Clinical Trial Goal
To find out if allogeneic BMT with TCR alpha beta-depletion is safe and works well to prevent graft-versus-host disease (GVHD) and to treat bone marrow failure disorders
You may be able to join this trial if you:
- Are 70 years old or younger
- Have one of the following bone marrow disorders:
- Dyskeratosis Congenita
- Fanconi anemia
- T Cell Receptor Alpha/Beta Depletion
- Telomere Biology Disorder
- Severe aplastic anemia (SAA)
- Have a donor who is your full or half match (haploidentical). Your doctor can tell you this
- Have not been diagnosed with a solid tumor cancer in the last 2 years
- 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.
Cyclophosphamide is a drug that doctors think will prevent GVHD.
Fludarabine, is a chemotherapy (chemo) drug that blocks the growth of cancer cells.
Granulocyte colony-stimulating factor (G-CSF) helps normal white blood cells recover from the transplant.
Rituximab is a monoclonal antibody that targets CD20 on certain cells.
Some people in this trial will get total body irradiation (TBI) given 1 time 6 days before transplant.
Everyone, will get standard drugs to prepare their body for transplant. Drugs you may get include:
Cyclophosphamide is a drug that doctors think will prevent GVHD.
Fludarabine, is a chemotherapy (chemo) drug that blocks the growth of cancer cells.
Granulocyte colony-stimulating factor (G-CSF) helps normal white blood cells recover from the transplant.
Rituximab is a monoclonal antibody that targets CD20 on certain cells.
Some people in this trial will get total body irradiation (TBI) given 1 time 6 days before transplant.
Everyone, will get standard drugs to prepare their body for transplant. Drugs you may get include:
- Alemtuzumab - Given as intravenous (IV) infusions 1 time each day for 5 days.
- Busulfan – Given as IV infusions 2 times each day for 2 days. The clinical trial doctors will decide who gets this
- Cyclophosphamide – Given as IV infusions 1 time each day for 4 days
- Fludarabine – Given as IV infusions 1 time each day for 4 days
- Melphalan - Given as an IV infusion 1 time
- Methylprednisolone – Given as IV infusions 2 times each day for 5 days
- Rituximab – Given as an IV infusion 1 time
Your donor’s blood-forming cells will be processed in a lab to remove the type of T cells that are more likely to cause GVHD after transplant. On transplant day, the blood-forming cells from your donor are given to you through an IV infusion.
You’ll then get G-CSF given as IV infusions each day until their blood counts recover.
You will have biopsies to see how well treatment is working. The clinical trial doctors will check your health for 1 year after transplant.
The Food and Drug Administration (FDA) has approved all of the drugs in this trial. Using TCR alpha beta-depleted allogeneic BMT to treat bone marrow failure disorders is new and unproven.
Locations
Masonic Cancer Center at University of MinnesotaRECRUITING
Minneapolis, Minnesota
Lisa Burke, RN, 612-273-8482, lburke3@Fairview.org
Sponsors
lead: Masonic Cancer Center, University of Minnesota
