentalPatients undergo SOC allogeneic HCT and receive SOC GVHD prophylaxis plus axatilimab as in stage 1 safety run-in in the absence of disease progression or unacceptable toxicity. Additionally, patients undergo x-ray or CT during screening and blood sample collection and bone marrow aspiration throughout the study. Patients may also undergo CT, MRI, or PET throughout the study and may optionally undergo skin biopsy on study.
Receives: Procedure: Allogeneic Hematopoietic Stem Cell Transplantation; Biological: Axatilimab; Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Computed Tomography; Drug: Cyclophosphamide; Procedure: Magnetic Resonance Imaging; Drug: Mycophenolate Mofetil; Procedure: Positron Emission Tomography; Procedure: Skin Biopsy; Drug: Tacrolimus; Procedure: X-Ray Imaging
Stage 2 arm II (SOC GVHD prophylaxis, placebo)Placebo comparatorPatients undergo SOC allogeneic HCT on day 0 and receive SOC GVHD prophylaxis consisting of cyclophosphamide IV over 1-2 hours on days +3 and +4, tacrolimus starting on day +5 and tapered through day +90 to +100, and mycophenolate mofetil IV or PO TID on days +5 to +35 in the absence of disease progression or unacceptable toxicity. Starting on day +35, patients also receive placebo IV over 30 minutes on day 1 of each cycle. Cycles repeat every 14 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Additionally, patients undergo x-ray or CT during screening and blood sample collection and bone marrow aspiration throughout the study. Patients may also undergo CT, MRI, or PET throughout the study and may optionally undergo skin biopsy on study.
Receives: Procedure: Allogeneic Hematopoietic Stem Cell Transplantation; Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Computed Tomography; Drug: Cyclophosphamide; Procedure: Magnetic Resonance Imaging; Drug: Mycophenolate Mofetil; Drug: Placebo Administration; Procedure: Positron Emission Tomography; Procedure: Skin Biopsy; Drug: Tacrolimus; Procedure: X-Ray Imaging

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Graft versus host disease (GVHD)-free survival

    Rates will be compared between the two arms. A patient will be considered a success for 1-year GVHD-free survival if they are alive without grade 3 or 4 acute or systemic immunosuppression-requiring chronic GVHD at one year post myeloablative allogeneic hematopoietic cell transplantation (HCT).

    Measured: At 1 year post-transplant

Secondary outcomes (11)

  1. Cumulative incidence of mild, moderate, and severe chronic GVHD

    As assessed by National Institutes of Health criteria. Will be estimated in each arm using the competing risk model, with death without chronic GVHD as a competing risk event. Differences between arms will be evaluated using Cox proportional hazard models with competing risk.

    Measured: Up to 1 year post-transplant

  2. Cumulative incidence of systemic corticosteroid requiring chronic GVHD

    Will be estimated in each arm using the competing risk model, with death without chronic GVHD requiring systemic corticosteroids as a competing risk event. Differences between arms will be evaluated using Cox proportional hazard models with competing risks.

    Measured: Up to 1 year post-transplant

  3. Cumulative incidence of grade II-IV and III-IV acute GVHD

    Will be estimated in each arm using the competing risk model, with death without grade II-IV or III-IV acute GVHD as a competing risk events respectively. Differences between arms will be evaluated using Cox proportional hazard models with competing risks.

    Measured: At 100 days and 180 days

  4. Cumulative incidence of non-relapse mortality

    Will be estimated in each arm using the competing risk model, with relapse/progression as a competing risk event. Differences between arms will be evaluated using Cox proportional hazard models with competing risks.

    Measured: Up to 1 year post-transplant

  5. Incidence of primary and secondary graft failure

    Will be estimated in each arm by the number of patients who experience graft failure divided by the total number of evaluable patients. Exact binomial 95% confidence intervals for the true success rate will be calculated. Differences in rates between arms will be evaluated using Fisher's exact test.

    Measured: At 30 days, 60 days, 100 days, 180 days, and 365 days

  6. Cumulative incidence of relapse/progression of the primary hematologic malignancy

    Will be estimated in each arm using the competing risk model, with death without relapse/progression as a competing risk event. Differences between arms will be evaluated using Cox proportional hazard models with competing risks.

    Measured: Up to 2 years

  7. Lineage specific chimerism kinetics

    The percentage of donor versus recipient cells will be evaluated for T cells (CD3+), myeloid cells (CD33+), B cells (CD19+), and NK cells (CD56+) at each time point to assess lineage specific chimerism kinetics. The percentages will be categorized for each cell type as full recipient (≤ 5%), mixed (6-94%), or full donor (≥ 95%). Values will be summarized descriptively (median, range, distribution across categories) and changes across time will be evaluated.

    Measured: At 30 days, 100 days, 180 days, and 365 days

  8. Immune reconstitution

    Immune profiles (including helper T cells (CD3+ CD4+), cytotoxic T cells (CD3+ CD8+), regulatory T cells (CD3+ CD4+ CD25+ CD127low/-FOXP3+), B cells (CD19+) will be evaluated at each time point to assess immune reconstitution following transplant. Values at each time point will be summarized descriptively (median, range) and changes across time will be evaluated.

    Measured: At 30 days, 100 days, 180 days, and 365 days

  9. Progression-free survival

    Defined as the time from transplant to the earliest date of documentation of relapse/progression or death due to any cause.

    Measured: Up to 2 years post-transplant

  10. Overall survival

    Defined as the time from transplant to death due to any cause.

    Measured: Up to 2 years post-transplant

  11. Incidence of adverse events

    Assessed using NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5. The maximum grade for each type of adverse event will be recorded for each patient, and frequency tables will be reviewed for each arm to determine patterns. Additionally, the relationship of the adverse event(s) to the study treatment will be taken into consideration.

    Measured: Up to 2 years post-transplant

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Jan 2026
  2. Study start1 Jun 2026 (estimated)
  3. Primary completion1 Jun 2028 (estimated)
  4. Study completion1 Jun 2033 (estimated)

Why it stopped: Sponsor declined to fund study due to inability to agree on IP.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Prevention
Allocation
Randomised
Intervention model
Sequential
Model description
Safety run-in followed by randomized phase II study design.
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07349771
Sponsor's protocol ID
MC230810
Other IDs
24-009803
Status last verified
Apr 2026
First submitted
15 Jan 2026
Last updated
3 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Allogeneic Hematopoietic Stem Cell Transplantation, Axatilimab and Biospecimen Collection in people with Hematopoietic and Lymphatic System Neoplasm, run by Mayo Clinic at 3 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 29 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 3 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Graft versus host disease (GVHD)-free survival (time frame: At 1 year post-transplant). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07349771.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Sep 2026.

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