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NCT00001255 · ClinicalTrials.gov registry record
Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study
A clinical trial, sponsored by National Human Genome Research Institute (NHGRI).
- Completed
- Registry status
- 10
- Enrollment target
NCT00001255: Completed study, sponsored by National Human Genome Research Institute (NHGRI).
NCT00001255 is a clinical trial that has completed, run by National Human Genome Research Institute (NHGRI). The registered enrollment target is 10 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00001255 has completed, sponsored by National Human Genome Research Institute (NHGRI).
- COMPLETED
- Registry status
- 10 participants
- Enrollment target
Study Summary
This study will monitor the long-term effects of gene therapy in patients with severe combined immunodeficiency disease (SCID) due to a deficiency in an enzyme called adenosine deaminase (ADA). It will also follow the course of disease in children who are not receiving gene therapy, but may have received enzyme replacement therapy with the drug PEG-ADA. ADA is essential for the growth and proper functioning of infection-fighting white blood cells called T and B lymphocytes. Patients who lack this enzyme are, therefore, immune deficient and vulnerable to frequent infections. Injections of PEG-ADA may increase the number of immune cells and reduce infections, but this enzyme replacement therapy is not a definitive cure. In addition, patients may become resistant or allergic to the drug. Gene therapy, in which a normal ADA gene is inserted into the patient's cells, attempts to correcting the underlying cause of disease. Patients with SCID due to ADA deficiency may be eligible for this study. Patients may or may not have received enzyme replacement therapy or gene transfer therapy, or both. Participants will have follow-up visits at the National Institutes of Health in Bethesda, Maryland, at least once a year for a physical examination, blood tests, and possibly the following additional procedures to evaluate immune function: 1. Bone marrow sampling - A small amount of marrow from the hip bone is drawn (aspirated) through a needle. The procedure can be done under local anesthesia or light sedation. 2. Injection of small amounts of fluids into the arm to study if the patient's lymphocytes respond normally. 3. Administration of vaccination shots. 4. Collection of white blood cells through apheresis - Whole blood is collected through a needle placed in an arm vein. The blood circulates through a machine that separates it into its components. The white cells are then removed, and the red cells, platelets and plasma are returned to the body, either through the same needle
Interventions
- DRUG ADA PBSC
- DRUG ADA Umbilical Cord Blood Cells
- DRUG Transduced Lymphocytes
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 1990-09 |
| Est. Completion | 2002-07 |
What the finished NCT00001255 record still lists
NCT00001255 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 10 participants, a relatively small participant target.
The record links to 0 conditions, and to 3 interventions - of which ADA PBSC is the first listed.
NCT00001255 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00001255 about?
NCT00001255 is a clinical study titled "Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study". This study will monitor the long-term effects of gene therapy in patients with severe combined immunodeficiency disease (SCID) due to a deficiency in an enzyme called adenosine deaminase (ADA). It will also follow the course of disease in children who are not receiving gene therapy, but may have rec...
What is the current status of trial NCT00001255?
This trial is currently completed. The enrollment target is 10 participants. The study started on 1990-09. Estimated completion is 2002-07.
What interventions are being tested in trial NCT00001255?
The interventions under investigation include: ADA PBSC (DRUG), ADA Umbilical Cord Blood Cells (DRUG), Transduced Lymphocytes (DRUG).
Who is sponsoring clinical trial NCT00001255?
This trial is sponsored by National Human Genome Research Institute (NHGRI), which has 185 total clinical trials registered on ClinicalTrials.gov.
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