Effects of Axatilimab on Skin in Chronic Graft-versus-Host Disease | NCT07728708

NCT07728708

Overview

NCT ID: NCT07728708
Lead Sponsor: Northwestern UniversityOTHER
Official Title: Mapping On-Target Effects of Axatilimab in Human Chronic Graft-versus-Host Disease Skin: A Prospective Observational Mechanistic Study
Brief Summary: 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.

Status and Timeline

Status: Not yet recruiting
Last Update Posted: Jul 27, 2026Actual
Start Date: Sep 2026Estimated
Primary Completion Date: Aug 2027Estimated
Completion Date

Participation Criteria

Ages Eligible for Study: Adult (18-64)Older Adult (65+)
Sexes Eligible for Study: All
Accepts Healthy Volunteers?: No
Eligibility Criteria: Inclusion Criteria:

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.

Able to provide informed consent.

Exclusion Criteria:

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.

Study Plan

Study Type: Observational
Phase: No data available
Enrollment: 15Estimated
Primary Purpose: No data available
Allocation: No data available
Interventional Model: No data available
Masking: No data available

Contacts and Locations

Central Contacts: Mary Carns, MSContact
m-carns@northwestern.edu+1-312-503-0406

Similar Studies

Participant Group/Arms

Axatilimab arm

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

Control arm

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.