Ignite Creation Date:
2024-05-05 @ 11:19 AM
Last Modification Date:
2024-10-26 @ 9:02 AM
Study NCT ID:
NCT00001244
Status:
RECRUITING
Last Update Posted:
2024-07-08
First Post:
1999-11-03
Brief Title:
Immune Regulation in Patients With Common Variable Immunodeficiency and Related Syndromes
Sponsor:
National Institute of Allergy and Infectious Diseases NIAID
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Studies of Immune Regulation in Patients With Common Variable Immunodeficiency and Related Humoral Immunodeficiency Syndromes
Status:
RECRUITING
Status Verified Date:
2024-10-01
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 explore the cause of immunodeficiency in common variable immunodeficiency CVI and other related immunodeficiency syndromes-IgA deficiency hyper IgM syndrome thymoma and agammaglobulinemia hypogammaglobulinemia associated with Epstein-Barr infection and others to better focus on how to correct the underlying defect
Patients with CVI and their family members may participate in this study Family members must be between the ages of 18 and 85 in good health and weigh at least 110 pounds
Patients will receive standard medical care for their illness Procedures may include a medical history and physical examination routine blood tests stool examination for infectious agents lung function tests chest and sinus X-rays Treatment may include administration of immune serum globulin antibiotics for infections and anti-inflammatory drugs if needed In addition patients may undergo the following
Lymphapheresis This procedure is done to collect large numbers of white blood cells lymphocytes Blood is collected through a needle in an arm vein similar to donating blood The blood is separated it into its components by centrifugation spinning the white cells are removed and the rest of the blood red cells plasma and platelets is returned to the body either through the same needle or through another needle in the other arm
Blood draw Blood may be drawn through a needle in an arm vein venipuncture No more than 450 milliliters 15 ounces of blood will be collected over a 6-week period from adults and no more than 7 ml 1 12 teaspoons per kilogram 22 pounds of body weight in children over the same time period
Lymph node biopsies Lymph node biopsies will be done only if required for diagnostic purposes Some of the biopsy tissue may be kept for research Up to two lymph nodes may be removed during each procedure For the procedure a painkiller is injected into and beneath the skin in the biopsy area and the node is removed surgically The incision is closed using dissolving sutures stitches that do not require removal The biopsy takes about 30 minutes Patients will be hospitalized at least overnight for observation
Intestinal biopsies Endoscopy and gastrointestinal biopsy will be done only if there is evidence of malabsorption Some of the biopsy tissue may be kept for research Patients are pre-medicated to allay anxiety but are fully conscious during the procedure A flexible tube is inserted into the stomach or small intestine through the mouth The tube allows the doctor to see the intestinal mucosa and to project the image onto a TV screen At various places in the mucosal surface small pieces of tissue are plucked out using a small space at the tip of the endoscope The procedure takes 30 to 60 minutes
Some of the blood collected may be used for genetic tests Some blood and tissue samples may be stored for future research labeled with a code such as a number that only the study team can link to the patient
Participating family members will provide a medical history and their pulse blood pressure and temperature will be taken They will have 10 to 120 ml 13 to 4 ounces of blood drawn from a vein in the arm Blood samples may be taken on repeated occasions as long as the relative remains in the study The blood will be used for research that may involve development of diagnostic tests for CVI evaluation of the structure and function of normal blood cells for comparison with those of patients with CVI and studies to try to determine possible genetic factors involved in susceptibility to CVI
Patients with CVI and their family members may participate in this study Family members must be between the ages of 18 and 85 in good health and weigh at least 110 pounds
Patients will receive standard medical care for their illness Procedures may include a medical history and physical examination routine blood tests stool examination for infectious agents lung function tests chest and sinus X-rays Treatment may include administration of immune serum globulin antibiotics for infections and anti-inflammatory drugs if needed In addition patients may undergo the following
Lymphapheresis This procedure is done to collect large numbers of white blood cells lymphocytes Blood is collected through a needle in an arm vein similar to donating blood The blood is separated it into its components by centrifugation spinning the white cells are removed and the rest of the blood red cells plasma and platelets is returned to the body either through the same needle or through another needle in the other arm
Blood draw Blood may be drawn through a needle in an arm vein venipuncture No more than 450 milliliters 15 ounces of blood will be collected over a 6-week period from adults and no more than 7 ml 1 12 teaspoons per kilogram 22 pounds of body weight in children over the same time period
Lymph node biopsies Lymph node biopsies will be done only if required for diagnostic purposes Some of the biopsy tissue may be kept for research Up to two lymph nodes may be removed during each procedure For the procedure a painkiller is injected into and beneath the skin in the biopsy area and the node is removed surgically The incision is closed using dissolving sutures stitches that do not require removal The biopsy takes about 30 minutes Patients will be hospitalized at least overnight for observation
Intestinal biopsies Endoscopy and gastrointestinal biopsy will be done only if there is evidence of malabsorption Some of the biopsy tissue may be kept for research Patients are pre-medicated to allay anxiety but are fully conscious during the procedure A flexible tube is inserted into the stomach or small intestine through the mouth The tube allows the doctor to see the intestinal mucosa and to project the image onto a TV screen At various places in the mucosal surface small pieces of tissue are plucked out using a small space at the tip of the endoscope The procedure takes 30 to 60 minutes
Some of the blood collected may be used for genetic tests Some blood and tissue samples may be stored for future research labeled with a code such as a number that only the study team can link to the patient
Participating family members will provide a medical history and their pulse blood pressure and temperature will be taken They will have 10 to 120 ml 13 to 4 ounces of blood drawn from a vein in the arm Blood samples may be taken on repeated occasions as long as the relative remains in the study The blood will be used for research that may involve development of diagnostic tests for CVI evaluation of the structure and function of normal blood cells for comparison with those of patients with CVI and studies to try to determine possible genetic factors involved in susceptibility to CVI
Detailed Description:
The purpose of this protocol is to carry out laboratory studies concerning the immunopathogenesis of Common Variable Immunodeficiency CVI and related primary humoral immunodeficiency diseases Additionally we aim to document and track the progression of known complications of this primary immunodeficiency Complications associated with CVID include recurrent respiratory and gastrointestinal bacterial infections pulmonary insufficiency nodular regenerative hyperplasia lymphoid malignancy and various autoimmune manifestations
Patients with CVI and related B-Cell immunodeficiencies will be enrolled into this natural history study Protocol procedures will include baseline measurements of and changes in lab studies First- or second-degree genetically related family members limited to mother father siblings grandparents children aunts uncles and first cousins of an affected patient may also be screened for clinical in vitro and genetic correlates of immune abnormalities Changes in the patients clinical state will be measured to determine the precursors of disease complications This may lead to developments in improving preventive measures and novel treatment options for this population
Patients with CVI and related B-Cell immunodeficiencies will be enrolled into this natural history study Protocol procedures will include baseline measurements of and changes in lab studies First- or second-degree genetically related family members limited to mother father siblings grandparents children aunts uncles and first cousins of an affected patient may also be screened for clinical in vitro and genetic correlates of immune abnormalities Changes in the patients clinical state will be measured to determine the precursors of disease complications This may lead to developments in improving preventive measures and novel treatment options for this population
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
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 |
|---|---|---|---|
| 89-I-0158 | None | None | None |