Stem Cell Transplant Treatment for Common Variable Immunodeficiency and Autoimmune Immune Disorders Using Reduced-Intensity Conditioning

What this Trial Is About

Researchers are evaluating hematopoietic stem cell transplantation HSCT with a reduced-intensity conditioning regimen including total body irradiation for people diagnosed with Common Variable Immunodeficiency CVID and other autoimmune manifestations of Primary Immune Regulatory Disorders PIRD. This Phase II, open-label study focuses on correcting inheritable immune defects in patients aged 5 to 40 years. Genetic screening is required to identify relevant immune deficiencies such as CID, IPEX syndrome, and others. Participants will receive an allogenic stem cell transplant from a related or unrelated donor matched fully or partially by human leukocyte antigen HLA. The conditioning regimen includes alemtuzumabCampath, anti-thymocyte globulin, fludarabine, melphalan, and total body irradiation. Graft sources include bone marrow or mobilized peripheral blood stem cells. After the transplant, subjects are monitored for two years following standard care practices. During the study, participants will undergo regular evaluations including chimerism testing at multiple time points up to 24 months, immune function assessments such as immunoglobulin levels, and monitoring for graft-versus-host disease. The primary outcome is survival two years after transplant. Secondary outcomes include immune cell engraftment, immunoglobulin independence, and incidence of acute and chronic graft-versus-host disease. Safety and function are closely followed throughout the study period.

Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)