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Completed

NCT Number: NCT00456365

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

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).

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Key information

Conditions

Polycystic Kidney, Autosomal Dominant Abnormalities, Multiple Cardiovascular Diseases Ciliopathies Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Hypertension Kidney Diseases Kidney Diseases, Cystic Male Urogenital Diseases Polycystic Kidney Diseases Urogenital Diseases Urologic Diseases Vascular Diseases

Age range

8 year–22 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

University of Colorado at Denver and Health Sciences Center

Denver, Colorado, 80262, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Pravastatin

Drug

Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years)

Placebo

Drug

Placebo daily

Primary outcomes

  1. Percent of Participants Demonstrating 20% or More Increase in Total Kidney Volume

    Time frame: 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 outcomes

  1. Percentage Change in Total Kidney Volume Corrected for Height

    Time frame: 3 years

  2. Left Ventricular Mass Index

    Time frame: 3 years

    left ventricular mass index in g/m^2 by MRI

  3. Urinary Albumin Excretion

    Time frame: 3 years

Sponsors and collaborators

Lead sponsor

University of Colorado, Denver

Other

Collaborators

  • National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Registry information

Official study title

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

Important dates

Study start
2006
Primary completion
2012
Study completion
2012
First posted
Apr 4, 2007
Registry last updated
Mar 9, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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