Actively Recruiting
Axatilimab for Sclerotic Chronic Graft-Versus-Host Disease
Led by Fred Hutchinson Cancer Center · Updated on 2026-03-27
50
Participants Needed
3
Research Sites
78 weeks
Total Duration
On this page
Sponsors
F
Fred Hutchinson Cancer Center
Lead Sponsor
I
Incyte Corporation
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating how well axatilimab works in treating sclerosis, a thickening or hardening of the skin, caused by chronic graft-versus-host disease (cGVHD) after a donor stem cell transplant. cGVHD is a serious complication of such transplants, with sclerosis leading to significant disabilities. Current treatments can cause major side effects and may not be fully effective. Axatilimab is a monoclonal antibody that blocks certain receptors involved in inflammation and fibrosis, aiming to improve or prevent worsening of skin sclerosis in these patients. Patients receive axatilimab through an intravenous (IV) infusion lasting 30 minutes on days 1 and 15 of the first six treatment cycles, and then only on day 1 of subsequent cycles. Each treatment cycle lasts 28 days, and patients may receive up to 24 cycles unless their disease progresses or they experience unacceptable side effects. Alongside treatment, blood samples are collected regularly. Additionally, participants can opt for skin biopsies and skin flexibility assessments throughout the study. Participants are closely monitored with blood tests and optional skin evaluations during treatment. After finishing the treatment phase, follow-up visits occur 30 days later and then continue for up to two years to assess overall response rate in skin sclerosis and other outcomes such as symptom changes and survival without treatment failure. Researchers track patient-reported symptoms, clinician assessments, and overall treatment effects to understand axatilimab's impact on sclerotic cGVHD.
CONDITIONS
Brief Title
Axatilimab for Sclerotic Chronic Graft-versus-Host Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults aged 18 and older
- Ability to understand and willing to sign informed consent
- Received allogeneic stem cell transplant with active chronic graft-versus-host disease (cGVHD) requiring systemic treatment
- Sclerotic skin score between 2 and 3 or PROM less than 24 due to cGVHD
- Initial diagnosis of sclerosis within the past 24 weeks (168 days)
- No new non-corticosteroid systemic immunosuppressive treatment within 28 days prior to screening, or plan to stop within 21 days after first axatilimab dose
- Systemic corticosteroids at or below 1 mg/kg prednisone daily allowed
- If previously treated with systemic immunosuppression for sclerosis, treatment was given at least 60 days without response or progression, or less than 60 days and stopped or planned to stop within 21 days after first axatilimab dose
- Karnofsky performance status 60% or higher
- Absolute neutrophil count at least 1.0 x 10^9/L during screening
- Platelet count at least 50 x 10^9/L during screening without recent transfusion
- Liver function tests within specified limits depending on liver cGVHD status
- Estimated creatinine clearance of at least 30 mL/min
- Willingness to use highly effective contraception during and for 90 days after treatment
You will not qualify if you...
- Hospitalization for infection evaluation or management within 28 days prior to screening
- Significant organ dysfunction making participation unsuitable
- Use of prednisone or equivalent above 1 mg/kg/day
- History of non-compliance
- Uncontrolled psychiatric illness limiting study compliance
- Receipt of investigational agent within 28 days prior to screening
- Evidence of relapse of underlying cancer or post-transplant lymphoproliferative disease
- Diagnosis of other malignancy within 3 years unless treated with curative intent
- Active hepatitis B or C infection
- Suspected active or latent tuberculosis
- History of acute or chronic pancreatitis
- History of myositis
- Pregnant or breastfeeding
- Previous exposure to colony stimulating factor 1 receptor (CSF-1R) therapies
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to approximately 24 cycles of 28 days each (about 24 months)
Participants 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. Participants also undergo blood sample collection throughout the study. Additionally, participants may undergo optional skin biopsies and optional skin flexibility assessments throughout the study.
2 visits per 28-day cycle for cycles 1-6, then 1 visit per 28-day cycle afterward; blood sample collection throughout; optional additional visits for skin biopsies and skin flexibility assessments
Duration - Up to 2 years
After completion of study treatment, participants are followed up at 30 days and then for up to 2 years to monitor their health and any long-term effects.
Visits at 30 days post-treatment and periodic visits during follow-up up to 2 years
Trial Site Locations
Total: 3 locations
1
Moffitt Cancer Center
Tampa, Florida, United States, 33612
Not Yet Recruiting
2
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Not Yet Recruiting
3
Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, United States, 98109
Actively Recruiting
Research Team
C
cGVHD Intake Coordinator
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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