Ignite Creation Date:
2024-05-05 @ 10:22 AM
Last Modification Date:
2024-10-26 @ 9:02 AM
Study NCT ID:
NCT00001905
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
1999-11-03
Brief Title:
Interferon Gamma to Treat Leukocyte Adhesion Deficiency Type I
Sponsor:
National Institute of Allergy and Infectious Diseases NIAID
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Interferon Gamma Administration in Leukocyte Adhesion Deficiency Type I
Status:
COMPLETED
Status Verified Date:
2002-03
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 evaluate the safety and effectiveness of the drug interferon gamma in treating leukocyte adhesion deficiency type I LAD I Patients with this inherited immune disorder do not have enough proteins called adhesion molecules on their infection-fighting white blood cells impairing the ability of these cells to get to the site of infection As a result patients have recurrent infections of soft tissues such as the skin gums and gastrointestinal tract and poor wound healing Infants with severe LAD I often die from multiple infections Interferon gamma may increase the number of adhesion molecules on white blood cells and thus improve their function
Patients with LAD I who weigh more than 13 kilograms 285 pounds may be eligible for this study Candidates will have personal and family medical histories taken a physical examination blood and urine tests and a chest X-ray or computed tomography CT scan
Participants will receive injections of interferon gamma under the skin 3 times a week for 3 months Adult patients will be taught how to give their own injections similar to insulin injections for diabetes and parents will be taught how to administer the shots to their child Blood samples usually be between 30 to 90 milliliters 2 to 6 tablespoons will be drawn just before starting medication and again 1 day 1 week 1 month 3 months and 4 months after therapy begins At these same time intervals patients will provide a salt-water mouth rinse specimen which will be tested for changes in the number of white blood cells during interferon gamma treatment
Patients will be admitted to the NIH Clinical Center for inpatient evaluations at the start of therapy and again after 1 week 1month 3 months and 4 months The initial screening visit will take a few days and subsequent visits will take 1 to 2 days
Patients with LAD I who weigh more than 13 kilograms 285 pounds may be eligible for this study Candidates will have personal and family medical histories taken a physical examination blood and urine tests and a chest X-ray or computed tomography CT scan
Participants will receive injections of interferon gamma under the skin 3 times a week for 3 months Adult patients will be taught how to give their own injections similar to insulin injections for diabetes and parents will be taught how to administer the shots to their child Blood samples usually be between 30 to 90 milliliters 2 to 6 tablespoons will be drawn just before starting medication and again 1 day 1 week 1 month 3 months and 4 months after therapy begins At these same time intervals patients will provide a salt-water mouth rinse specimen which will be tested for changes in the number of white blood cells during interferon gamma treatment
Patients will be admitted to the NIH Clinical Center for inpatient evaluations at the start of therapy and again after 1 week 1month 3 months and 4 months The initial screening visit will take a few days and subsequent visits will take 1 to 2 days
Detailed Description:
Leukocyte adhesion deficiency type I LAD I is a primary immunodeficiency disease resulting from mutations in the gene encoding CD18 Markedly reduced or absent expression of the leukocyte integrin component CD18 causes significant impairment in leukocyte mobilization to inflammatory sites Clinically patients have marked leukocytosis and recurrent infections involving soft tissues such as skin the gastrointestinal tract and gingiva Death due to infections in early infancy is common with the severe form of LAD I CD18 expression less than 05 but patients with the moderate phenotype CD18 expression 1-10 may survive into young adulthood To date therapy consists of antibiotic treatment for infections and bone marrow transplantation when possible LAD I is also a candidate for future gene therapy Recently it has been shown that in vivo administration of interferon gamma IFN-gamma upregulates CD18 expression in normals and alters leukocyte trafficking We hypothesize that modest increases in CD18 expression in LAD I patients with the moderate phenotype or alterations in CD18 independent trafficking could result in detectable clinical changes and possible clinical improvement
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 |
|---|---|---|---|
| 99-I-0089 | None | None | None |