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ACTIVE NOT RECRUITING
NCT02629120
PHASE1/PHASE2

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)

View on ClinicalTrials.gov

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

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.

Locations (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, United States