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NCT00028236 · ClinicalTrials.gov registry record · Phase 1
Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)
A Phase 1 study, sponsored by National Institute of Allergy and Infectious Diseases (NIAID).
- Completed
- Registry status
- Phase 1
- Development phase
- 3
- Enrollment target
NCT00028236 is a Phase 1 study that has completed, run by National Institute of Allergy and Infectious Diseases (NIAID). The registered enrollment target is 3 participants.
The verdict
NCT00028236, a Phase 1 study, has completed, sponsored by National Institute of Allergy and Infectious Diseases (NIAID).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 3 participants
- Enrollment target
Study Summary
This is a clinical trial of gene therapy for X-linked severe combined immunodeficiency (XSCID), a genetic disease caused by defects in a protein called the common gamma chain, which is normally on the surface of immune cells called lymphocytes. XSCID patients cannot make T lymphocytes, and their B lymphocytes fail to make essential antibodies for fighting infections. Without T and B lymphocytes patients develop fatal infections in infancy unless they are rescued by a bone marrow transplant from a healthy donor. However, even transplanted patients may achieve only partial immune recovery and still suffer from many infections, auto-immunity and/or and poor growth. A recent, successful trial in France used gene therapy instead of bone marrow transplantation for infants with XSCID. This experience indicates that gene therapy can provide clinical benefit to XSCID patients. We will enroll eight older XSCID patients (1.5-20 years-old), who have previously received at least one bone marrow transplant, but still have poor T and B lymphocyte function that compromises their quality of life. Before enrollment, these subjects will have had some of their own blood-forming stem cells harvested and frozen in a blood bank. These cells have a defective gene, but a correct copy of the gene will be inserted while the cells are grown in sterile conditions outside the patient's body. To do this, the cells will be unfrozen and exposed for four days in a row to growth factors and particles of a retrovirus we have constructed and tested called "GALV MFGS-gc." Retrovirus particles will attach to the patient cells and introduce a correct copy of the common gamma chain gene into cells capable of growing into all types of blood cells, including T and B lymphocytes. XSCID patients who are enrolled in the study will receive a single dose of their own cells that have been modified by the GALV MFGS-gc treatment and also will be given another drug called palifermin to help prevent side effects from
Interventions
- DRUG Gene-Transduced Autologous CD34+ Stem Cells
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 3 participants |
| Start Date | 2001-12-10 |
| Est. Completion | 2011-07-25 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00028236
The ClinicalTrials.gov registry entry for NCT00028236 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 3 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Institute of Allergy and Infectious Diseases (NIAID), which has 2,006 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Gene-Transduced Autologous CD34+ Stem Cells is the first listed.
NCT00028236 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00028236 about?
NCT00028236 is a clinical study titled "Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)". This is a clinical trial of gene therapy for X-linked severe combined immunodeficiency (XSCID), a genetic disease caused by defects in a protein called the common gamma chain, which is normally on the surface of immune cells called lymphocytes. XSCID patients cannot make T lymphocytes, and their B l...
What is the current status of trial NCT00028236?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 3 participants. The study started on 2001-12-10. Estimated completion is 2011-07-25.
What interventions are being tested in trial NCT00028236?
The interventions under investigation include: Gene-Transduced Autologous CD34+ Stem Cells (DRUG).
Who is sponsoring clinical trial NCT00028236?
This trial is sponsored by National Institute of Allergy and Infectious Diseases (NIAID), which has 2,006 total clinical trials registered on ClinicalTrials.gov.
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