The Baltimore Polycystic Kidney Disease Clinical and Translational Core Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 350
- 试验地点
- 1
- 主要终点
- Renal volume by MRI
研究概览
简要总结
Advances in our understanding of the pathogenesis of autosomal dominant polycystic kidney disease (ADPKD) have opened up possibilities of new therapies to prevent disease progression. High quality clinical investigations in patients with ADPKD, however, pose significant challenges to investigators including limited access to patients with ADPKD,insufficient guidance by experienced investigators and lack of resources to conduct these studies.
The Polycystic Kidney Disease Research Clinical and Translational Core (P30) aims to establish an infrastructure that will assist investigators in designing and conducting highest quality clinical and translational research focused on a diverse group of patients with ADPKD.
Objective 1: To establish a Mid-Atlantic cohort of ADPKD patients (N=350) with baseline clinical phenotyping performed at the General Clinical Research Unit of the University of Maryland School of Medicine.
Objective 2: To establish a state-of-the-art biobank of specimens from the ADPKD cohort including serum, plasma,urine and DNA.
Objective 3: To develop a collaborative network of physicians and practices in the Mid-Atlantic region who will contribute to the ADPKD cohort and will be willing to refer patients for future studies and trials.
Objective 4: To establish a web-based registry of ADPKD patients in the Mid-Atlantic area.
详细描述
The purpose of this study is to establish a prospective observational cohort of 350 well-characterized adults with ADPKD, and an associated biorepository of DNA, plasma, serum, and urine. Baseline clinical phenotyping includes measurement of renal filtration function, total kidney volume, clinical and family history, presence and history of renal and extra-renal ADPKD manifestations, cardiac function, vascular stiffness, and health-related quality of life.
Prospective characterization will include the development of ADPKD complications (e.g., infection, stones, cyst hemorrhage) and other acute medical events, and changes in symptoms and QoL.
In addition, an electronic PKD patient registry will collect demographic and contact information on adults with ADPKD interested in participating in future clinical trials and/or observational cohort studies.
No treatment interventions will be performed in these observational studies
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- •End Stage Renal Disease or presently on dialysis or a prior kidney transplant
- •-Pregnant, lactating, or intention to get pregnant in next 6 weeks
- •Another systemic disease such as cancer or lupus
- •Life expectancy less than 2 years
- •Current participation in a drug treatment trial
- •Non English speaking
- •Uncontrolled diabetes A1C 7.0 or more within 6 months of study visit; and/or on more than one oral hypoglycemic agent
- •Diabetic nephropathy
结局指标
主要结局
Renal volume by MRI
时间窗: Baseline and 3 Year follow up measures to assess changes between the time points
Calculations of the volume will be based on summation of the products of the area measurements of the kidneys and/or liver and slice thickness. A region-based signal threshold method will be applied to calculate total cyst volume, and the remaining parenchymal renal and hepatic volume.
次要结局
- Quality of Life Instruments(Annually up to 3 Year follow up measures to assess changes between the time points)
研究者
Terry Watnick
Principal Investigator
University of Maryland, Baltimore