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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Effect of Statin Therapy on Disease Progression in Autosomal Dominant Polycystic Kidney Disease

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 Summary:

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 is 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. We 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 100 subjects will be enrolled in this research study. This study will involve pediatric subjects because we believe that early intervention is critical if we 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.


Sponsor: University of Colorado, Denver

Current Primary Outcome:

  • Total renal volume [ Time Frame: 3 years ]
  • Left ventricular mass index [ Time Frame: 3 years ]
  • Urinary albumin excretion [ Time Frame: 3 years ]
  • Endothelial-dependent vasodilation [ Time Frame: 3 years ]


Original Primary Outcome:

  • Total renal volume
  • Left ventricular mass index
  • Urinary albumin excretion
  • Endothelial-dependent vasodilation


Current Secondary Outcome:

  • Change in primary outcome measures with respect to LDL cholesterol [ Time Frame: 3 years ]
  • Change in primary outcome measures with respect to blood pressure [ Time Frame: 3 years ]


Original Secondary Outcome:

  • Change in primary outcome measures with respect to LDL cholesterol
  • Change in primary outcome measures with respect to blood pressure


Information By: University of Colorado, Denver

Dates:
Date Received: March 12, 2007
Date Started: November 2006
Date Completion:
Last Updated: December 12, 2012
Last Verified: December 2012