Phase III Study of Allogeneic Stem Cell Transplant Using Alemtuzumab, Busulfan, Total Body Irradiation, and Cytokine Antagonists in Patients with Chronic Granulomatous Disease

What this Trial Is About

Researchers are studying Chronic Granulomatous Disease CGD, an inherited immune disorder that increases the risk of infections and inflammatory complications. Current treatments can cause serious side effects, and while stem cell transplants can cure CGD, they dont always succeed. This trial aims to evaluate whether a new drug regimen can improve the success rates of stem cell transplants in people with CGD, including those with high inflammation levels. Participants will be divided into two groups based on their inflammation marker levels. Both groups receive conditioning drugs including alemtuzumab, busulfan, and total body irradiation, followed by donor peripheral blood stem cell infusions through a catheter placed in a vein. The high-risk group with elevated inflammation also receives additional drugs targeting specific inflammatory cytokines. Post-transplant medications such as sirolimus and cyclophosphamide will be given to prevent complications. During the study, participants undergo thorough screening including physical exams, blood and urine tests, heart and lung function tests, imaging scans, and bone marrow biopsies. They will stay in the hospital for pre-transplant treatments and for six weeks after receiving the stem cells, followed by frequent clinic visits for three months and continued follow-up for five years. The main outcome measured is overall survival one year after transplant, with other assessments including infection rates, engraftment success, and transplant-related complications.

Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists