Clinical Trial Goal
To find out if the combination of treosulfan, fludarabine and rATG before allogeneic BMT is safe and works well to treat BMF disorders in children, teens and adults
You may be able to join this trial if you:
- Are 1 - 49 years old
- Have a bone marrow failure disorder that can be treated with allogeneic BMT. Some examples are:
- Congenital amegakaryocytic thrombocytopenia (CAMT)
- Congenital sideroblastic anemia (CSA)
- Diamond Blackfan anemia (DFA)
- GATA2 deficiency
- Paroxysmal nocturnal hemoglobinuria (PNH)
- SAMD9 disorder
- SAMD9L disorder
- Shwachman-Diamond syndrome (SDS)
- Other bone marrow failure disorder, even if the cause is unknown
- Do not have idiopathic aplastic anemia
- Do not have Fanconi anemia
- Do not have dyskeratosis congenita (telomere biology disorder (TBD))
- Do not have congenital neutropenia
- Do not have myelodysplastic syndrome (MDS)
- Do not have leukemia
- Have not had an allogeneic BMT
- Agree to have other standard tests done to see if you can be in the clinical trial
Trial Details
rATG and tacrolimus are standard drugs used to prevent graft-versus-host disease (GVHD).
Fludarabine, methotrexate and treosulfan are chemotherapy (chemo) drugs that block the growth of cancer.
Fludarabine, methotrexate and treosulfan are chemotherapy (chemo) drugs that block the growth of cancer.
Before transplant, you'll get:
- Fludarabine – Given as intravenous (IV) infusions 1 time each day for 5 days. Each infusion takes 1 hour
- rATG – Given as IV infusions 1 time each day for 3 days. Each infusion takes up to 6 hours
- Tacrolimus – Given as IV infusions starting 2 days before transplant day
- Treosulfan – Given as IV infusions 1 time each day for 3 days. Each infusion takes 2 hours
On transplant day, the blood-forming cells from your donor are given to you through an IV infusion.
After transplant, you'll get:
- Methotrexate – Given as IV infusions 4 times
- Tacrolimus – Given as IV infusions or as a pill that you take by mouth until at least 6 months after transplant
You'll have have biopsies to see how well the treatment is working. The clinical trial doctors will check your health for 1 year.
The Food and Drug Administration (FDA) has approved all of the drugs used in this trial.
Locations
Sponsors
collaborator: Blood and Marrow Transplant Clinical Trials Network, collaborator: National Cancer Institute (NCI), collaborator: National Marrow Donor Program, collaborator: National Heart, Lung, and Blood Institute (NHLBI), lead: Fred Hutchinson Cancer Center
