Low Intensity Preparative Regimen Followed by HLA-Matched, Mobilized Peripheral Blood Stem Cell Transplantation for Chronic Granulomatous Disease
Chronic Granulomatous Disease (CGD) is an inherited disorder of neutrophil function.
Patients are profoundly immunocompromised, and are plagued early in life with recurrent, and
life threatening infections. Allogeneic stem cell transplantation is the only cure for CGD.
The goal of this phase I/II study is to investigate the safety and efficacy of a novel
approach to allogeneic stem cell transplantation aimed at decreasing transplant related
morbidity and mortality. We will treat patients with CGD with an allogeneic, G-CSF
mobilized peripheral blood stem cell transplant from an HLA identical family member. The
graft will be T-cell depleted which will decrease the incidence of acute graft vs. host
disease. Donor T-cells will be infused post-transplant if engraftment of donor stem cells
is unsatisfactory. The preparative regimen utilized will provide intense immunosuppression
without myeloablation. It is designed to allow donor stem cell engraftment while minimizing
serious transplant related toxicity.
The end points of this study are engraftment, degree of donor-host chimerism, incidence of
acute and chronic GVHD, transplant related morbidity and mortality as well as overall
Endpoint Classification: Safety Study, Primary Purpose: Treatment
United States: Federal Government
|National Institute of Allergy and Infectious Diseases (NIAID)||Bethesda, Maryland 20892|