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NCT00018018 · ClinicalTrials.gov registry record · Phase 1
Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency
A Phase 1 study of Severe Combined Immunodeficiency Syndrome, sponsored by National Human Genome Research Institute (NHGRI).
- Completed
- Registry status
- Phase 1
- Development phase
- 8
- Enrollment target
- 1
- Study location
NCT00018018 is a Phase 1 study of Severe Combined Immunodeficiency Syndrome that has completed, run by National Human Genome Research Institute (NHGRI). The registered enrollment target is 8 participants. The trial reports 1 study location across 1 state.
The verdict
NCT00018018, a Phase 1 study of Severe Combined Immunodeficiency Syndrome, has completed, sponsored by National Human Genome Research Institute (NHGRI).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 8 participants
- Enrollment target
- 1
- Study location
Study Summary
This study will evaluate a new method for delivering gene transfer therapy to patients with severe combined immunodeficiency disease (SCID) due to a defective adenosine deaminase (ADA) gene. This gene codes for the adenosine deaminase enzyme, which is essential for the proper growth and function of infection-fighting white blood cells called T and B lymphocytes. Patients who lack this enzyme are vulnerable to frequent and severe infections. Some patients with this disease receive enzyme replacement therapy with weekly injections of the drug PEG-ADA (ADAGEN). This drug may increase the number of immune cells and reduce infections, but it is not a cure. Gene transfer therapy, in which a normal ADA gene is inserted into the patient s cells, attempts to correct the underlying cause of disease. This therapy has been tried in a small number of patients with varying degrees of success. In this study, the gene will be inserted into the patient s stem cells (cells produced by the bone marrow that mature into the different blood components white cells, red cells and platelets). Patients with ADA deficiency and SCID who are taking PEG-ADA and are not candidates for HLA-identical sibling donor bone marrow transplantation may be eligible for this study. Participants will be admitted to the NIH Clinical Center for 2 to 3 days. Stem cells will be collected either from cord blood (in newborn patients) or from the bone marrow. The bone marrow procedure is done under light sedation or general anesthesia. It involves drawing a small amount of marrow through a needle inserted into the hip bone. The stem cells in the marrow will be grown in the laboratory and a normal human ADA gene will be transferred into them through a special type of disabled mouse virus. A few days later, the patient will receive the ADA-corrected cells through an infusion in the vein that will last from 10 minutes to 2 hours. Patients will be evaluated periodically for immune function with blood tests, skin te
Conditions Studied
Interventions
- DRUG CD34+ cells transduced with ADA retrovir
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center, 9000 Rockville Pike - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 8 participants |
| Start Date | 2001-06-20 |
| Est. Completion | 2014-09-17 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00018018
The ClinicalTrials.gov registry entry for NCT00018018 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 8 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Human Genome Research Institute (NHGRI), which has 185 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Severe Combined Immunodeficiency Syndrome appearing as the primary indexed condition, and to 1 intervention - of which CD34+ cells transduced with ADA retrovir is the first listed.
NCT00018018 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.
Frequently Asked Questions
What is clinical trial NCT00018018 about?
NCT00018018 is a clinical study titled "Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency". This study will evaluate a new method for delivering gene transfer therapy to patients with severe combined immunodeficiency disease (SCID) due to a defective adenosine deaminase (ADA) gene. This gene codes for the adenosine deaminase enzyme, which is essential for the proper growth and function of ...
What is the current status of trial NCT00018018?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 8 participants. The study started on 2001-06-20. Estimated completion is 2014-09-17.
What conditions does trial NCT00018018 study?
This clinical trial studies the following conditions: Severe Combined Immunodeficiency Syndrome.
What interventions are being tested in trial NCT00018018?
The interventions under investigation include: CD34+ cells transduced with ADA retrovir (DRUG).
Who is sponsoring clinical trial NCT00018018?
This trial is sponsored by National Human Genome Research Institute (NHGRI), which has 185 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00018018 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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