Clinical Research Directory
Browse clinical research sites, groups, and studies.
High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
Summary
Chronic granulomatous disease (CGD) affects white blood cell function. Currently, the only curative treatment is bone marrow transplant to replace the abnormal stem cells with new ones (donor cells) capable of making a normal immune system. Transplant problems include graft versus host disease (GvHD) and graft rejection. With GvHD, donor cells attack the recipient s normal tissue. Researchers want to use preparation drugs and a high cell dose to increase graft success. They want to use 2 immunosuppressive drugs (cyclophosphamide and sirolimus) to lessen the risk of GvHD. ...
Key Details
Gender
All
Age Range
4 Years - 65 Years
Study Type
INTERVENTIONAL
Enrollment
45
Start Date
2015-12-17
Completion Date
2028-12-30
Last Updated
2026-04-01
Healthy Volunteers
No
Conditions
Interventions
Alemtuzumab
Transplant Conditioning Drug: Monoclonal antibody that targets recipient and donor T-cells to prevent graft verses host disease. Not an IND. This is a well studied drug, and is not under an IND.
Busulfan
Transplant Conditioning Drug: Chemotherapy to create space in the patient's bone marrow so that the donor peripheral blood stem cells can repopulate in the patient's bone marrow. This is a well studied drug, and is not under an IND.
Sirolimus
Immunosuppressant to prevent donor peripheral blood stem cell rejection and graft versus host disease. This is a well studied drug, and is not under an IND.
Cyclophosphamide
Post transplant cyclophosphamide given to prevent graft verses host disease. This is a well studied drug, and is not under an IND.
Total Body Irradiation
Transplant Conditioning Total Body Radiation (300cGy in 2 fractionated doses), given only to patient receiving matched unrelated donor cells, to create space in the patient's bone marrow so that the donor peripheral blood stem cells can repopulate in the patient's bone marrow.
Peripheral blood stem cells
Donor Peripheral blood stem cells, either matched unrelated donor or matched related relative to replace the patient's immune cells with functional immune cells. The peripheral blood stem cells are not regulated by the FDA.
Locations (1)
National Institutes of Health Clinical Center
Bethesda, Maryland, United States