High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

45

Participants

Timeline

Start Date

December 17, 2015

Primary Completion Date

December 30, 2027

Study Completion Date

December 30, 2028

Conditions
Chronic Granulomatous Disease Transplant
Interventions
DRUG

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.

DRUG

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.

DRUG

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.

DRUG

Cyclophosphamide

Post transplant cyclophosphamide given to prevent graft verses host disease. This is a well studied drug, and is not under an IND.

RADIATION

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.

BIOLOGICAL

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.

Trial Locations (1)

20892

National Institutes of Health Clinical Center, Bethesda

All Listed Sponsors
lead

National Institute of Allergy and Infectious Diseases (NIAID)

NIH

NCT02629120 - High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease | Biotech Hunter | Biotech Hunter