Phase II Study of Axatilimab Intravenous Treatment for Skin Sclerosis in Adults with Chronic Graft-Versus-Host Disease After Stem Cell Transplant

What this Trial Is About

Researchers are evaluating axatilimab in a phase II trial for patients with thickening or hardening of the skin sclerosis caused by chronic graft-versus-host disease cGVHD after a donor stem cell transplant. cGVHD is a serious complication after transplant that can cause disabilities despite not increasing the risk of death. Current treatments can have significant side effects and may not work well. Axatilimab is a monoclonal antibody that targets specific receptors involved in inflammation and fibrosis, potentially improving skin sclerosis related to cGVHD. Participants receive axatilimab intravenously over 30 minutes on days 1 and 15 during the first six 28-day treatment cycles, then on day 1 of subsequent cycles. Treatment can continue for up to 24 cycles unless the disease worsens or side effects become unacceptable. Blood samples are collected throughout the study. Optional skin biopsies and skin flexibility assessments may also be performed. After treatment ends, participants are followed for 30 days and then monitored for up to 2 years. During the study, participants undergo regular evaluations including blood tests, symptom questionnaires, and optional skin exams. Researchers measure overall response to axatilimab in skin sclerosis at 24 weeks and other timepoints up to 48 weeks. They also track survival without treatment failure and symptom changes for up to 2 years. Safety and long-term effects are monitored through follow-up visits after treatment completion, with total participation lasting up to about 5 years from start to study end.

Axatilimab for Sclerotic Chronic Graft-versus-Host Disease