Ignite Creation Date:
2024-05-05 @ 11:22 AM
Last Modification Date:
2024-10-26 @ 9:02 AM
Study NCT ID:
NCT00001255
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
1999-11-03
Brief Title:
Gene Transfer Therapy for Severe Combined Immunodeficieny Disease SCID Due to Adenosine Deaminase ADA Deficiency A Natural History Study
Sponsor:
National Human Genome Research Institute NHGRI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Treatment of Severe Combined Immunodeficiency Disease SCID Due to Adenosine Deaminase ADA Deficiency With Autologous Lymphocytes of CD34 Cells Transduced With a Human ADA Gene A Natural History Study
Status:
COMPLETED
Status Verified Date:
2002-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 patients 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 patients 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 patients lymphocytes
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 patients 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 patients 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 patients lymphocytes
Detailed Description:
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
No new subjects will be enrolled in this protocol
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 90-HG-0195 | None | None | None |