Effects of Axatilimab on Skin in Chronic Graft-versus-Host Disease
Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorNorthwestern University
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age 18 years or older.
Diagnosis of chronic graft-versus-host disease (cGVHD) with skin involvement.
Planned initiation of axatilimab as part of routine clinical care (axatilimab arm) or not planning initiation of axatilimab as part of routine clinical care (control arm).
Able to undergo a skin biopsy at the time of enrollment.
Disqualifiers
Unable to provide informed consent.
Contraindication to skin biopsy, including severe thrombocytopenia or other conditions that make skin biopsy unsafe in the judgment of the investigator.
Active uncontrolled infection.
Trial population
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.
Trial design
Design model
Case-control
Time perspective
Prospective
Treatments tested in this trial
Axatilimab
DrugAxatilimab 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.
Treatment groups
15 Participants
are divided into 2 treatment groupsGroup A: Axatilimab arm1 intervention
Group B: Control arm0 interventions
Trial outcomes
Primary outcomes
1
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 12Secondary outcomes
1
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 122
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 123
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 124
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 12Sponsors and contacts
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Source ClinicalTrials.gov
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