Actively Recruiting
Haploidentical HCT for Severe Aplastic Anemia
Led by St. Jude Children's Research Hospital · Updated on 2025-07-04
21
Participants Needed
1
Research Sites
492 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This study is a prospective, single center phase II clinical trial in which patients with Severe Aplastic Anemia (SAA) ) will receive a haploidentical transplantation. The purpose of this study is to learn more about newer methods of transplanting blood forming cells donated by a family member that is not fully matched to the patient. This includes studying the effects of the chemotherapy, radiation, the transplanted cell product and additional white blood cell (lymphocyte) infusions on the patient's body, disease and overall survival. The primary objective is to assess the rate of engraftment at 30 days and overall survival (OS) and event free survival (EFS) at 1 year post-hematopoietic cell transplantation (HCT). Primary Objectives * To estimate the rate of engraftment at 30 days after TCR αβ+ T-cell-depleted graft infusion in patients receiving a single dose of post graft infusion cyclophosphamide. * To estimate the overall survival and event free survival at 1-year post transplantation. Secondary Objectives * To calculate the incidence of acute and chronic GVHD after HCT. * To calculate the rate of secondary graft rejection at 1-year post transplantation * To calculate the cumulative incidence of viral reactivation (CMV, EBV and adenovirus). * To describe the immune reconstitution after TCR αβ+ T-cell-depleted graft infusion at 1 month, 3 months, 6 months, 9 months, and 1 year. Exploratory Objectives * To longitudinally assess the phenotype and epigenetic profile of T-cells in SAA patients receiving HCT for SAA. * To assess the phenotype and epigenetic profile of T-cells in DLI administered to SAA patients post HCT. * To longitudinally assess CD8 T cell differentiation status in SAA patients using an epigenetic atlas of human CD8 T cell differentiation. * To examine the effector functions and proliferative capacity of CD8 T cells isolated from SAA patients before and after DLI. * Quantify donor derived Treg cells at different time points in patients received HCT. * Determine Treg activation status at different stages after HCT. * Are specific features of the DLI product associated with particular immune repertoire profiles post-transplant? * How does the diversity and functional profile of the DLI product alter the response to pathogens in the recipient? * Do baseline features of the recipient's innate and adaptive immune cells correlate with post-transplant immune repertoires and response profiles?
CONDITIONS
Official Title
Haploidentical HCT for Severe Aplastic Anemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 21 years or younger at enrollment
- Confirmed diagnosis of Severe Aplastic Anemia or single lineage cytopenia with low bone marrow cellularity and specific blood count criteria
- No suitable HLA-matched sibling or unrelated donor available in time
- Failed at least one immunosuppressive therapy trial or relapsed after treatment, except very severe cases
- Has a suitable single haplotype matched family donor (3 of 6 or greater)
- Patient or legal guardian signed informed consent
- Adequate organ function including heart, kidney, lung, liver, and performance status
- Agree to use two effective contraception methods or abstinence until after last chemotherapy dose
You will not qualify if you...
- Diagnosis of Fanconi anemia
- Clinical or genetic diagnosis of dyskeratosis congenita
- Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome or myelodysplastic syndrome
- Presence of anti-donor HLA antibodies
- Prior allogeneic hematopoietic cell transplant or solid organ transplant
- Life-threatening allergic reaction to anti-thymocyte globulin
- Uncontrolled bacterial, viral, or fungal infection
- Female patients who are pregnant or breastfeeding
- Prior malignancies within 5 years except certain treated cancers
- Receipt of alemtuzumab or anti-thymocyte globulin within 2 weeks before enrollment
- Donor must be at least 18 years old, HIV negative, not pregnant or breastfeeding, and able to donate peripheral blood stem cells
- Donors with telomere disease ruled out by clinical and diagnostic tests
- Donor or legal guardian must be able to sign informed consent
AI-Screening
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Trial Site Locations
Total: 1 location
1
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Actively Recruiting
Research Team
A
Amr Qudeimat, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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