Phase 1 Study of Emapalumab with Post-Transplant Cyclophosphamide, Tacrolimus, and Mycophenolate Mofetil to Prevent Graft-Versus-Host Disease After Reduced-Intensity Donor Hematopoietic Cell Transplant for AML or MDS

What this Trial Is About

Researchers are evaluating the safety, side effects, and effectiveness of adding emapalumab to a combination of post-transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil to prevent graft-versus-host disease GVHD in patients with acute myeloid leukemia AML or myelodysplastic syndrome MDS undergoing reduced-intensity donor hematopoietic cell transplantation HCT. GVHD is a condition where donor cells attack the patients tissues after transplant, which remains a significant complication despite established treatments. This phase I study aims to assess how well this new drug combination may reduce immune response and inflammation after transplant. Participants receive chemotherapy with fludarabine and melphalan or busulfan and fludarabine before the allogeneic HCT to kill cancer cells and prepare the bone marrow. The transplant infusion is given on day 0. Emapalumab is administered intravenously on days -8, -7, -1, 14, and 21. Cyclophosphamide is given intravenously on days 3 and 4. Tacrolimus and mycophenolate mofetil are given either intravenously or by mouth from days 5 through 95 and days 5 through 35, respectively. Baseline imaging including chest CT and heart function tests ECHO or MUGA are done, along with ongoing blood sample collection throughout the study. During the study, participants are monitored for safety, side effects, and incidence of GVHD up to one year after transplant. Researchers collect blood samples to measure drug levels, immune cell recovery, inflammatory markers, and GVHD biomarkers. They also assess quality of life and gut microbiome diversity at several time points. Follow-up visits occur at 100 days, 6 months, and 1 year post-transplant to evaluate overall survival, disease progression, infection rates, and other health outcomes over time.

Emapalumab With Post-Transplant Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil for the Prevention of Graft-versus-Host Disease After Donor Reduced-Intensity Hematopoietic Cell Transplant