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

NCT07349771 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: MC23081024-009803
• Study Type: interventional
• Target: 72
• Locations: 1 country
• Sites: 3

Brief 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. 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.

Conditions

Hematopoietic and Lymphatic System Neoplasm

Outcome Measures

Primary Outcomes (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).

  At 1 year post-transplant

Secondary Outcomes (11)

• Cumulative incidence of mild, moderate, and severe chronic GVHD

  Up to 1 year post-transplant

• Cumulative incidence of systemic corticosteroid requiring chronic GVHD

  Up to 1 year post-transplant

• Cumulative incidence of grade II-IV and III-IV acute GVHD

  At 100 days and 180 days

• Cumulative incidence of non-relapse mortality

  Up to 1 year post-transplant

• Incidence of primary and secondary graft failure

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

Study Arms (3)

Stage 1 safety run-in (SOC GVHD prophylaxis, axatilimab)

EXPERIMENTAL

Patients 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.

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)

EXPERIMENTAL

Patients 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.

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 COMPARATOR

Patients 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.

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

Interventions

Axatilimab BIOLOGICAL

Given IV

Also known as: Anti-M-CSFR Monoclonal Antibody SNDX-6352, Axatilimab-csfr, INCA 034176, INCA 34176, INCA-034176, INCA-34176, INCA034176, INCA34176, Niktimvo, SNDX 6352, SNDX-6352, SNDX6352, UCB6352

Stage 1 safety run-in (SOC GVHD prophylaxis, axatilimab); Stage 2 arm I (SOC GVHD prophylaxis, axatilimab)

Biospecimen Collection PROCEDURE

Undergo blood sample collection

Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection

Stage 1 safety run-in (SOC GVHD prophylaxis, axatilimab); Stage 2 arm I (SOC GVHD prophylaxis, axatilimab); Stage 2 arm II (SOC GVHD prophylaxis, placebo)

Bone Marrow Aspiration PROCEDURE

Undergo bone marrow aspiration

Stage 1 safety run-in (SOC GVHD prophylaxis, axatilimab); Stage 2 arm I (SOC GVHD prophylaxis, axatilimab); Stage 2 arm II (SOC GVHD prophylaxis, placebo)

Computed Tomography PROCEDURE

Undergo CT

Also known as: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, Computerized axial tomography (procedure), Computerized Tomography, Computerized Tomography (CT) scan, CT, CT Scan, Diagnostic CAT Scan, Diagnostic CAT Scan Service Type, tomography

Stage 1 safety run-in (SOC GVHD prophylaxis, axatilimab); Stage 2 arm I (SOC GVHD prophylaxis, axatilimab); Stage 2 arm II (SOC GVHD prophylaxis, placebo)

Cyclophosphamide DRUG

Given IV

Also known as: (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Asta B 518, B 518, B-518, B518, Carloxan, Ciclofosfamida, Ciclofosfamide, Cicloxal, Clafen, Claphene, CP monohydrate, CTX, CYCLO-cell, Cycloblastin, Cycloblastine, Cyclophospham, Cyclophosphamid monohydrate, Cyclophosphamide Monohydrate, Cyclophosphamidum, Cyclophosphan, Cyclophosphane, Cyclophosphanum, Cyclostin, Cyclostine, Cytophosphan, Cytophosphane, Cytoxan, Fosfaseron, Genoxal, Genuxal, Ledoxina, Mitoxan, Neosar, Revimmune, Syklofosfamid, WR 138719, WR- 138719, WR-138719, WR138719

Stage 1 safety run-in (SOC GVHD prophylaxis, axatilimab); Stage 2 arm I (SOC GVHD prophylaxis, axatilimab); Stage 2 arm II (SOC GVHD prophylaxis, placebo)

Magnetic Resonance Imaging PROCEDURE

Undergo MRI

Also known as: Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, MRIs, NMR Imaging, NMRI, Nuclear Magnetic Resonance Imaging, sMRI, Structural MRI

Stage 1 safety run-in (SOC GVHD prophylaxis, axatilimab); Stage 2 arm I (SOC GVHD prophylaxis, axatilimab); Stage 2 arm II (SOC GVHD prophylaxis, placebo)

