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Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)

NCT03597594 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2025-07-01

No results posted yet for this 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.

Conditions

  • Severe Combined Immunodeficiency

Interventions

DRUG

Anti-thymocyte globulin (rabbit)

given intravenously

DRUG

Busulfan

given intravenously

DRUG

Fludarabine

given intravenously

DRUG

Thiotepa

given intravenous infusion

DEVICE

CliniMACS

The mechanism of action of the CliniMACS Cell Selection System is based on magnetic-activated cell sorting (MACS). The CliniMACS device is a powerful tool for the isolation of many cell types from heterogeneous cell mixtures, (e.g. apheresis products). These can then be separated in a magnetic field using an immunomagnetic label specific for the cell type of interest, such as CD3+ human T cells.

OTHER

Donor Lymphocyte Infusion

given intravenous infusion

Sponsors & Collaborators

Principal Investigators

  • Ewelina Mamcarz, MD · St. Jude Children's Research Hospital

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
2 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-09-02
Primary Completion
2027-07-01
Completion
2028-07-01
FDA Device
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03597594 on ClinicalTrials.gov