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ACTIVE NOT RECRUITING Phase 1

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

NCT03597594 · View on ClinicalTrials.gov ↗

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.

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

FieldValue
Enrollment Target 4 participants
Start Date 2021-09-02
Est. Completion 2028-07-01
Phase Phase 1

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT03597594

The ClinicalTrials.gov registry entry for NCT03597594 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 4 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is St. Jude Children's Research Hospital, which has 441 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT03597594 reports 1 study location spanning 1 distinct geographic area — top geographies include Tennessee. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

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). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT03597594?

This trial is sponsored by St. Jude Children's Research Hospital, which has 441 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

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.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial