Graft -Versus-host-diseasePhase 2ABRAXASNCT07808112
CSF-1R Inhibitor, Axatilimab, for Prevention of Acute and Chronic GVHD After HLA-Matched Related and Unrelated Donor Allogeneic Stem Cell Transplantation
A Phase 2 study testing Hematopoietic Cell Transplantation, Cyclophosphamide and Tacrolimus in people with Graft -Versus-host-disease, run by Medical College of Wisconsin at 1 site in United States.
Trial summary
This is a randomized, multisite, Phase II study designed to compare the 1-year GVHD-free survival of the control arm (no axatilimab) to the experimental arm (axatilimab) after myeloablative allogeneic HCT. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Graft -Versus-host-disease
- What is being tested?
- Hematopoietic Cell Transplantation, Cyclophosphamide and Tacrolimus
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 72 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2026to Nov 2031
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 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:
- 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.
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- Alanine aminotransferase (ALT) and aspartate transaminase (AST) ≤2.5 x ULN).
- 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.
- 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).
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.
- 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 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 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Medical College of Wisconsin Cancer Center Clinical Trials Office contact
United States1 site
Froedtert & the Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Sameem Abedin, MD, 414-805-4600, sabedin@mcw.edu
Sameem Abedin, MD
About this study
Official title: Phase II Open-Label, Randomized-Controlled Trial of CSF-1R Inhibitor, Axatilimab, for Prevention of Acute and Chronic GVHD After HLA-Matched Related and Unrelated Donor Allogeneic Stem Cell Transplantation
This is a randomized, multisite, Phase II study designed to compare the 1-year GVHD-free survival of the control arm (no axatilimab) to the experimental arm (axatilimab) after myeloablative allogeneic HCT.
Read the detailed description
This study will determine if axatilimab in combination with standard of care posttransplant cyclophosphamide + tacrolimus/mycophenolate mofetil is safe, tolerable, and efficacious as GVHD prophylaxis in a population of adult patients with hematologic malignancies receiving a first HLA-matched related or unrelated donor myeloablative allogeneic HCT.
- Conditions
- Graft -Versus-host-disease
- Being tested
- Hematopoietic Cell Transplantation, Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil (MMF), Axatilimab
- Keywords
- hematologic malignancy, myeloablative allogeneic hematopoietic cell transplantation
- Medical subject headings
- Graft vs Host Disease, Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hematopoietic Cell TransplantationProcedure or surgery
Subjects will receive a first HLA-matched related or (7/8 or 8/8) unrelated donor myeloablative allogeneic HCT.
CyclophosphamideDrug
Also called Cytoxan, Neosar, cytophosphane
Cyclophosphamide will be given on Day +3 and Day +4 per institutional practices, at a dose of 50 mg/kg IBW. Cyclophosphamide will be given as an IV infusion over 1-2 hours (depending on volume), or according to institutional standards.
TacrolimusDrug
Tacrolimus will be given per institutional practices, starting on Day +5. Subsequent dosing will be based on blood levels per institutional guidelines with a suggested range of 5-12 ng/mL.
Mycophenolate Mofetil (MMF)Drug
Mycophenolate mofetil (MMF) will be given per institutional practices, at a dose of 15 mg/kg three times daily (TID; based upon actual body weight) with the maximum total daily dose not to exceed 3 grams (1g TID, IV or PO). MMF prophylaxis will start Day +5 and continue until Day +35 post-transplant, at which time it should be discontinued (no taper necessary).
