Clinical trial NCT01873235: PKD Clinical and Translational Core Study
Official title: The Baltimore Polycystic Kidney Disease Clinical and Translational Core Study
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Polycystic Kidney Disease
Lead sponsor: University of Maryland, Baltimore
Sponsor class: OTHER
Collaborators: name: National Institutes of Health (NIH)
name: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
First posted: 2013-06-10
Enrollment: 350
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adults with diagnosed Polycystic Kidney Disease

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

Eligibility criteria: Inclusion criteria:

* Age 18 and older
* ADPKD confirmed by genetic testing or ultrasound criteria using modified Ravine criteria: with family history:several cysts per kidney (3 by sonography, 5 if by computerized tomography or MRI)without family history: 10 cysts (by any radiologic method) per kidney and exclusion of other cystic kidney diseases
* Ability to provide written informed consent prior to initiation of any study procedures and the ability in the opinion of the investigator to comply with all requirements of the study
* Glomerular Filtration Rate (GFR) greater than 15ml/min/1.73m2

Exclusion Criteria:

* 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

Primary outcomes:
measure: Renal volume by MRI
timeFrame: Baseline and 3 Year follow up measures to assess changes between the time points
Secondary outcomes:
measure: Quality of Life Instruments
timeFrame: Annually up to 3 Year follow up measures to assess changes between the time points
Other outcomes:
measure: Hospitalizations
timeFrame: Annually up to 8 Year follow up to assess changes between time points
Locations:
city: Baltimore
country: United States
facility: University of Maryland School of Medicine General Clinical Research Center
state: Maryland
Study officials:
affiliation: University of Maryland, Baltimore
name: Terry J Watnick, MD
role: PRINCIPAL_INVESTIGATOR
References: