HLA-Matched, Peripheral Blood Stem Cell Transplantation Using Low Intensity Conditioning to Treat Patients With Chronic Granulomatous Disease Who Are Actively Infected
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. The prognosis for CGD patients whose infection persists
despite appropriate medical and surgical intervention is extremely poor. Allogeneic stem
cell transplantation can cure CGD however the mortality of this procedure is high for
patients who are actively infected. Ongoing clinical trials at the NIH and elsewhere
suggest that the use of non-myeloablative conditioning for allogeneic stem cell
transplantation is safer and less toxic for patients who are free of infection at the time
of transplant. The goal of this phase II study is to investigate the safety and efficacy of
this novel approach to allogeneic stem cell transplantation for CGD patients who are
actively infected. Following a preparative regimen designed to provide intense
immunosuppression without myeloablation, patients will receive a peripheral blood stem cell
graft from an HLA identical parent or sibling. Donor T-cells will be infused
post-transplant if donor stem engraftment is unsatisfactory.
The end points of this study are engraftment, degree of donor-host chimerism, incidence of
acute and chronic GvHD, infection status, immune reconstitution, and transplant related
morbidity and mortality.
Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment
United States: Federal Government
|National Institute of Allergy and Infectious Diseases (NIAID)||Bethesda, Maryland 20892|