AxatilimabDrug
Also called Niktimvo, axatilimab-csfr
The dose is dependent on the results of the safety run-in. The starting dose of the safety run-in is 0.3 mg/kg (maximum of 35 mg). If Dose Level 1 has an unacceptable level of toxicity (≥2 patients having significant toxicity), then axatilimab may be de-escalated to 0.2 mg/kg (maximum of 23 mg).
| Study group | Role | What they receive |
|---|---|---|
| Safety Run-in | Experimental | An early safety analysis will be performed after the first 6 evaluable patients have been accrued to Dose Level 1 of the study and observed for four cycles of axatilimab (Cycles 1-4). Accrual will be temporarily halted while these patients are evaluated. If 2 or more of the first 6 patients experience a significant toxicity, then the protocol may be amended and the dose of axatilimab may be de-escalated to Dose Level -1. Receives: Procedure: Hematopoietic Cell Transplantation; Drug: Cyclophosphamide; Drug: Tacrolimus; Drug: Mycophenolate Mofetil (MMF); Drug: Axatilimab |
| Axatilimab Treatment | Experimental | Starts 35 (±5) days from stem cell transplant; Dosing will occur every 14 days for a maximum of 12 cycles. Receives: Procedure: Hematopoietic Cell Transplantation; Drug: Cyclophosphamide; Drug: Tacrolimus; Drug: Mycophenolate Mofetil (MMF); Drug: Axatilimab |
| No Axatilimab Treatment | Experimental | Starts 35 (±5) days from stem cell transplant. Receives: Procedure: Hematopoietic Cell Transplantation; Drug: Cyclophosphamide; Drug: Tacrolimus; Drug: Mycophenolate Mofetil (MMF) |
What is being measured
The primary outcome is the main question the study is designed to answer.
GVHD-free survival
A patient will be considered a success for 1-year GVHD-free survival if they are alive without Grade 3 or 4 acute GVHD or systemic immunosuppression requiring chronic GVHD at one year post transplant. Death, Grade 3 or 4 acute GVHD, and immunosuppression-requiring chronic GVHD (of any severity) prior to 1 year will be considered as events.
Measured: 1 Year Post HCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2026
- Study startNov 2026 (estimated)
- Primary completionNov 2031 (estimated)
- Study completionNov 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Graft -Versus-host-diseaseStudies of the same condition, open and under way.
- More trials run by Medical College of WisconsinThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 72 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Medical College of Wisconsin
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mehdi Hamadani, Medical College of Wisconsin
- Investigators
- Mehdi Hamadani, MD, Study chair, Medical College of WisconsinSameem Abedin, MD, Principal investigator, Medical College of Wisconsin
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07808112
- Sponsor's protocol ID
- IIT-HAMADANI-ABRAXAS
- Acronym
- ABRAXAS
- Status last verified
- Sep 2026
- First submitted
- 3 Sep 2026
- Last updated
- 8 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Open-Label, Randomized-Controlled Trial of CSF-1R Inhibitor, Axatilimab, for Prevention of Acute and Chronic GVHD After HLA-Matched Related and Unrelated Donor Allogeneic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT07808112
- Acronym / sponsor ID
- ABRAXAS / IIT-HAMADANI-ABRAXAS
- Conditions
- Graft -Versus-host-disease
- Keywords
- hematologic malignancy, myeloablative allogeneic hematopoietic cell transplantation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 72 participants (estimated)
- Lead sponsor
- Medical College of WisconsinUniversity, hospital or other
- Study start
- Nov 2026 (estimated)
- Primary completion
- Nov 2031 (estimated)
- Study completion
- Nov 2031 (estimated)
- First posted
- 8 Sep 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a randomized, multisite, Phase II study designed to compare the 1-year GVHD-free survival of the control arm (no axatilimab) to the experimental arm (axatilimab) after myeloablative allogeneic HCT.
