Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

50

Participants

Timeline

Start Date

July 8, 2022

Primary Completion Date

December 31, 2032

Study Completion Date

December 31, 2032

Conditions
Chronic Granulomatous Disease
Interventions
DRUG

Sirolimus

Post transplant drug - 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 drug - cyclophosphamide give to prevent graft versus host disease. This is a well studied drug and s not under an IND.

DRUG

Alemtuzumab

Transplant Conditioning Drug - Monoclonal antibody that targets recipient and donor T-cells to prevent graft versus 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 IND.

BIOLOGICAL

Pheripheral blood stem cells

Donor peripheral blood stem cells either matched unrelated donors 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.

DRUG

Emapalumab-Izsg

Transplant conditioning drug for High Risk Group only - An interferon gamma blocking antibody used to prevent inflammation/engraftment syndrome post HSC transplant.

DRUG

Tociluzumab

Transplant Conditioning Drug - An interleukin-6 (IL-6) receptor antagonist used to decrease Chronic Granulomatous Disease (CGD) inflammation during the HSC transplant process.

DRUG

Total Body Irradiation

Transplant Conditioning - Total Body Irradiation (300 cGy in fractionated doses) to create space n the subject's bone marrow so that the donor peripheral blood stem cells can repopulate in the subject's bone marrow.

Trial Locations (1)

20892

RECRUITING

National Institutes of Health Clinical Center, Bethesda

All Listed Sponsors
lead

National Institute of Allergy and Infectious Diseases (NIAID)

NIH