Phase II Study of AlphaBeta T Cell Receptor Depleted Stem Cell Transplantation for Patients With Inherited Bone Marrow Failure Disorders

What this Trial Is About

Researchers are evaluating a phase II trial of T cell receptor alphabeta depletion TCD peripheral blood stem cell transplantation in patients with inherited bone marrow failure BMF disorders. The goal is to avoid routine immune suppression for graft-versus-host disease GVHD, allowing earlier immune recovery and potentially reducing severe infection risks after transplantation. This trial is sponsored by the Masonic Cancer Center, University of Minnesota. Participants receive one of several treatment plans involving drugs such as total body irradiation TBI, cyclophosphamide, fludarabine, methylprednisolone, rituximab, busulfan, alemtuzumab, melphalan, and a T cell receptor depleted peripheral blood stem cell infusion on day 0. The conditioning regimens vary based on disease features, donor type, and patient tolerance, with some patients receiving TBI and others alternative drug combinations. G-CSF is administered after transplant to support neutrophil recovery. During the study, participants undergo monitoring for immune recovery and complications including acute and chronic GVHD, engraftment of neutrophils and platelets, infections, regimen-related toxicity, and overall survival up to one year after transplant. Evaluations include clinical assessments and laboratory tests at defined time points such as day 42, day 100, and one year post-transplant. The study aims to describe outcomes for approximately 3 to 5 patients treated annually under these protocols.

Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders