A Phase 2 interventional study of Allogeneic Hematopoietic Stem Cell Transplantation and Axatilimab in Hematopoietic and Lymphatic System Neoplasm, sponsored by Mayo Clinic. Withdrawn at 3 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-03.
Sponsored by Mayo Clinic · Phase 2, Interventional, and Prevention
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.
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Patients with a history of a hematologic malignancy with a planned myeloablative conditioning (MAC) peripheral blood allogeneic HCT
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
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:
Any of the following current or prior therapies:
Patients with active hepatitis B or C
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
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
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
Undergo SOC allogeneic HCT
Also known as: Allogeneic, Allogeneic Hematopoietic Cell Transplantation, Allogeneic Stem Cell Transplantation, HSC, HSCT, Stem Cell Transplantation, Allogeneic
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
Undergo blood sample collection
Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection
Undergo bone marrow aspiration
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
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
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
Given IV or PO
Also known as: CellCept, MMF
Given IV
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
Undergo skin biopsy
Also known as: Biopsy of Skin
Given tacrolimus
Also known as: FK 506, FK-506, FK506, Fujimycin, Hecoria, Prograf, Protopic, Tacforius
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
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).
Time frame: At 1 year post-transplant
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.
Time frame: Up to 1 year post-transplant
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.
Time frame: Up to 1 year post-transplant
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.
Time frame: At 100 days and 180 days
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.
Time frame: Up to 1 year post-transplant
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.
Time frame: At 30 days, 60 days, 100 days, 180 days, and 365 days
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.
Time frame: Up to 2 years
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.
Time frame: At 30 days, 100 days, 180 days, and 365 days
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.
Time frame: At 30 days, 100 days, 180 days, and 365 days
Progression-free survival
Defined as the time from transplant to the earliest date of documentation of relapse/progression or death due to any cause.
Time frame: Up to 2 years post-transplant
Overall survival
Defined as the time from transplant to death due to any cause.
Time frame: Up to 2 years post-transplant
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.
Time frame: Up to 2 years post-transplant
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