Role of Interferon-gamma 1-b (IFN-γ) on Cells of the Innate Immune System: Functional, Biochemical and Gene Expression Studies in Patients With Chronic Granulomatous Disease

Study Overview
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Brief Summary
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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.

Official Title
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Role of Interferon-gamma 1-b (IFN-γ) on Cells of the Innate Immune System: Functional, Biochemical and Gene Expression Studies in Patients With Chronic Granulomatous Disease

Conditions
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Chronic Granulomatous Disease

Intervention / Treatment
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* Drug: Interferon Gamma-1B


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 5 Years
Maximum Age: 60 Years

Sexes Eligible for Study
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All

Study Plan
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How is the study designed?
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Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Interferon Gamma-1B|Study function and biochemistry of neutrophils off the drug for one week and then 12 hours after first dose and fourth dose.|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | Determine changes in neutrophil function in neutrophils from patients with CGD | 1 week off drug, 12 hours after a week of treatment | 
| 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. | Determine changes in gene expression in neutrophils from patients with CGD. | 1 week off drug, 12 hours after a week of treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | Determine changes in biochemical tests in neutrophils from CGD patients. | 1 week off drug, 12 hours after first dose of IFN-gamma; 12 hours after a week of treatment |