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NCT04558736 · ClinicalTrials.gov registry record · Phase 2
Haploidentical HCT for Severe Aplastic Anemia
A Phase 2 study of Aplastic Anemia and Bone Marrow Failure Syndrome, sponsored by St. Jude Children's Research Hospital.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 21
- Enrollment target
- 1
- Study location
NCT04558736 is a Phase 2 study of Aplastic Anemia and Bone Marrow Failure Syndrome that is actively recruiting participants, run by St. Jude Children's Research Hospital. The registered enrollment target is 21 participants, below the 42-participant average among 23 other Aplastic Anemia trials with a reported enrollment target (50% lower). The trial reports 1 study location across 1 state.
The verdict
NCT04558736, a Phase 2 study of Aplastic Anemia and Bone Marrow Failure Syndrome, is actively recruiting participants, sponsored by St. Jude Children's Research Hospital.
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 21 participants
- Enrollment target
- 1
- Study location
Study Summary
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 receiv
Conditions Studied
Interventions
- DRUG Cyclophosphamide
- DRUG Fludarabine
- DRUG Mesna
- DRUG G-CSF
- DRUG Anti-Thymocyte Globulin (Rabbit)
Study Locations (1)
Tennessee
- St. Jude Children's Research Hospital - Memphis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 21 participants |
| Start Date | 2021-01-21 |
| Est. Completion | 2030-07-01 |
| Phase | Phase 2 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04558736
The ClinicalTrials.gov registry entry for NCT04558736 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 21 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 42-participant average among 23 other Aplastic Anemia trials with a reported enrollment target (50% lower). The listed sponsor is St. Jude Children's Research Hospital, which has 410 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with Aplastic Anemia appearing as the primary indexed condition, and to 5 interventions - of which Cyclophosphamide is the first listed.
NCT04558736 reports 1 study location spanning 1 distinct geographic area - top geographies include Tennessee.
Frequently Asked Questions
What is clinical trial NCT04558736 about?
NCT04558736 is a clinical study titled "Haploidentical HCT for Severe Aplastic Anemia". 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 ...
What is the current status of trial NCT04558736?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 21 participants. The study started on 2021-01-21. Estimated completion is 2030-07-01.
What conditions does trial NCT04558736 study?
This clinical trial studies the following conditions: Aplastic Anemia, Bone Marrow Failure Syndrome.
What interventions are being tested in trial NCT04558736?
The interventions under investigation include: Cyclophosphamide (DRUG), Fludarabine (DRUG), Mesna (DRUG), G-CSF (DRUG), Anti-Thymocyte Globulin (Rabbit) (DRUG).
Who is sponsoring clinical trial NCT04558736?
This trial is sponsored by St. Jude Children's Research Hospital, which has 410 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04558736 being conducted?
This trial has 1 study location across Tennessee. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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