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.

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NCT04558736

Haploidentical HCT for Severe Aplastic Anemia

Recruiting
PHASE2Up to 21InterventionalTreatment
St. Jude Children's Research Hospital
~21 participants
Updated 2025-07-04 on ClinicalTrials.gov
What's tested:Anti-Thymocyte Globulin (Rabbit)FludarabineCyclophosphamideMesnaG-CSFTotal Lymphoid Irradiation (TLI)

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Engraftment
Measured over 30 days
+1 more outcome measured
Aplastic Anemia
Bone Marrow Failure Syndrome
1 sites across 1 states
Tennessee1
  • Amr Qudeimat, MD · PRINCIPAL_INVESTIGATOR · St. Jude Children's Research Hospital
  • Akshay Sharma, MBBS · PRINCIPAL_INVESTIGATOR · St. Jude Children's Research Hospital

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Eligibility criteria

Inclusion

i. Bone marrow cellularity \< 25% or hypocellular marrow for age, AND
ii. One or more of the following (in peripheral blood): (i) Neutrophils \< 0.5 x10\^9/L (ii) Platelets \< 20 x10\^9/L, or platelet transfusion dependence (iii) Hemoglobin \<8g/dL, or red blood cell transfusion dependence 3. Does not have a suitable HLA-matched sibling donor (MSD) or volunteer 10/10 HLA-matched unrelated donor (MUD) available in the necessary time for progenitor cell donation. 4. Failed at least one trial of immunosuppressive therapy (IST) by being refractory (persistence of severe cytopenias and fulfillment of SAA disease criteria at least 3 months after initial IST) or having relapsed (initial improvement of cytopenias after first-line IST but then a later return to fulfillment of SAA disease criteria when IST is decreased or ceased). IST could have included ATG based regimens, calcineurin inhibitors and/or other higher dose therapy directed at the treatment of primary SAA. Patients with very severe aplastic anemia who are likely not to benefit from IST do not need to have failed a trial of IST and can proceed directly to HCT if they meet the rest of the criteria. 5. Has a suitable single haplotype matched (≥ 3 of 6) family member donor. 6. Patient and/or legal guardian must sign informed consent for HCT. 7. Adequate organ function defined as:
  • 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.