Chronic Graft Versus Host DiseasePhase 2NCT07011810
Axatilimab for Sclerotic Chronic Graft-versus-Host Disease
A Phase 2 study testing Axatilimab, Biospecimen Collection and Questionnaire Administration in people with Chronic Graft Versus Host Disease, run by Fred Hutchinson Cancer Center at 3 sites in United States.
Trial summary
This phase II trial tests how well axatilimab works in treating patients with thickening or hardening (sclerosis) of the skin related to chronic graft-versus-host disease after a donor stem cell transplant. Chronic graft-versus-host disease (cGVHD) remains a major complication of donor stem cell transplants. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Graft Versus Host Disease
- What is being tested?
- Axatilimab, Biospecimen Collection and Questionnaire Administration
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 3 sitesUnited States
- Study dates
- 6 Aug 2025to 10 Feb 2030
- 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
- Adults aged 18 and older
- Ability to understand and willingness to sign a written informed consent document
- Allogeneic stem cell transplant, with active cGVHD requiring systemic treatment. Active cGVHD is defined as the presence of signs and symptoms of cGVHD diagnosed per the 2014 National Institutes of Health (NIH) Consensus Development Project on Criteria for Clinical trials in cGVHD
- Sclerotic skin score 2-3 or PROM < 24 or Joints/Fascia score 2-3 due to cGVHD
- Initial diagnosis of sclerosis within the past 12 months.
- For example, if a patient is diagnosed with sclerosis on 01/01/2026, they remain eligible through 01/01/2027
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- No new non-corticosteroid systemic immunosuppressive agent within 28 days prior to screening, unless there is a plan to stop them no later than 21 days after the first dose of axatilimab. Receipt of systemic corticosteroids ≤ 1 mg/kg prednisone or prednisone equivalent daily is allowed at the time of enrollment and may be continued after axatilimab initiation
- If patient has been previously treated with systemic immunosuppression for sclerosis, one of the following two conditions must be true: (a) the systemic immunosuppressive treatment(s) were given for at least 60 days and the sclerotic cGVHD either did not respond, progressed, or initially improved but has not continued to improve in the last 6 weeks; (b) the systemic immunosuppressive treatment(s) were given for less than 60 days due to lack of sclerotic cGVHD response, sclerotic cGVHD progression, toxicity or logistic reasons and have or will be stopped no later than 21 days after the first dose of axatilimab
- Karnofsky performance status ≥ 60%
- Absolute neutrophil count ≥ 1.0 x 10^9/L (evaluated during the 28-day screening period)
- Platelet count ≥ 50 x 10^9/L (evaluated during the 28-day screening period) (without transfusion within 2 weeks of study entry)
- If no suspected or proven liver cGVHD, alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 x upper limit of normal (ULN) (evaluated during the 28-day screening period) unless due to Gilbert's disease
- If no suspected or proven liver cGVHD, total bilirubin ≤ 1.5 x ULN (evaluated during the 28-day screening period) unless due to Gilbert's disease
- For patients with suspected or documented liver cGVHD, ALT and AST ≤ 5 x ULN (evaluated during the 28-day screening period) unless due to Gilbert's disease
- For patients with suspected or documented liver cGVHD, total bilirubin ≤ 1.5 x ULN (evaluated during the 28-day screening period) unless due to Gilbert's disease
- Estimated creatinine clearance ≥ 30 mL/min based on the institutional formula
- Male and female participants of reproductive potential must be willing to employ highly effective and acceptable forms of contraception from screening through 90 days after the last dose of study treatment.
- Adolescent and adult male patients capable of fathering a child who are non-sterilized and who are not abstinent and intend to be sexually active with a female partner of childbearing potential must use two methods of birth control from the time of screening throughout the total duration of the study intervention treatment period and 90 days after the last dose of study intervention. Male patients should refrain from sperm donation throughout this period
- Female patients of childbearing potential who are not abstinent and intend to be sexually active with a non-sterilized male partner must use at least one highly effective method of contraception from the time of screening throughout the total duration of the study intervention treatment period and 90 days after the last dose of study intervention. Periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of birth control. Female patients should also refrain from breastfeeding throughout this period
- Female subjects of childbearing potential must have a negative serum pregnancy test within 72 hours prior to treatment initiation. Females of childbearing potential are defined as sexually mature females without prior hysterectomy or who have had any evidence of menses in the past 12 months. However, females who have been amenorrheic for 12 or more months are still considered to be of childbearing potential if the amenorrhea is possibly due to prior chemotherapy, anti-estrogens, or ovarian suppression
Who cannot join
- Hospitalization for evaluation or management of an infection within 28 days prior to screening
- History or other evidence of significant organ dysfunction that would make the patient, in the opinion of the investigator, unsuitable for the study
- On more than 1 mg/kg/day prednisone or prednisone equivalent
- History of non-compliance
- History or other evidence of uncontrolled psychiatric illness that that would limit compliance with study requirements
- Receipt of an investigational agent within 28 days prior to screening
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- Any evidence (histologic, cytogenetic, molecular, hematologic, or mixed) of relapse of the underlying cancer or post-transplant lymphoproliferative disease at the time of screening
- Diagnosed with another malignancy (other than malignancy for which transplant was performed) within 3 years of study enrollment, unless previously treated with curative intent (e.g. complete resected basal or squamous cell carcinoma of the skin)
- Active hepatitis B (defined as hepatitis B virus [HBV] surface antigen positive and HBV core antibody positive, with positive HBV deoxyribonucleic acid [DNA], or HBV positive core antibody alone with positive HBV DNA) or hepatitis C (defined as positive hepatitis C [HCV] antibody with positive HCV ribonucleic acid [RNA])
- Suspected active or untreated/inadequately treated latent tuberculosis (as confirmed by a positive QuantiFERON® test or other tuberculosis blood test if untreated)
- History of clinically significant acute pancreatitis within 3 years prior to enrollment or evidence of chronic pancreatitis
- History of myositis
- Pregnant or breastfeeding
- Previous exposure to colony stimulating factor 1 receptor (CSF-1R) therapies
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
Corey Cutler, MD, MPH
- Recruiting
Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, 98109
cGVHD Intake Coordinator, 206-667-4160, cgvhd@fredhutch.org
Stephanie J. Lee, MD, MPH
- Not yet recruiting
Moffitt Cancer Center
Tampa, Florida, 33612
Joseph Pidala, MD, PhD
About this study
Official title: Axatilimab for Sclerotic Chronic Graft-Versus-Host Disease
This phase II trial tests how well axatilimab works in treating patients with thickening or hardening (sclerosis) of the skin related to chronic graft-versus-host disease after a donor stem cell transplant. Chronic graft-versus-host disease (cGVHD) remains a major complication of donor stem cell transplants. Sclerosis, while not associated with a higher risk of death, can lead to serious disabilities. Usual treatments for cGVHD can be associated with significant side effects and unsatisfactory outcomes. A monoclonal antibody, like axatilimab, 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). Axatilimab blocks a receptor and depletes cells that may be involved in the development of inflammation and fibrosis in cGVHD. Giving axatilimab may improve or prevent worsening of sclerosis related to cGVHD in patients after a donor stem cell transplant.
Read the detailed description
OUTLINE:
Patients receive axatilimab IV over 30 minutes on days 1 and 15 of cycles 1-6 and then on day 1 of remaining cycles. Cycles repeat every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo blood sample collection throughout the study. Additionally, patients may undergo optional skin biopsies and optional skin flexibility assessments throughout the study.
After completion of study treatment, patients are followed up at 30 days then for up to 2 years.
- Conditions
- Chronic Graft Versus Host Disease
- Being tested
- Axatilimab, Biospecimen Collection, Questionnaire Administration, Skin Biopsy, Skin Measurement
- Medical subject headings
- Bronchiolitis Obliterans Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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
Questionnaire AdministrationOther
Ancillary studies
Skin BiopsyProcedure or surgery
Also called Biopsy of Skin
Undergo optional skin biopsy
Skin MeasurementProcedure or surgery
Also called Myoton PRO
Undergo optional skin flexibility assessment
| Study group | Role | What they receive |
|---|---|---|
| Treatment (axatilimab) | Experimental | Patients receive axatilimab IV over 30 minutes on days 1 and 15 of cycles 1-6 and then on day 1 of remaining cycles. Cycles repeat every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo blood sample collection throughout the study. Additionally, patients may undergo optional skin biopsies and optional skin flexibility assessments throughout the study. Receives: Biological: Axatilimab; Procedure: Biospecimen Collection; Other: Questionnaire Administration; Procedure: Skin Biopsy; Procedure: Skin Measurement |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate (ORR) in sclerotic manifestations
Will be defined as the proportion of patients with objective response per 2014 National Institutes of Health (NIH) skin and joint criteria.
