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
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- 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.
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 5 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
结局指标
主要结局
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.
时间窗: 1 week off drug, 12 hours after a week of treatment
Determine changes in gene expression in neutrophils from patients with CGD.
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.
时间窗: 1 week off drug, 12 hours after a week of treatment
Determine changes in neutrophil function in neutrophils from patients with CGD
次要结局
- 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)