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Haploidentical HCT for Severe Aplastic Anemia

StatusRecruiting
PhasePhase 2
Started2021-01-21
View on ClinicalTrials.gov ↗

Amendment history

2025-07-03
Study Status v11
Primary completion date2025-07-01→2029-07-01
Completion date2026-07-01→2030-07-01
2024-10-14
Study Status v10
Re-verified, no change to tracked fields
2024-08-21
Active, Not Recruiting→Recruiting Study Status, Contacts/Locations v9
Trial statusActive, Not Recruiting→Recruiting
Study sitesdetails revised at 1 of 1 site
2023-08-17
Recruiting→Active, Not Recruiting Study Status, Contacts/Locations v8
Trial statusRecruiting→Active, Not Recruiting
Study sitesdetails revised at 1 of 1 site
2022-08-26
Study Status v7
Re-verified, no change to tracked fields
Show 6 earlier versions
2021-08-26
Study Status, Study Description, Conditions, Arms and Interventions, Eligibility, Contacts/Locations, References v6
Eligibility criteria-157 characters
[...] years at time of enrollment. Confirmed diagnosis of SAA or TBD:a single lineage cytopenia (a) SAA or single lineage cytopenia will be defined as follows: [...] Hemoglobin <8g/dL, or red blood cell transfusion dependence TBD will be defined as follows: i. Hypocellular bone marrow failure and clinically relevant cytopenias, AND ii. Age-adjusted mean telomere length < 1%ile in peripheral blood lymphocytes on any occasion, as reported by a CLIA-approved laboratory Does not have a suitable HLA-matched sibling donor (MSD) or [...] t be excluded by diepoxybutane (DEB) or equivalent testing. Known clinical or ge [...] [...]
2021-03-29
Study Status v5
Start dateestimated 2021-02→confirmed 2021-01-21
2021-02-02
Study Status v4
Start date2021-01→2021-02
2021-01-06
Study Status v3
Start date2020-11→2021-01
2020-10-06
Not Yet Recruiting→Recruiting Study Status, Contacts/Locations v2
Trial statusNot Yet Recruiting→Recruiting
Start date2020-09→2020-11
Study sitesdetails revised at 1 of 1 site
2020-09-22
Study Status v1
Re-verified, no change to tracked fields
2020-09-15
Original filing
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?
Trial Details
NCT Number NCT04558736
Lead Sponsor St. Jude Children's Research Hospital
Conditions Aplastic Anemia, Bone Marrow Failure Syndrome
Enrollment 21 participants
Start Date 2021-01-21
Primary Completion 2029-07-01 (estimated)
Study Completion 2030-07-01 (estimated)
Updated on ClinicalTrials.gov 2026-09-16