Measured: Up to 24 weeks, cycle 7 day 1 (cycle length = 28 days)
Secondary outcomes (6)
Failure-free survival
Will be defined as survival free from addition of another systemic chronic graft-versus-host disease treatment, relapse or death. Kaplan Meier curves will be generated.
Measured: Up to 2 years
Modified Lee Symptom Scale summary score
The modified Lee symptom scale will be scored according to the recommendation of the developer. A change in the summary score between baseline and follow up of ≥ 6 points will be considered clinically meaningful.
Measured: Up to 2 years
ORR in sclerotic manifestations
Will be defined as the proportion of patients with objective response per 2014 NIH skin and joint criteria.
Measured: Up to and at cycle 13 day 1, 48 weeks (cycle length = 28 days)
Change in patient 0-10 sclerotic scale
Will be defined as the proportion who change by 2 or more points. A change of 2 points on this scale is considered clinically meaningful.
Measured: Up to and at cycle 7 day 1 (24 weeks) and cycle 13 day 1 (48 weeks) (cycle length = 28 days)
Change in clinician 0-10 sclerotic scale
Will be defined as the proportion who change by 2 or more points. A change of 2 points on this scale is considered clinically meaningful.
Measured: Up to and at cycle 7 day 1 (24 weeks and cycle 13 day 1 (48 weeks) (cycle length = 28 days)
ORR in all manifestations
Will be defined as the proportion of patients with objective response per 2014 NIH criteria.
Measured: Up to and at cycle 7 day 1 (24 weeks and cycle 13 day 1 (48 weeks) (cycle length = 28 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2025
- Study start6 Aug 2025
- Primary completion10 Aug 2028 (estimated)
- Study completion10 Feb 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Graft Versus Host DiseaseStudies of the same condition, open and under way.
- More trials run by Fred Hutchinson Cancer CenterThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fred Hutchinson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Incyte Corporation
- Responsible party
- Sponsor
- Investigators
- Stephanie J. Lee, MD, MPH, Principal investigator, Fred Hutch/University of Washington Cancer Consortium
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07011810
- Sponsor's protocol ID
- RG1125196
- Other IDs
- NCI-2025-01612, FHIRB0020804
- Status last verified
- Aug 2026
- First submitted
- 2 Jun 2025
- Last updated
- 24 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 24 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Axatilimab for Sclerotic Chronic Graft-Versus-Host Disease
- ClinicalTrials.gov ID
- NCT07011810
- Acronym / sponsor ID
- RG1125196
- Conditions
- Chronic Graft Versus Host Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Fred Hutchinson Cancer CenterUniversity, hospital or other
- Collaborators
- Incyte Corporation
- Study start
- 6 Aug 2025 (actual)
- Primary completion
- 10 Aug 2028 (estimated)
- Study completion
- 10 Feb 2030 (estimated)
- First posted
- 10 Jun 2025 (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 phase II trial tests how well axatilimab works in treating patients with thickening or hardening (sclerosis) of the skin related to chronic graft-versus-host disease after a donor stem cell transplant. Chronic graft-versus-host disease (cGVHD) remains a major complication of donor stem cell transplants. Sclerosis, while not associated with a higher risk of death, can lead to serious disabilities. Usual treatments for cGVHD can be associated with significant side effects and unsatisfactory outcomes. A monoclonal antibody, like axatilimab, 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). Axatilimab blocks a receptor and depletes cells that may be involved in the development of inflammation and fibrosis in cGVHD. Giving axatilimab may improve or prevent worsening of sclerosis related to cGVHD in patients after a donor stem cell transplant.
Detailed description
OUTLINE:
Patients receive axatilimab IV over 30 minutes on days 1 and 15 of cycles 1-6 and then on day 1 of remaining cycles. Cycles repeat every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo blood sample collection throughout the study. Additionally, patients may undergo optional skin biopsies and optional skin flexibility assessments throughout the study.
After completion of study treatment, patients are followed up at 30 days then for up to 2 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (axatilimab) | Experimental | Biological: Axatilimab, Procedure: Biospecimen Collection, Other: Questionnaire Administration, Procedure: Skin Biopsy, Procedure: Skin MeasurementPatients receive axatilimab IV over 30 minutes on days 1 and 15 of cycles 1-6 and then on day 1 of remaining cycles. Cycles repeat every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo blood sample collection throughout the study. Additionally, patients may undergo optional skin biopsies and optional skin flexibility assessments throughout the study. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- Adults aged 18 and older
- Ability to understand and willingness to sign a written informed consent document
- Allogeneic stem cell transplant, with active cGVHD requiring systemic treatment. Active cGVHD is defined as the presence of signs and symptoms of cGVHD diagnosed per the 2014 National Institutes of Health (NIH) Consensus Development Project on Criteria for Clinical trials in cGVHD
- Sclerotic skin score 2-3 or PROM < 24 or Joints/Fascia score 2-3 due to cGVHD
- Initial diagnosis of sclerosis within the past 12 months.
Key exclusion criteria (14 listed)
- Hospitalization for evaluation or management of an infection within 28 days prior to screening
- History or other evidence of significant organ dysfunction that would make the patient, in the opinion of the investigator, unsuitable for the study
- On more than 1 mg/kg/day prednisone or prednisone equivalent
- History of non-compliance
- History or other evidence of uncontrolled psychiatric illness that that would limit compliance with study requirements
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 |
|---|---|---|
| AxatilimabAlso: Anti-M-CSFR Monoclonal Antibody SNDX-6352, Axatilimab-csfr, INCA 034176, INCA 34176Given IV | Biological | Treatment (axatilimab) |
| Biospecimen CollectionAlso: Biological Sample Collection, Biospecimen Collected, Specimen CollectionUndergo blood sample collection | Procedure or surgery | Treatment (axatilimab) |
| Questionnaire AdministrationAncillary studies | Other | Treatment (axatilimab) |
| Skin BiopsyAlso: Biopsy of SkinUndergo optional skin biopsy | Procedure or surgery | Treatment (axatilimab) |
| Skin MeasurementAlso: Myoton PROUndergo optional skin flexibility assessment | Procedure or surgery | Treatment (axatilimab) |
Primary outcome
Overall response rate (ORR) in sclerotic manifestations
Time frame: Up to 24 weeks, cycle 7 day 1 (cycle length = 28 days)
Will be defined as the proportion of patients with objective response per 2014 National Institutes of Health (NIH) skin and joint criteria.
6 secondary outcomes
Failure-free survival
Time frame: Up to 2 years
Modified Lee Symptom Scale summary score
Time frame: Up to 2 years
ORR in sclerotic manifestations
Time frame: Up to and at cycle 13 day 1, 48 weeks (cycle length = 28 days)
Change in patient 0-10 sclerotic scale
Time frame: Up to and at cycle 7 day 1 (24 weeks) and cycle 13 day 1 (48 weeks) (cycle length = 28 days)
Change in clinician 0-10 sclerotic scale
Time frame: Up to and at cycle 7 day 1 (24 weeks and cycle 13 day 1 (48 weeks) (cycle length = 28 days)
ORR in all manifestations
Time frame: Up to and at cycle 7 day 1 (24 weeks and cycle 13 day 1 (48 weeks) (cycle length = 28 days)
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 24 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.
Stephanie J. Lee, MD, MPH principal investigator
Fred Hutch/University of Washington Cancer Consortium
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Axatilimab, Biospecimen Collection and Questionnaire Administration in people with Chronic Graft Versus Host Disease, run by Fred Hutchinson Cancer Center 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 20 inclusion and 14 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: looking for participants now, as of the sponsor's last update on 24 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. 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 cGVHD Intake Coordinator (206-667-4160, cgvhd@fredhutch.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response rate (ORR) in sclerotic manifestations (time frame: Up to 24 weeks, cycle 7 day 1 (cycle length = 28 days)). 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 24 Sep 2026; AzTrials last refreshed its copy on 7 Nov 2026.