Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide

EARLY_PHASE1Active, not recruitingINTERVENTIONAL
Enrollment

4

Participants

Timeline

Start Date

October 28, 2019

Primary Completion Date

June 15, 2034

Study Completion Date

June 15, 2034

Conditions
Chronic Granulomatous Disease
Interventions
DRUG

Busulfan

3 days of IV Busulfan. An alkylating chemotherapeutic agent determined to have broad myelosuppressive effects. On this study is being used as part of conditioning regimen for myelosuppressive properties as per package insert and standard of care.

DRUG

Alemtuzumab

5 days of IV Alemtuzumab. A humanized monoclonal antibody directed against CD52 (which is abundantly expressed on all human lymphocytes), and causes T cell activation in vitro as well as complement-mediated lysis and antibody-dependent cellular toxicity. For this study, being used as part of the conditioning regimen per package insert and standard of practice.

DRUG

Cyclophosphamide

2 days of IV Cyclophosphamide. This is an antineoplastic, and for this study is bine used for its immunosuppressive mechanism of action per package insert and standard of care

DRUG

Sirolimus

Post Peripheral blood stem cell infusion. It is used for its immunosuppressive mechanism of action.

RADIATION

Total Body Irradiation

2 fractionated doses on -2 day. It is used for its immunosuppressive mechanism of action. It is SOP for conditioning for transplant.

BIOLOGICAL

Allogeneic peripheral blood stem cell

IV infused donor peripheral blood stem cell. Stem cells are cells that give rise to the blood cells - red blood cells that carry oxygen, white blood cells that help the body to fight infections, and platelets that help make the blood clot. Collected and infused per the standard of operating procedures established by the Department of Transfusion Medicine.

Trial Locations (1)

20892

National Institutes of Health Clinical Center, Bethesda

All Listed Sponsors
lead

National Institute of Allergy and Infectious Diseases (NIAID)

NIH

NCT03910452 - Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide | Biotech Hunter | Biotech Hunter