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Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study Clinical Trials Information presented on Clinical Trials Search is not designed to be a substitute for proven healthcare advice, travels to or treatment by using a genuine medical doctor. We are not physicians. Always confer with your doctor on Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study conditions. Clinical Trials Search.org is a site devoted to listing clinical research studies in human subjects. Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study Clinical research trials and Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study healthcare trials take place in many of cities across the United States of America. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials generally evaluate the effectiveness of new drugs. The function of the studies / undertakings is to answer specific human medical questions. Clinical trials are a popular means for mDs, government agencies, and private sector companies to find treatments for all forms of conditions, including Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study. Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study Clinical Trials and other clinical trials allow for volunteers to access medical treatment alternatives before they are available to the masses. Many times the test subjects undergo treatment for without cost, and occasionally they are compensated for their time. Occasionally there is a cost for a Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study clinical trial. Test subjects oftentimes recieve the best healthcare possible for their Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study condition. Hazards are a reality, nonetheless, and might include additional or frequent doctor trips, healthcare hazards (perhaps life-jeopardizing), and/or the treatment being ineffective. Trials are federally regulated with rigid guidelines to protect clinical trials subjects.
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Home > "G" Clinical Trials Conditions > Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study  Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study
Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study
For Condition: Severe Combined Immunodeficiency
Status: Completed
Sponsor(s): National Human Genome Research Institute (NHGRI) ,
Synopsis: 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 used to draw the blood or through a second needle placed in the other arm. 5. Blood drawings to obtain and study the patient's lymphocytes.
Details: The primary purpose of this study is to continue to provide clinical follow-up for ADA-deficient patients treated with gene therapy under the original protocol 90-HG-0195 (IND 3624) and its amendments (IND 4647 and IND 5056). The objectives are the long-term monitoring of the beneficial effects of gene therapy and continued surveillance of potential adverse effects associated with the gene transfer procedures. No new subjects will be enrolled in this protocol.
Eligibility:
Study Type: Observational, Natural History
Minimum Age/Maximum Age: /
Genders: Both
Protocol Entry Criteria: New patients will not be treated under protocol 90-HG-0195 as new and improved vectors and technologies have become available in the recent years. New patients with ADA deficiency, however, may be enrolled in protocol 90-HG-0195 for clinical evaluation of their immune system and pre-treatment testing of transduction procedures.
Total Enrollment: 10
Location and Contact Information:
National Human Genome Research Institute (NHGRI)
Bethesda, Maryland, 20892
United States
Additional Information:
Study ID Numbers: 900195; 90-HG-0195
Study Start Date: September 4, 1990
Record last reviewed: July 22, 2002
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00001255
Other Severe Combined Immunodeficiency Studies:
1. Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies
2. Use of G-CSF to Obtain Blood Cell Precursors
3. Pilot Study of Allogeneic Bone Marrow Transplantation Plus Cyclosporine and Mycophenolate Mofetil to Induce Mixed Hematopoietic Chimerism in Patients With Primary T-Cell Immunodeficiency Disorders
4. Genetic Basis of Immunodeficiency
5. Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)
Related Studies:
Other Severe Combined Immunodeficiency Clinical Trials
Other Maryland Clinical Trials
Other Bethesda Clinical Trials
Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study
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