Novel Inflammatory Biomarkers Complement 5A and Hepcidin in Patients With Gaucher Disease (GD)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 34
- 试验地点
- 1
- 主要终点
- Change in Plasma Hepcidin Concentration
研究概览
简要总结
The objective of this study is to evaluate oxidative stress and/or inflammation in patients with Gaucher disease type I using a series of biomarkers and correlate with measurements of currently used diagnostic biomarkers.
详细描述
We will determine oxidative stress and/or inflammation related biomarkers in whole blood and/or plasma in adult subjects with Gaucher disease. Fifteen milliliter blood sample will be collected during three independent visits over a period of approximately 3 months. These samples will be processed to separate plasma from red blood cells and frozen until assays are performed. Standardized immunoassay methods and LC/MS based methods will be adopted to assay a series of biomarkers in these samples. These data will be correlated with currently used diagnostic biomarkers.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All participants must be 18 years or older.
- •All enrollees must understand and cooperate with requirements of the study in the opinion of the investigators and must be able to provide written informed consent.
- •Individuals with Gaucher disease who are medically stable for participation in study in the opinion of the investigator.
- •GD subjects must be stable on a specific ERT and/or SRT therapy at a specific dose (for e.g. on a units/kg basis) for at least 2 years or be naïve to these therapies (no therapy for 2 years).
- •GD1 patients, who have had a change in therapy i.e. a change in dose or switch from one drug to another, can be enrolled after at least 6 months have elapsed since the change and is considered stable in the opinion of the clinician providing care to the patient.
- •All participants must not have taken antioxidants coenzyme Q-10, vitamin C, or vitamin E for 3 weeks prior to the study.
排除标准
- •Medically unstable conditions in any group as determined by the investigators
- •Concurrent disease; medical condition; or an extenuating circumstance that, in the opinion of the investigator, might compromise subject safety, study compliance, completion of the study, or the integrity of the data collected for the study.
- •Females who are pregnant or lactating or of child-bearing age who are not using acceptable forms of contraception
- •History of asthma that is presently being treated
- •Subjects who cannot or are unwilling to have blood drawn
- •Unable to adhere to study protocol for whatever reason
研究组 & 干预措施
Treatment naive GD1
Type 1 Gaucher disease subjects who are naive to any treatment
Treated GD1
Type 1 Gaucher disease who are stable on therapy (on the specific ERT and/or SRT and specific dose for at least 2 years)
Healthy Volunteers (No longer recruiting)
Age matched healthy controls. No new participants will be enrolled to this arm.
结局指标
主要结局
Change in Plasma Hepcidin Concentration
时间窗: 6 months
Outcome will be reported as the change in plasma concentration of hepcidin (ng/ml) from baseline to 6 months in participants with Gaucher disease type 1
Change in Plasma C5a Concentration
时间窗: 6 months
Outcome will be reported as the change in plasma concentration of C5a (ng/ml) from baseline to 6 months in participants with Gaucher disease type 1
次要结局
- Change in Blood Glutathione Concentration(6 months)
- Change in Plasma Tumor Necrosis Factor (TNF)-alpha Concentration(6 months)