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.
OUTLINE: 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. After completion of study treatment, patients are followed up at 30 days then for up to 2 years.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
50
Given IV
Undergo blood sample collection
Ancillary studies
Undergo optional skin biopsy
Undergo optional skin flexibility assessment
Moffitt Cancer Center
Tampa, Florida, United States
NOT_YET_RECRUITINGDana-Farber Cancer Institute
Boston, Massachusetts, United States
NOT_YET_RECRUITINGFred Hutch/University of Washington Cancer Consortium
Seattle, Washington, United States
RECRUITINGOverall response rate (ORR) in sclerotic manifestations
Will be defined as the proportion of patients with objective response per 2014 National Institutes of Health (NIH) skin and joint criteria.
Time frame: Up to 24 weeks, cycle 7 day 1 (cycle length = 28 days)
Failure-free survival
Will be defined as survival free from addition of another systemic chronic graft-versus-host disease treatment, relapse or death. Kaplan Meier curves will be generated.
Time frame: Up to 2 years
Modified Lee Symptom Scale summary score
The modified Lee symptom scale will be scored according to the recommendation of the developer. A change in the summary score between baseline and follow up of ≥ 6 points will be considered clinically meaningful.
Time frame: Up to 2 years
ORR in sclerotic manifestations
Will be defined as the proportion of patients with objective response per 2014 NIH skin and joint criteria.
Time frame: Up to and at cycle 13 day 1, 48 weeks (cycle length = 28 days)
Change in patient 0-10 sclerotic scale
Will be defined as the proportion who change by 2 or more points. A change of 2 points on this scale is considered clinically meaningful.
Time frame: Up to and at cycle 7 day 1 (24 weeks) and cycle 13 day 1 (48 weeks) (cycle length = 28 days)
Change in clinician 0-10 sclerotic scale
Will be defined as the proportion who change by 2 or more points. A change of 2 points on this scale is considered clinically meaningful.
Time frame: Up to and at cycle 7 day 1 (24 weeks and cycle 13 day 1 (48 weeks) (cycle length = 28 days)
ORR in all manifestations
Will be defined as the proportion of patients with objective response per 2014 NIH criteria.
Time frame: Up to and at cycle 7 day 1 (24 weeks and cycle 13 day 1 (48 weeks) (cycle length = 28 days)
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