Effect of Statin Therapy on Disease Progression in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Phase 3

Completed

• Conditions: Polycystic Kidney, Autosomal Dominant
• Interventions: Drug: pravastatin; Drug: Placebo

• Registration Number: NCT00456365
• Lead Sponsor: University of Colorado, Denver

Brief Summary

The purpose of this study is to determine whether the medication pravastatin will ameliorate renal and cardiovascular disease over a 3-year period in children and young adults with autosomal dominant polycystic kidney disease (ADPKD).

Detailed Description

Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary kidney disease, affecting 1 in 400 to 1000 individuals and accounting for 4% of end-stage renal disease in the United States and 8-10% in Europe. The condition is characterized by progressive development of kidney cysts with kidney enlargement and associated loss of kidney function. High blood pressure and cardiovascular disease are common in patients with ADPKD. Although the condition is often thought to affect primarily adults, it is clear that the disease can be present in the fetus and young children.

This study was designed to determine if treatment with the medicine pravastatin can slow the progression of kidney and heart disease when initiated early in life in patients with ADPKD. The Investigators will assess differences between pravastatin and placebo study groups over the three-year study period with respect to: 1) total kidney volume as assessed by magnetic resonance imaging (MRI); 2) left ventricular mass index as assessed by MRI; 3) urinary albumin excretion; and 4) endothelial-dependent vasodilation as assessed by brachial ultrasound. A total of 110 subjects were enrolled in this research study. This study involved pediatric subjects because the Investigators believe that early intervention is critical if they are to decrease the morbidity and mortality associated with this condition. If pravastatin is shown to be effective in ameliorating progression of renal and cardiovascular disease in this study, routine management of people with this condition will be drastically altered.

Study Timeline

• Study Start: 2006-11
• Study First Submitted: 2007-03-12
• Study First Submitted QC: 2007-04-02
• Study First Posted: 2007-04-04
• Primary Completion: 2012-10
• Study Completion: 2012-10
• Results First Submitted: 2014-05-27
• Status Verified: 2018-02
• Results First Submitted QC: 2018-02-08
• Last Update Submitted: 2018-02-08
• Results First Posted: 2018-03-09
• Last Update Posted: 2018-03-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 110

Inclusion Criteria

• Age 8-22 years
• Autosomal dominant polycystic kidney disease
• Normal kidney function

Exclusion Criteria

• Abnormal kidney function
• Past allergic history to medications used in study
• Liver disease
• Muscle disease/dystrophy
• Pregnancy, planned pregnancy, or lactation within study period
• Inability to cooperate with or clinical contraindication for magnetic resonance imaging

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Pravastatin	pravastatin	Pravastatin
Placebo	Placebo	Placebo

Primary Outcome Measures

Name	Time	Method
Percent of Participants Demonstrating 20% or More Increase in Total Kidney Volume	3 years	Percent of participants demonstrating 20% or more increase in total kidney volume corrected for height, left ventricular mass index, or urinary albumin excretion over the three year study period

Secondary Outcome Measures

Name	Time	Method
Percentage Change in Total Kidney Volume Corrected for Height	3 years
Left Ventricular Mass Index	3 years	left ventricular mass index in g/m\^2 by MRI
Urinary Albumin Excretion	3 years

Trial Locations

Locations (1)

University of Colorado at Denver and Health Sciences Center; 🇺🇸 Denver, Colorado, United States