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NCT03597594 · ClinicalTrials.gov registry record · Phase 1
Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)
A Phase 1 study of Severe Combined Immunodeficiency, sponsored by St. Jude Children's Research Hospital.
- Active
- Registry status
- Phase 1
- Development phase
- 4
- Enrollment target
- 1
- Study location
NCT03597594: Active Phase 1 study of Severe Combined Immunodeficiency, sponsored by St. Jude Children's Research Hospital.
NCT03597594 is a Phase 1 study of Severe Combined Immunodeficiency that is active but no longer recruiting, run by St. Jude Children's Research Hospital. The registered enrollment target is 4 participants, below the 397-participant average among 5 other Severe Combined Immunodeficiency trials with a reported enrollment target (99% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03597594, a Phase 1 study of Severe Combined Immunodeficiency, is active but no longer recruiting, sponsored by St. Jude Children's Research Hospital.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 4 participants
- Enrollment target
- 1
- Study location
Study Summary
Infants with severe combined immunodeficiency (SCID) have a profound decrease in number and function of immune cells, and therefore remain highly vulnerable to infection. If not corrected this often leads to death. Hematopoietic cell transplantation (HCT) from matched sibling donor is the standard treatment for these patients, unfortunately though; most SCID patients lack a sibling donor. Building upon experience and existing data, the investigators are proposing a trial the goals of which are: to provide a conditioning regimen that is well tolerated, and provision of immune cells that altogether should establish rapid immune recovery providing protection from life threatening infections without increasing the risk of dangerous Graft-Versus-Host-Disease. Primary Objectives 1. To evaluate the safety of a TCRα/β/CD19-depleted graft with CD45RA-depleted DLI in infants with SCID 2. To estimate overall survival at 1 year post transplantation Exploratory Objectives 1. To evaluate the significant donor T cell reconstitution of a TCRα/β/CD19 depleted graft with CD45RA-depleted DLI at 1 year (+/-2 weeks). 2. To evaluate engraftment at day 30, 100, month 6, and years 1 to 10 post HCT. 3. To evaluate B cell reconstitution at years 1 to 10 post HCT. 4. To evaluate biomarkers of immune reconstitution at day 30, 60 100, month 6 and years 1 to 10; e.g. immunophenotype (including epigenetic profiling) of T, B, and NK cells, and assays to determine their function. 5. To evaluate clinical outcomes, post HCT. 6. To define the incidence and severity of acute (at day 100, month 6), and chronic (month 6, 12, 24) GVHD following HCT.
Primary Outcome
Treatment related deaths will be considered as one of the primary measures to evaluate the safety of a TCRα/β/CD19-depleted graft with CD45RA-depleted DLI in infants with SCID. Number of patients with treatment related deaths will be provided.
Conditions Studied
Interventions
- DRUG Fludarabine
- DRUG Busulfan
- DRUG Thiotepa
- DEVICE CliniMACS
- DRUG Anti-thymocyte globulin (rabbit)
Study Locations (1)
Tennessee
- St. Jude Children's Research Hospital - Memphis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2021-09-02 |
| Est. Completion | 2028-07-01 |
| Phase | Phase 1 |
What the registry record for NCT03597594 still lists
NCT03597594 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 4 participants, a relatively small participant target, below the 397-participant average among 5 other Severe Combined Immunodeficiency trials with a reported enrollment target (99% lower).
The record links to 1 condition, with Severe Combined Immunodeficiency appearing as the primary indexed condition, and to 5 interventions - of which Fludarabine is the first listed.
NCT03597594 reports a single indexed study location in Tennessee.
Frequently Asked Questions
What is clinical trial NCT03597594 about?
NCT03597594 is a clinical study titled "Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)". Infants with severe combined immunodeficiency (SCID) have a profound decrease in number and function of immune cells, and therefore remain highly vulnerable to infection. If not corrected this often leads to death. Hematopoietic cell transplantation (HCT) from matched sibling donor is the standard t...
What is the current status of trial NCT03597594?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 4 participants. The study started on 2021-09-02. Estimated completion is 2028-07-01.
What conditions does trial NCT03597594 study?
This clinical trial studies the following conditions: Severe Combined Immunodeficiency.
What interventions are being tested in trial NCT03597594?
The interventions under investigation include: Fludarabine (DRUG), Busulfan (DRUG), Thiotepa (DRUG), CliniMACS (DEVICE), Anti-thymocyte globulin (rabbit) (DRUG).
Who is sponsoring clinical trial NCT03597594?
This trial is sponsored by St. Jude Children's Research Hospital, which has 410 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03597594 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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