Featured Trial
Our review committee has reviewed this trial and it meets our criteria for going above and beyond. Learn more about featured trials.
Clinical Trial Goal
To find out whether allogeneic BMT or immunosuppressive therapy is more effective in treatment of SAA that has not yet been treated in children, teens and young adults
You may be able to join this trial if you:
You or your child:
- Is 25 years old or younger
- Has SAA
- Has two bone marrow donor matches on the public registry
- Does not have a family member who is their full match and can be their stem cell donor
- Does not have dyskeratosis congenita, Fanconi anemia, Shwachman-Diamond syndrome or other forms of inherited (genetic) marrow failure syndromes
- Has not had a BMT
- You agree to have other standard tests done to see if your child can be in the clinical trial
Trial Details
Anti-thymocyte globulin (ATG) and cyclosporine are standard drugs used to prevent graft-versus-host disease (GVHD) or to slow down the immune system.
Cyclophosphamide and fludarabine are chemotherapy (chemo) drugs that slow down the immune system.
Methotrexate is a chemo drug that prevents GVHD.
Cyclophosphamide and fludarabine are chemotherapy (chemo) drugs that slow down the immune system.
Methotrexate is a chemo drug that prevents GVHD.
In this trial, you or your child will be randomized to 1 of 2 groups. Once randomized, you or your child will be told what group you/they're in:
- Group 1 – Standard immunosuppressive therapy
- Group 2 – Unrelated allogeneic BMT
Randomized means doctors will use a computer to assign you or your child to either group. A computer assigns by chance, like flipping a coin or drawing a name out of a hat. You, your child's doctor or the clinical trial doctor won’t have any control over which group is assigned. This means you won’t be able to choose you o your child's group.
In Group 1, you or your child will get standard immunosuppressive therapy with:
- Cyclosporine - A pill taken by mouth
- ATG – Given as intravenous (IV) infusions
In Group 2, you or your child will get an allogeneic BMT.
First, you/they will get:
- ATG - Given as IV infusions
- Cyclophosphamide – Given as IV infusions
- Fludarabine – Given as IV infusions
- Total body irradiation (TBI) – A treatment done 1 time to prepare the body for transplant
On transplant day, the blood-forming cells from you or your child's donor are given through an IV infusion.
After transplant, you or your child will get:
- Cyclosporine – Given as IV infusions
- Methotrexate – Given as IV infusions
You or your child may continue treatment for as long as the clinical trial doctors think it’s best for your/their health. Biopsies will be done to see how well the treatment is working. The clinical trial doctors will check you or your child's health for up to 5 years.
The Food and Drug Administration (FDA) has approved allogeneic BMT and all of the drugs used in this trial.
The Food and Drug Administration (FDA) has approved allogeneic BMT and all of the drugs used in this trial.
Contacts
Kristi Wilmes, MS, (763) 406-3416, kwilmes@NMDP.ORG
Leah Cheng, MA, 857-218-4731, leah.cheng@childrens.harvard.edu
Locations
Sponsors
lead: Boston Children's Hospital, collaborator: Center for International Blood and Marrow Transplant Research, collaborator: National Institutes of Health (NIH), collaborator: National Heart, Lung, and Blood Institute (NHLBI), collaborator: North American Pediatric Aplastic Anemia Consortium, collaborator: Pediatric Transplantation and Cellular Therapy Consortium, collaborator: Blood and Marrow Transplant Clinical Trials Network
