Haploidentical Transplant for Severe Aplastic Anemia in Children
This study is looking at a new way to perform blood stem cell transplants for children and young adults (up to 21 years old) with severe aplastic anemia (a condition where your body stops producing enough new blood cells) or a related bone marrow problem. It's called a "haploidentical" transplant because the donor is a family member who is only a half-match, which can make it easier to find a donor. Researchers are studying how well the transplanted cells grow and how safe the process is, including the effects of medicines like Anti-Thymocyte Globulin, Fludarabine, Cyclophosphamide, Mesna, and G-CSF. The main goals are to see if the new cells successfully grow in your body within 30 days and to track your overall health and survival for one year after the transplant. This study aims to reduce the risk of graft-versus-host disease (GVHD), a common complication of transplants.
- Study design
- This is a single-center study that plans to enroll 21 participants. It is an interventional study, meaning participants will receive specific treatments.
- What's involved
- You would receive chemotherapy, antibodies, and radiation to prepare your body for the donor cells. You would then receive two types of donor blood cell infusions.
- Compensation
- Not stated in the trial record.
- Follow-up
- Researchers will track your health for at least one year after the transplant to assess overall and event-free survival.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Haploidentical HCT for Severe Aplastic Anemia
At a glance
Conditions
Where it's being run
1 sites across 1 statesStudy leadership
- Amr Qudeimat, MD · PRINCIPAL_INVESTIGATOR · St. Jude Children's Research Hospital
- Akshay Sharma, MBBS · PRINCIPAL_INVESTIGATOR · St. Jude Children's Research Hospital
Who to contact
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Do you actually qualify for this trial?
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Inclusion
What this trial measures
- Engraftment30 days
Rate of patients engrafting at day 30 after TCR αβ+ T-cell-depleted graft infusion in patients receiving a single dose of post graft infusion cyclophosphamide.
- Overall and event free survival1 year
Rate of overall survival and event free survival at 1-year post transplantation.