Clinical Trial Goal
To find out if haploidentical BMT with TCR alpha beta depletion and CD19 depletion is safe and works well to treat blood and marrow disorders in children, teens and young adults
You may be able to join this trial if you:
Your child:
- Is 0-21 years old
- Has one of the following blood or marrow disorders:
- Bone marrow failure syndromes including:
- Amegakaryocytic thrombocytopenia (AMT)
- Autoimmune hemolytic anemia (AIHA)
- Severe aplastic anemia (SAA)
- Diamond blackfan anemia (DBA)
- Fanconi anemia
- Glanzmann Thrombasthenia
- Immune thrombocytopenia
- Pure red cell aplasia (PRCA)
- Severe congenital neutropenia
- Shwachman-diamond syndrome
- Other marrow failure disorders not otherwise specified
- Sickle cell disease (SCD)
- Thalassemia major
- Bone marrow failure syndromes including:
- Has a family member who is a half match and can be a donor. Biological parents and children are always a half-match for you
- Does not have a family member who is a full match and can be a donor
- Does not have GVHD higher than grade 2. Your doctor can tell you this
- Does not have myelodysplastic syndrome (MDS)
- Does not have acute myeloid leukemia (AML)
- You agree to have other standard tests done to see if your child can be in the clinical trial
Trial Details
Before transplant, you’ll get standard drugs to prepare your body for transplant.
The donor's blood-forming cells will be processed in a lab to take out the type of T cells that are more likely to cause GVHD after transplant.
On transplant day, the blood-forming cells from your child's donor are given to them through an intravenous (IV) infusion.
Your child will have biopsies and/or scans to see how well the treatment is working. The clinical trial doctors will check your child's health for up to 2 years.
Haploidentical BMT using donated cells with with TCR alpha beta depletion and CD19 depletion to treat blood and marrow disorders in children, teens and young adults is new and unproven.
On transplant day, the blood-forming cells from your child's donor are given to them through an intravenous (IV) infusion.
Your child will have biopsies and/or scans to see how well the treatment is working. The clinical trial doctors will check your child's health for up to 2 years.
Haploidentical BMT using donated cells with with TCR alpha beta depletion and CD19 depletion to treat blood and marrow disorders in children, teens and young adults is new and unproven.
Locations
Sponsors
lead: Johns Hopkins All Children's Hospital