Detailed description
This study will determine if axatilimab in combination with standard of care posttransplant cyclophosphamide + tacrolimus/mycophenolate mofetil is safe, tolerable, and efficacious as GVHD prophylaxis in a population of adult patients with hematologic malignancies receiving a first HLA-matched related or unrelated donor myeloablative allogeneic HCT.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Safety Run-in | Experimental | Procedure: Hematopoietic Cell Transplantation, Drug: Cyclophosphamide, Drug: Tacrolimus, Drug: Mycophenolate Mofetil (MMF), Drug: AxatilimabAn early safety analysis will be performed after the first 6 evaluable patients have been accrued to Dose Level 1 of the study and observed for four cycles of axatilimab (Cycles 1-4). Accrual will be temporarily halted while these patients are evaluated. If 2 or more of the first 6 patients experience a significant toxicity, then the protocol may be amended and the dose of axatilimab may be de-escalated to Dose Level -1. |
| Axatilimab Treatment | Experimental | Procedure: Hematopoietic Cell Transplantation, Drug: Cyclophosphamide, Drug: Tacrolimus, Drug: Mycophenolate Mofetil (MMF), Drug: AxatilimabStarts 35 (±5) days from stem cell transplant; Dosing will occur every 14 days for a maximum of 12 cycles. |
| No Axatilimab Treatment | Experimental | Procedure: Hematopoietic Cell Transplantation, Drug: Cyclophosphamide, Drug: Tacrolimus, Drug: Mycophenolate Mofetil (MMF)Starts 35 (±5) days from stem cell transplant. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Age ≥18 years.
- Patients with a history of a hematologic malignancy with a planned myeloablative conditioning (MAC) peripheral blood allogeneic HCT.
- 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:
- 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)
Key exclusion criteria (28 listed)
- 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:
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Hematopoietic Cell TransplantationSubjects will receive a first HLA-matched related or (7/8 or 8/8) unrelated donor myeloablative allogeneic HCT. | Procedure or surgery | Axatilimab Treatment, No Axatilimab Treatment, Safety Run-in |
| CyclophosphamideAlso: Cytoxan, Neosar, cytophosphaneCyclophosphamide will be given on Day +3 and Day +4 per institutional practices, at a dose of 50 mg/kg IBW. Cyclophosphamide will be given as an IV infusion over 1-2 hours (depending on volume), or according to institutional standards. | Drug | Axatilimab Treatment, No Axatilimab Treatment, Safety Run-in |
| TacrolimusTacrolimus will be given per institutional practices, starting on Day +5. Subsequent dosing will be based on blood levels per institutional guidelines with a suggested range of 5-12 ng/mL. | Drug | Axatilimab Treatment, No Axatilimab Treatment, Safety Run-in |
| Mycophenolate Mofetil (MMF)Mycophenolate mofetil (MMF) will be given per institutional practices, at a dose of 15 mg/kg three times daily (TID; based upon actual body weight) with the maximum total daily dose not to exceed 3 grams (1g TID, IV or PO). MMF prophylaxis will start Day +5 and continue until Day +35 post-transplant, at which time it should be discontinued (no taper necessary). | Drug | Axatilimab Treatment, No Axatilimab Treatment, Safety Run-in |
| AxatilimabAlso: Niktimvo, axatilimab-csfrThe dose is dependent on the results of the safety run-in. The starting dose of the safety run-in is 0.3 mg/kg (maximum of 35 mg). If Dose Level 1 has an unacceptable level of toxicity (≥2 patients having significant toxicity), then axatilimab may be de-escalated to 0.2 mg/kg (maximum of 23 mg). | Drug | Axatilimab Treatment, Safety Run-in |
Primary outcome
GVHD-free survival
Time frame: 1 Year Post HCT
A patient will be considered a success for 1-year GVHD-free survival if they are alive without Grade 3 or 4 acute GVHD or systemic immunosuppression requiring chronic GVHD at one year post transplant. Death, Grade 3 or 4 acute GVHD, and immunosuppression-requiring chronic GVHD (of any severity) prior to 1 year will be considered as events.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical College of Wisconsin Cancer Center Clinical Trials Office contact
Mehdi Hamadani, MD study chair
Medical College of Wisconsin
Sameem Abedin, MD principal investigator
Medical College of Wisconsin
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Hematopoietic Cell Transplantation, Cyclophosphamide and Tacrolimus in people with Graft -Versus-host-disease, run by Medical College of Wisconsin at 1 site 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 15 inclusion and 28 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: opening soon, as of the sponsor's last update on 8 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 1 site 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. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Medical College of Wisconsin Cancer Center Clinical Trials Office (866-680-0505 ext. 8900, cccto@mcw.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
GVHD-free survival (time frame: 1 Year Post HCT). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 8 Sep 2026.