Phase II Study of Donor Stem Cell Transplant to Treat VEXAS Syndrome Using Reduced Intensity Conditioning and GVHD Prevention

What this Trial Is About

Researchers are studying whether allogeneic hematopoietic stem cell transplantation HSCT can be successfully performed in adults aged 18 to 75 with VEXAS Syndrome, a condition involving severe inflammatory and blood-related problems that are often resistant to standard treatments. This disease causes systemic inflammation and bone marrow failure, leading to serious health issues and increased mortality. The study aims to see if HSCT can reverse the disease symptoms and achieve sustained donor cell engraftment. Participants receive different reduced intensity conditioning regimens depending on their donor match status before undergoing HSCT. Those with an 88 HLA matched donor receive fludarabine and busulfan, while those with a 78 matched or haploidentical donor receive fludarabine, cyclophosphamide, total body irradiation, and busulfan. After transplant, all participants receive drugs to prevent graft-versus-host disease GVHD, including cyclophosphamide, mycophenolate mofetil, and tacrolimus. These treatments are given according to a detailed schedule around the transplant day. Participants undergo extensive screening including physical exams, blood and urine tests, imaging scans, and specialist evaluations. After transplant, they stay in or near the hospital for at least 100 days with weekly visits, followed by study visits at 30, 60, 100, 180, 210, 240, 300, and 360 days post-transplant, then yearly visits for two years and annual phone contacts thereafter. Researchers monitor disease reversal, donor cell engraftment, safety, GVHD incidence, survival, and other outcomes over several years.

A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome