Chronic Granulomatous Disease
Conditions
Brief summary
The overall goal of the study is to investigate the functional, biochemical, and gene expression effects of Interferon-gamma 1-b (IFN-γ) on the neutrophils of patients with Chronic Granulomatous Disease (CGD). The investigators hypothesize that the clinical effects demonstrated in patients with CGD treated with IFN-γ (decreased number and severity of infections) are the result of biochemical processes and upregulation of specific genes, which lead to enhanced functionality of this immune cell population.
Interventions
DRUGInterferon Gamma-1B
Study function and biochemistry of neutrophils off the drug for one week and then 12 hours after first dose and fourth dose.
Sponsors
University of Colorado, Denver
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
5 Years to 60 Years
Inclusion criteria
* Current treatment with IFN-γ or intent to treat with this drug * Acute infections resolved * Subject is off treatment medications * Women of childbearing potential must have: * a negative urine beta-HCG, and * be willing to practice two forms of effective contraception for the duration of study participation
Exclusion criteria
* Pregnancy, * Breast-feeding, or * Unwillingness to use effective contraception * Recent vaccination (within the last fourteen days) * Current infection or acute health condition requiring treatment * Chronic inflammatory complications of CGD including: * gastric outlet or urinary tract obstruction, * pleuritis, * pericarditis, * inflammatory bowel disease, or * colitis requiring treatment. * Un-associated chronic lung, liver, kidney, or cardiac disorders requiring active treatment
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Changes in Neutrophil bacterialcidal activity in patients with Chronic Granulomatous Disease (CGD) on standard treatment schedule and dose of IFN-γ in comparison to results for the patients off IFN-γ. | 1 week off drug, 12 hours after a week of treatment | Determine changes in neutrophil function in neutrophils from patients with CGD |
| Changes in Gene expression as measured with RNA and affimetrics gene chips in patients with Chronic Granulomatous Disease (CGD) on standard treatment schedule and dose of IFN-γ in comparison to results for the patients off IFN-γ. | 1 week off drug, 12 hours after a week of treatment | Determine changes in gene expression in neutrophils from patients with CGD. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Differences in Neutrophil biochemical studies in patients with Chronic Granulomatous Disease (CGD) on standard treatment schedule and dose of IFN-γ in comparison to results for the patients off IFN-γ. | 1 week off drug, 12 hours after first dose of IFN-gamma; 12 hours after a week of treatment | Determine changes in biochemical tests in neutrophils from CGD patients. |
Countries
United States
Outcome results
None listed