Mycophenolate Mofetil DRUG

Given IV or PO

Also known as: CellCept, MMF

Stage 1 safety run-in (SOC GVHD prophylaxis, axatilimab); Stage 2 arm I (SOC GVHD prophylaxis, axatilimab); Stage 2 arm II (SOC GVHD prophylaxis, placebo)

Placebo Administration DRUG

Given IV

Stage 2 arm II (SOC GVHD prophylaxis, placebo)

Positron Emission Tomography PROCEDURE

Undergo PET

Also known as: Medical Imaging, Positron Emission Tomography, PET, PET Scan, Positron emission tomography (procedure), Positron Emission Tomography Scan, Positron-Emission Tomography, PT

Stage 1 safety run-in (SOC GVHD prophylaxis, axatilimab); Stage 2 arm I (SOC GVHD prophylaxis, axatilimab); Stage 2 arm II (SOC GVHD prophylaxis, placebo)

Skin Biopsy PROCEDURE

Undergo skin biopsy

Also known as: Biopsy of Skin

Stage 1 safety run-in (SOC GVHD prophylaxis, axatilimab); Stage 2 arm I (SOC GVHD prophylaxis, axatilimab); Stage 2 arm II (SOC GVHD prophylaxis, placebo)

Tacrolimus DRUG

Given tacrolimus

Also known as: FK 506, FK-506, FK506, Fujimycin, Hecoria, Prograf, Protopic, Tacforius

Stage 1 safety run-in (SOC GVHD prophylaxis, axatilimab); Stage 2 arm I (SOC GVHD prophylaxis, axatilimab); Stage 2 arm II (SOC GVHD prophylaxis, placebo)

X-Ray Imaging PROCEDURE

Undergo x-ray

Also known as: Conventional X-Ray, Diagnostic Radiology, Medical Imaging, X-Ray, Plain film radiographs, Radiographic Imaging, Radiographic imaging procedure (procedure), Radiography, RG, Static X-Ray, X-Ray

Stage 1 safety run-in (SOC GVHD prophylaxis, axatilimab); Stage 2 arm I (SOC GVHD prophylaxis, axatilimab); Stage 2 arm II (SOC GVHD prophylaxis, placebo)

Allogeneic Hematopoietic Stem Cell Transplantation PROCEDURE

Undergo SOC allogeneic HCT

Also known as: Allogeneic, Allogeneic Hematopoietic Cell Transplantation, Allogeneic Stem Cell Transplantation, HSC, HSCT, Stem Cell Transplantation, Allogeneic

Stage 1 safety run-in (SOC GVHD prophylaxis, axatilimab); Stage 2 arm I (SOC GVHD prophylaxis, axatilimab); Stage 2 arm II (SOC GVHD prophylaxis, placebo)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Mayo Clinic: lead

Study Sites (3)

Mayo Clinic in Arizona

Scottsdale, Arizona, 85259, United States

Location

Mayo Clinic in Florida

Jacksonville, Florida, 32224-9980, United States

Location

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Interventions

Stem Cell Transplantation; axatilimab; Specimen Handling; Cyclophosphamide; Magnetic Resonance Spectroscopy; Mycophenolic Acid; Tacrolimus; X-Rays; Phantoms, Imaging

Intervention Hierarchy (Ancestors)

Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Spectrum Analysis; Chemistry Techniques, Analytical; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids; Macrolides; Lactones; Electromagnetic Radiation; Electromagnetic Phenomena; Magnetic Phenomena; Physical Phenomena; Radiation; Radiation, Ionizing; Equipment and Supplies

Study Officials

• Saurabh Chhabra, MBBS, MS

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Clinical Trials Referral Office

CONTACT

855-776-0015; mayocliniccancerstudies@mayo.edu

Cancer Center Clinical Trials

CONTACT

507-293-6386

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: PREVENTION
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Safety run-in followed by randomized phase II study design.

Study Record Dates

• First Submitted: January 15, 2026
• First Posted: January 20, 2026
• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): June 1, 2033
• Last Updated: April 16, 2026

Record last verified: 2026-04

Locations

US (3)