Stem Cell Transplant Following Low-Intensity Chemotherapy to Treat Chronic Granulomatous Disease
Chronic Granulomatous Disease
About this trial
This is an interventional treatment trial for Chronic Granulomatous Disease focused on measuring Engraftment, Minitransplant, Fludarabine, Neutrophil, Allogenic, Donor Apheresis, Anti-Thymocyte Globin, Cyclophosphamide, Nonmyeloablative Bone Marrow Transplantation, Graft-versus-host Disease, Chronic Granulomatous Disease
Eligibility Criteria
INCLUSION CRITERIA: PATIENT CRITERIA: Ages 1 to 55 years DHR proven Chronic Granulomatous Disease: Includes gp91phox, p47phox, p22phox and p67phox deficiency. Free of active infection. Patient has experienced 2 or more prior infections requiring treatment with intravenous anti-bacterial or anti-fungal therapy. HIV negative. No major organ dysfunction precluding transplantation. HLA identical sibling or parent compatible at all 6 of the HLA A, B, and DR antigens by serotyping or DNA typing techniques. Left ventricular ejection fraction: greater than 40% predicted. ECOG performance status of 0 or 1. DONOR CRITERIA: HLA identical sibling donor or parent donor. Fit to receive G-CSF and give peripheral blood stem cells (normal blood count, normotensive, no history of stroke, no history of severe heart disease). Female x-linked CGD carriers must have greater than 30% normal neutrophils. EXCLUSION CRITERIA: Patient or donor pregnant. Age greater than 55 years. ECOG performance status of 2 or more. Evidence of rapid deterioration due to progressive infection and/or organ damage. Left ventricular ejection fraction: greater than or equal to 35 percent predicted. Creatinine Clearance greater than or equal to 50. Serum bilirubin greater than or equal to 4 mg/dl, transaminases greater than or equal to 3x upper limit or normal. HIV negative. Donors who are positive for HBV, HCV or HTLV will be used at the discretion of the investigator. Malignant diseases liable to relapse or progress within 5 years. Donors unfit to receive G-CSF and undergo apheresis. (Uncontrolled hypertension, history of stroke, history of heart disease, thrombocytopenia.)
Sites / Locations
- National Institute of Allergy and Infectious Diseases (NIAID)