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

NCT07728708 | Not Yet Recruiting FDA Drug

• 2 other identifiers: STU00226060I-34176-26-13
• Study Type: observational
• Target: 15
• Locations: 1 country
• Sites: 1

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.

Conditions

Chronic Graft vs. Host Disease; GVHD; cGVHD; Graft Versus Host Disease in Skin

Keywords

cGVHD; GVHD; axatiliamab

Outcome Measures

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.

  Baseline, Week 2, and Week 12

Secondary Outcomes (6)

• Change in fibroblast activation in skin

  Baseline, Week 2, and Week 12

• Change in immune cell organization in skin

  Baseline, Week 2, and Week 12

• Change in circulating biomarkers

  Baseline, Week 2, and Week 12

• Change in NIH Skin Score

  Baseline and Week 12

• Change in Modified Lee Symptom Scale

  Baseline and Week 12

Study Arms (2)

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.

Interventions

Axatilimab DRUG

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.

Axatilimab arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study 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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Northwestern University: lead

Study Sites (1)

Northwestern Medicine

Chicago, Illinois, 60611, United States

Location

Related Publications (1)

• Wolff D, Cutler C, Lee SJ, Pusic I, Bittencourt H, White J, Hamadani M, Arai S, Salhotra A, Perez-Simon JA, Alousi A, Choe H, Kwon M, Bermudez A, Kim I, Socie G, Chhabra S, Radojcic V, O'Toole T, Tian C, Ordentlich P, DeFilipp Z, Kitko CL; AGAVE-201 Investigators. Axatilimab in Recurrent or Refractory Chronic Graft-versus-Host Disease. N Engl J Med. 2024 Sep 19;391(11):1002-1014. doi: 10.1056/NEJMoa2401537.

  PMID: 39292927 BACKGROUND

Biospecimen

Retention: SAMPLES WITH DNA

Cryopreserved peripheral blood mononuclear cells (PBMC), frozen serum samples, and formalin-fixed paraffin-embedded (FFPE) skin biopsies.

MeSH Terms

Interventions

axatilimab

Study Officials

• Alexander Misharin, MD, PhD

  Northwestern University

  PRINCIPAL INVESTIGATOR

• Kehinde Adekola, MD

  Northwestern University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Mary Carns, MS

CONTACT

+1-312-503-0406; m-carns@northwestern.edu

Phillip Cooper

CONTACT

+1-312-503-0406; p-cooper@northwestern.edu

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: July 22, 2026
• First Posted: July 27, 2026
• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: July 27, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)