PKD Clinical and Translational Core Study
- Conditions
- Polycystic Kidney Disease
- Registration Number
- NCT01873235
- Lead Sponsor
- University of Maryland, Baltimore
- Brief Summary
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.
- Detailed Description
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
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 350
Not provided
-
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
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Renal volume by MRI 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.
- Secondary Outcome Measures
Name Time Method Quality of Life Instruments Annually up to 3 Year follow up measures to assess changes between the time points Self-reported Quality of Life (pain, anxiety, depression, physical activity, fatigue).
Trial Locations
- Locations (1)
University of Maryland School of Medicine General Clinical Research Center
🇺🇸Baltimore, Maryland, United States