Clinical Trial Goal
To find out if allogeneic BMT is safe and works well to treat CGD
You may be able to join this trial if you:
- Are 4 – 65 years old
- Have CGD
- Agree to have other standard tests done to see if you can be in the clinical trial
Trial Details
Alemtuzumab is a monoclonal antibody that targets CD52 on certain cells.
Busulfan is a chemotherapy (chemo) drugs that block the growth of cancer.
Cyclophosphamide is a chemotherapy (chemo) drug that helps prevent graft-versus-host disease (GVHD) after allogeneic BMT.
Emapalumab is a monoclonal antibody that blocks interferon gamma in certain cells.
Sirolimus is a drug that slows down the immune system.
Tocilizumab is a monoclonal antibody that blocks IL-6 on certain cells.
Busulfan is a chemotherapy (chemo) drugs that block the growth of cancer.
Cyclophosphamide is a chemotherapy (chemo) drug that helps prevent graft-versus-host disease (GVHD) after allogeneic BMT.
Emapalumab is a monoclonal antibody that blocks interferon gamma in certain cells.
Sirolimus is a drug that slows down the immune system.
Tocilizumab is a monoclonal antibody that blocks IL-6 on certain cells.
In this trial, you’ll be put in 1 of 2 groups depending on
- Group 1 – CGD that doctors think is standard risk
- Group 2 – CGD that doctors think is higher risk
Before transplant, you’ll get treatment with the following:
- Alemtuzumab – Given as intravenous (IV) infusions on 5 days
- Busulfan – Given as IV infusions on 2 days
- Tociluzumab – Given as IV infusions on 1 or 2 days
- Total body irradiation – Group 1 only - A treatment given 2 times on 1 day
- Emapalumab – Group 2 only - Given as an IV infusion 1 time
On transplant day, the blood-forming cells from your donor are given to you through an IV infusion.
After transplant, you’ll get:
- Cyclophosphamide – Given as intravenous (IV) infusions
- Sirolimus – A pill that you take by mouth
You'll have biopsies to see how well the treatment is working. The clinical trial doctors will check your health for 5 years.
The Food and Drug Administration (FDA) has approved all of the drugs used in this trial. Using them together with allogeneic BMT to treat CGD is new and unproven.
Contacts
Sandra M Maxwell, R.N., (240) 627-3078, maxwells@mail.nih.gov
Elizabeth M Kang, M.D., (301) 402-7567, ekang@niaid.nih.gov
Locations
National Institutes of Health Clinical CenterRECRUITING
Bethesda, Maryland
For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR), 800-411-1222, ccopr@nih.gov
Sponsors
lead: National Institute of Allergy and Infectious Diseases (NIAID)
