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NCT00228852 · ClinicalTrials.gov registry record · Phase 1

IMM 0212: Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell Immunodeficiency

A Phase 1 study of Severe Combined Immunodeficiency and T-Cell Immune Deficiency Diseases, sponsored by Emory University.

Compiled by PlainTrial · August 8, 2026

Completed
Registry status: Phase 1
Development phase: 1
Study location

NCT00228852: Completed Phase 1 study of Severe Combined Immunodeficiency and T-Cell Immune Deficiency Diseases, sponsored by Emory University.

NCT00228852 is a Phase 1 study of Severe Combined Immunodeficiency and T-Cell Immune Deficiency Diseases that has completed, run by Emory University. The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT00228852, a Phase 1 study of Severe Combined Immunodeficiency and T-Cell Immune Deficiency Diseases, has completed, sponsored by Emory University.

COMPLETED
Registry status: Phase 1
Development phase: 1
Study location

Study Summary

This is a multi-institution, single arm, non-randomized pilot study coordinated by the Pediatric Blood and Marrow Transplant Consortium. Eligible patients will have severe combined immunodeficiency syndrome (SCID) or severe T-cell immunodeficiency disorder. Patients with these disorders do not have properly functioning immune systems. Without treatment, these disorders result in early childhood death. The standard treatment used for these diseases is to give the patient a stem cell transplant from a matched donor. The donor cells can be from a family member, an unrelated marrow donor or umbilical cord blood. The donor source will impact on transplant risks and approaches to the preparative regimen. There have been many different preparative regimens used for patients with SCIDS or severe T-cell immunodeficiency syndromes. Some patients have gotten no preparative regimen, while others have gotten only antithymocyte globulin (ATG; immune proteins made in horses that, when given, will kill lymphocytes). Still other patients have gotten conventional chemotherapy. In children treated with nothing or ATG alone, there is an increased risk of graft failure or only partial engraftment. When this happens, patients need life-long therapy with immunoglobulins to support the immune system. Children treated with chemotherapy generally have full immune recovery, but also may have major side effects from the chemotherapy. The side effects include infection, organ failure and infertility. This protocol, in combination with a parallel study with a separate preparative regimen, will attempt to answer the question of which patients with primary immunodeficiencies need a preparative regimen and what intensity is needed. Patients will be enrolled according to disease type and donor source. The purpose of this study is to see how much chemotherapy is actually needed for the transplant to work. To be able to do this and still make the transplant work, the drugs used to temporarily weaken

Conditions Studied

Severe Combined Immunodeficiency T-Cell Immune Deficiency Diseases

Interventions

DRUG Busulfan, Fludarabine and ATG

Study Locations (1)

Georgia

Children's Healthcare of Atlanta - Atlanta

Trial Details

Field	Value
Est. Completion	2006-11
Phase	Phase 1

Emory University

1,208 total trials

What the finished NCT00228852 record still lists

NCT00228852 is an interventional study that assigns participants to a tested intervention. An enrollment target was not published in the registry record, which is common for early-stage or observational entries.

The record links to 2 conditions, with Severe Combined Immunodeficiency appearing as the primary indexed condition, and to 1 intervention - of which Busulfan, Fludarabine and ATG is the first listed.

NCT00228852 reports a single indexed study location in Georgia.

Frequently Asked Questions

What is clinical trial NCT00228852 about?

NCT00228852 is a clinical study titled "IMM 0212: Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell Immunodeficiency". This is a multi-institution, single arm, non-randomized pilot study coordinated by the Pediatric Blood and Marrow Transplant Consortium. Eligible patients will have severe combined immunodeficiency syndrome (SCID) or severe T-cell immunodeficiency disorder. Patients with these disorders do not have ...

What is the current status of trial NCT00228852?

This trial is currently completed. It is a Phase 1 study. Estimated completion is 2006-11.

What conditions does trial NCT00228852 study?

This clinical trial studies the following conditions: Severe Combined Immunodeficiency, T-Cell Immune Deficiency Diseases.

What interventions are being tested in trial NCT00228852?

The interventions under investigation include: Busulfan, Fludarabine and ATG (DRUG).

Who is sponsoring clinical trial NCT00228852?

This trial is sponsored by Emory University, which has 1,208 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT00228852 being conducted?

This trial has 1 study location across Georgia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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Source: ClinicalTrials.gov NCT00228852, the US trial registry maintained by the National Library of Medicine. NCT00228852 (unsized enrollment · single site footprint · completed) retrieved and formatted by PlainTrial, see methodology.