Drug Development in Autosomal Dominant Polycystic Kidney Disease: Opportunities and Challenges.

Autosomal dominant polycystic kidney disease (ADPKD) is a hereditary disorder characterized by relentless growth of innumerable renal cysts bilaterally, associated with decline in glomerular filtration rate over the course of decades. The burden of ADPKD and its treatment is associated with a significant economic and societal cost. Despite several clinical studies conducted over the past decade, only one treatment has been approved by regulatory agencies to slow disease progression in ADPKD.

Autosomal dominant polycystic kidney disease.

Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary kidney disease and one of the most common causes of end-stage kidney disease. Multiple clinical manifestations, such as enlarged kidneys filled with growing cysts, hypertension, and multiple extrarenal complications, including liver cysts, intracranial aneurysms, and cardiac valvular disease, show that ADPKD is a systemic disorder. New information derived from clinical research using molecular genetics and advanced imaging techniques has provided enhanced tools for assessing the diagnosis and prognosis for individual patients and their families.

Addressing the Need for Clinical Trial End Points in Autosomal Dominant Polycystic Kidney Disease: A Report From the Polycystic Kidney Disease Outcomes Consortium (PKDOC).

Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary kidney disease. Expansion of multiple cysts throughout both kidneys is thought to lead to progressive loss of kidney function and kidney failure in some patients. In recent years, much has been learned about the pathophysiology of ADPKD.

Low-Osmolar Diet and Adjusted Water Intake for Vasopressin Reduction in Autosomal Dominant Polycystic Kidney Disease: A Pilot Randomized Controlled Trial.

Background: Autosomal dominant polycystic kidney disease (ADPKD) affects millions of people worldwide. Vasopressin promotes disease progression.

Study Design: A randomized controlled trial with equal (1:1) allocation.

Therapeutic Area Data Standards for Autosomal Dominant Polycystic Kidney Disease: A Report From the Polycystic Kidney Disease Outcomes Consortium (PKDOC).

Data standards provide a structure for consistent understanding and exchange of data and enable the integration of data across studies for integrated analysis. There is no data standard applicable to kidney disease. We describe the process for development of the first-ever Clinical Data Interchange Standards Consortium (CDISC) data standard for autosomal dominant polycystic kidney disease (ADPKD) by the Polycystic Kidney Disease Outcomes Consortium (PKDOC).

Patient-reported outcomes in clinical trials of CKD-related therapies: report of a symposium sponsored by the national kidney foundation and the U.S. Food and Drug Administration.

The National Kidney Foundation and the U.S. Food and Drug Administration (FDA) convened a symposium in September 2010, bringing together more than 70 experts, including representatives from the FDA, the National Institutes of Health, the Critical Path Institute, nephrologists, patients, and the pharmaceutical industry to discuss the feasibility and process of developing patient-reported outcome (PRO) measures to access how patients feel or function to be used in clinical trials for regulatory review of treatment benefit.