An observational study in Chronic Graft vs. Host Disease, GVHD and cGVHD, sponsored by Northwestern University. Not yet recruiting at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-22.
Sponsored by Northwestern University · Observational
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Among 454 studies in Graft vs Host Disease with a site in United States — 50 open to participants now, 71 under way, 2 of them phase 3. Graft vs Host Disease · United States.
Among the 454 studies in Graft vs Host Disease with a site in United States, this one is the 36th largest by planned enrollment of the 43 observational ones that give a count, is one of 13 that started in 2026, is one of 43 observational ones, reaches primary completion later than 31 of the 66 other live ones, and was the 450th registered.
804 studies on the registry are indexed under Graft vs Host Disease; 149 are open to participants now.
Browse Graft vs Host Disease studies →50 of the 454 studies in Graft vs Host Disease with a site in United States are open now. Recruiting first, the newest first:
City of Hope Medical Center · Sites in United States
Ages 7+
Memorial Sloan Kettering Cancer Center · Sites in United States
Ages 18+
Regenerative Ocular Immunobiologics LLC · Sites in United States
Ages 18+
Northwell Health · Sites in United States
Ages 18+
Sawa Ito, MD · Sites in United States
Ages 18+
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 103 Graft vs Host Disease studies recruiting now, 51 (50%) set no upper age limit and 42 (41%) stop at 75 or younger; 42 take children under 18; 9 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
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454 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 145 · Phase 2 227 · Phase 3 48 · Phase 4 5
First in 1986; this study’s year is marked. 13 started in 2026.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 15 is below the median of 72 across 98 observational studies indexed under Graft vs Host Disease.
Median enrollment 30 across the 389 interventional studies, 81 across the 43 observational. Of the 347 that completed or were terminated, 166 (48%) have results posted.
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80 of 454 have an industry lead sponsor. The 10 most frequent:
Northwestern University is the lead sponsor of 1,424 studies on the registry; 199 are open to participants now.
Of its 105 completed or terminated interventional studies of FDA-regulated products, 78 (74%) have results posted.
Northwestern University has 4 studies filed under Graft vs Host Disease — fewer than ten, so there is no comparison to draw.
21 of the studies in Graft vs Host Disease with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT07776210Phase 3Regenerative Ocular Immunobiologics LLCStudy of d-MAPPS™ Ophthalmic Solution in Adults With Chronic Ocular Graft-Versus-Host Disease (oGVHD) | 2027-03-31 |
| NCT06649201Case Comprehensive Cancer CenterVulvovaginal Graft-versus-Host Disease: Diagnosis and Microbiome Evaluation | 2026-11 |
| NCT05675930Memorial Sloan Kettering Cancer CenterA Study of Photobiomodulation (PBM) Therapy in People With Oral Graft-Versus-Host Disease (GVHD) After Stem Cell Transplant | 2026-12-22 |
| NCT04044365National Cancer Institute (NCI)Pediatric cGVHD Symptom Scale | 2026-12-31 |
| NCT03533816University of Kansas Medical CenterExpanded/Activated Gamma Delta T-cell Infusion Following Hematopoietic Stem Cell Transplantation and Post-transplant Cyclophosphamide | 2027-01 |
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Among the 454 studies in Graft vs Host Disease with a site in United States, from their registry updates:
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Chronic graft-versus-host disease (cGVHD) is a condition that can develop after a stem cell transplant. It occurs when donor immune cells attack the body's tissues. In many people, cGVHD affects the skin, causing inflammation, thickening, and scarring that can limit movement and reduce quality of life.
Axatilimab is a medicine approved by the U.S. Food and Drug Administration (FDA) to treat adults with cGVHD after at least two previous treatments have not worked well enough. Although axatilimab can improve cGVHD, researchers do not fully understand how it changes immune cells in the skin.
The purpose of this study is to learn how axatilimab affects immune cells in the skin and blood of people with cGVHD. Researchers will study skin and blood samples collected before and after participants start axatilimab. The goal is to better understand how the medicine reduces inflammation and scarring and to identify biological changes that occur during treatment.
This is an observational research study. The study does not decide whether participants receive axatilimab, when treatment starts, or what dose they receive. All treatment decisions are made by the participant's treating physician as part of routine medical care.
About 15 adults with cGVHD involving the skin will participate. Ten participants who are starting axatilimab as part of their usual care will have three study visits: before treatment begins, about 2 weeks after starting treatment, and about 12 weeks after starting treatment. At each visit, participants will provide a small skin biopsy and a blood sample. Five participants with cGVHD who are not receiving axatilimab will serve as a comparison group and will complete one study visit with one skin biopsy and one blood sample. Researchers will also review participants' medical records and collect information about their skin disease over time.
Information from this study may help researchers better understand how axatilimab works in people with cGVHD. The findings may also help improve future research and guide the development of treatments for cGVHD and other diseases that cause tissue scarring.
Chronic graft-versus-host disease (cGVHD) is a common long-term complication of allogeneic stem cell transplantation. It develops when donor immune cells attack the recipient's healthy tissues. The skin is one of the organs most commonly affected. Skin cGVHD can cause inflammation, thickening, hardening, and scarring (fibrosis), which may lead to pain, reduced flexibility, impaired mobility, and decreased quality of life.
Axatilimab is a monoclonal antibody that blocks colony-stimulating factor-1 receptor (CSF1R), a protein found on monocytes and macrophages, which are immune cells involved in inflammation and tissue repair. Axatilimab is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with recurrent or refractory cGVHD after at least two prior lines of systemic therapy. Although clinical trials have shown that axatilimab improves symptoms in many patients, the biological mechanisms by which it reduces inflammation and fibrosis in human tissues are not fully understood.
The purpose of this prospective observational study is to better understand how axatilimab changes immune cells in the skin and blood of people with cGVHD. The study focuses on macrophages because growing evidence suggests that these cells play an important role in promoting tissue fibrosis. Researchers will investigate how macrophages change after treatment with axatilimab and how these changes affect interactions with other cell types, including fibroblasts, which produce connective tissue, and T cells, which regulate immune responses. Understanding these cellular changes may help explain how axatilimab works and identify biological markers associated with treatment response.
This is an observational, non-interventional study. The study does not assign treatment, change a participant's medical care, or determine whether axatilimab is prescribed. Participants who receive axatilimab will do so because their treating physician has independently decided that treatment is appropriate as part of routine clinical care. Researchers will observe biological changes associated with treatment without influencing clinical decisions.
Approximately 15 adults with skin involvement from cGVHD will participate. Ten participants who are planning to start axatilimab as part of their routine care will undergo study evaluations before treatment begins, approximately 2 weeks after starting treatment, and approximately 12 weeks after starting treatment. Five participants with skin cGVHD who are not starting axatilimab will serve as an observational comparison group and will complete one study visit.
Study procedures include collection of small skin punch biopsies and blood samples, review of medical records, and assessment of skin disease. The skin and blood samples will be analyzed using laboratory methods that can examine individual cells and determine where different cell types are located within the skin. These analyses will allow researchers to evaluate changes in immune cell populations, gene activity, and tissue organization before and after treatment. Researchers will also compare these biological changes with clinical measures of skin disease to determine whether early changes in tissue are associated with later clinical improvement.
The primary goal of this study is to characterize the early biological effects of axatilimab on macrophages in the skin. Secondary objectives include evaluating changes in fibroblasts, immune cell organization, and blood biomarkers, and determining whether these biological changes are associated with improvements in skin disease. The study will also compare tissue findings from cGVHD with existing data from systemic sclerosis, another disease characterized by skin fibrosis, to determine whether similar biological pathways are involved.
This study is designed to improve scientific understanding of how axatilimab works in human tissues rather than to evaluate its clinical effectiveness or safety. The knowledge gained may support the development of biomarkers that predict treatment response, improve understanding of cGVHD biology, and guide future research into treatments for cGVHD and other fibrotic diseases.
Adults with chronic graft-versus-host disease (cGVHD) involving the skin who are receiving care at Northwestern Medicine. The study will enroll participants who are initiating axatilimab as part of routine clinical care and an observational comparison cohort of participants with skin cGVHD who are not initiating axatilimab.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
GVHD (graft-versus-host disease)
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants with chronic graft-versus-host disease (cGVHD) involving the skin who are initiating axatilimab as part of routine clinical care. This is an observational study; axatilimab is prescribed by the treating physician independent of study participation and is not administered by the study protocol. Participants will undergo study evaluations before starting treatment and approximately 2 and 12 weeks after treatment initiation. Study procedures include skin biopsies, blood collection, review of medical records, and clinical assessment of skin disease to evaluate biological changes associated with axatilimab treatment.
Drug: Axatilimab
Participants with mild or controlled chronic graft-versus-host disease (cGVHD) involving the skin who are not initiating axatilimab and are receiving standard clinical management without CSF1R-targeted therapy. This observational comparison group will complete one study visit that includes a skin biopsy, blood collection, review of medical records, and clinical assessment of skin disease. Data and biospecimens from this group will provide a reference for comparison with participants receiving axatilimab to help distinguish biological changes associated with treatment from those associated with chronic cGVHD itself.
Axatilimab is an FDA-approved monoclonal antibody that blocks colony-stimulating factor-1 receptor (CSF1R). In this observational study, axatilimab is prescribed and administered as part of routine clinical care by the treating physician. The study does not assign or administer treatment; it evaluates biological changes associated with axatilimab therapy using skin biopsies, blood samples, and clinical assessments.
Change in CSF1R+ profibrotic macrophages in skin
Within-participant change in the abundance and activation state of CSF1R-positive profibrotic macrophages in skin biopsy specimens after initiation of axatilimab.
Time frame: Baseline, Week 2, and Week 12
Change in fibroblast activation in skin
Within-participant change in the abundance and activation state of fibroblast subsets in skin biopsy specimens after initiation of axatilimab.
Time frame: Baseline, Week 2, and Week 12
Change in immune cell organization in skin
Within-participant change in the spatial organization and interactions of macrophages, fibroblasts, and T cells within skin biopsy specimens after initiation of axatilimab.
Time frame: Baseline, Week 2, and Week 12
Change in circulating biomarkers
Within-participant change in blood biomarkers associated with CSF1R pathway activity and immune responses, including CSF1, IL-34, CXCL9, and CXCL10.
Time frame: Baseline, Week 2, and Week 12
Change in NIH Skin Score
Change in National Institutes of Health (NIH) Skin Score (a 4-point body surface area (BSA) scale and a 3-point sclerosis features scale) from baseline following initiation of axatilimab.
Time frame: Baseline and Week 12
Change in Modified Lee Symptom Scale
Change in Modified Lee Symptom Scale score from baseline following initiation of axatilimab. The modified Lee Symptom Scale (mLSS) ranges from 0 to 100, with higher scores indicating greater cGVHD symptom burden.
Time frame: Baseline and Week 12
Change in corticosteroid dose
Change in systemic corticosteroid dose after initiation of axatilimab.
Time frame: Baseline and Week 12
Plan to share: No — Individual participant data (IPD) collected for this study will not be made publicly available because of the small sample size and the detailed clinical and molecular data generated, which could increase the risk of participant re-identification despite removal of direct identifiers. Summary results, including aggregate analyses and study findings, will be reported through scientific publications, presentations, and ClinicalTrials.gov, as appropriate.
Start is estimated for Nov 2026; primary completion for Aug 2027, 9 months later.
Of 45 non-industry observational studies under Graft vs Host Disease, half reached primary completion within 60 months of starting (the median), counting those still running; a quarter within 38 and three quarters within 154. This study's planned 9 months is below that.
Counting every sponsor, half of 46 studies reached it within 60 months; a quarter within 37 and three quarters within 154.
How these figures are worked out, and which studies are left out of them.
This study is not yet recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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