Recruiting
Phase 2

Axatilimab

Sponsor:

Fred Hutchinson Cancer Center

Code:

NCT07011810

Conditions

Chronic Graft Versus Host Disease

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Axatilimab

Biospecimen Collection

Questionnaire Administration

Skin Biopsy

Skin Measurement

Study Details

Brief 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. 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.

Conditions

Chronic Graft Versus Host Disease

Study ID

NCT07011810

Start date

Aug 6, 2025

Status verified date

Aug, 2026

Completion date

Feb 10, 2030

Anticipated

Primary completion date

Aug 10, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 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
  • 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

Exclusion Criteria:

  • 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
  • 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

Study Design

Enrollment

50 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Treatment (axatilimab)

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.

Interventions

Axatilimab

Given IV

Biospecimen Collection

Undergo blood sample collection

Questionnaire Administration

Ancillary studies

Skin Biopsy

Undergo optional skin biopsy

Skin Measurement

Undergo optional skin flexibility assessment

Primary outcome measure

  • Overall response rate (ORR) in sclerotic manifestations [ Time Frame: Up to 24 weeks, cycle 7 day 1 (cycle length = 28 days) ]

Central Contacts and Locations

Central contacts

cGVHD Intake Coordinator

206-667-4160cgvhd@fredhutch.org

Locations

Fred Hutch/University of Washington Cancer Consortium

Recruiting

Seattle, Washington, United States, 98109

Contacts

cGVHD Intake Coordinator

206-667-4160cgvhd@fredhutch.org

Principal Investigator:

Stephanie J. Lee, MD, MPH

More Information

Sponsor

Fred Hutchinson Cancer Center

Last update posted

Aug 12, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Fred Hutchinson Cancer Center on 2026-08-12.