Phase II Study of Allogeneic Stem Cell Transplant to Treat Patients Aged 4 to 69 with Inborn Errors of Immunity

What this Trial Is About

Researchers are evaluating allogeneic hematopoietic stem cell transplantation HSCT as a potential treatment for people aged 4 to 69 years with inborn errors of immunity IEI, also known as primary immunodeficiency diseases PID or Primary Immune Regulatory Disorders PIRD. These conditions cause significant health problems due to immune system defects, often leading to recurrent infections or immune dysregulation. The study aims to assess whether HSCT can restore normal immune function and reverse disease symptoms by establishing sustained donor blood cell engraftment. The study offers different transplant conditioning regimens based on patient needs, including low, intermediate, and high intensity options. These regimens involve drugs like fludarabine and busulfan, dosed according to pharmacokinetic monitoring, and may include alemtuzumab for immune dysregulation or total body irradiation for certain donor matches. After conditioning, participants receive stem cells from matched or haploidentical donors through transplantation. Post-transplant, patients receive medications such as cyclophosphamide, mycophenolate mofetil, and tacrolimus to prevent graft-versus-host disease. Treatment schedules and drug doses vary by transplant intensity and donor matching. Participants undergo thorough screening including medical history, physical exams, blood and urine tests, heart tests, and imaging scans before transplantation. They are hospitalized for at least four weeks during the transplant and receive ongoing monitoring through blood, urine, bone marrow, and stool sample collections. Follow-up visits occur at days 30, 60, 100, 180, 360, 24 months post-transplant, and annually for about five years to assess immune recovery, disease reversal, transplant complications, and survival outcomes. The study closely tracks safety and effectiveness measures related to donor cell engraftment and immune function restoration.

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity