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

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ClinicalTrials.gov Identifier: NCT00456365
Recruitment Status : Completed
First Posted : April 4, 2007
Results First Posted : March 9, 2018
Last Update Posted : March 9, 2018
Sponsor:
Collaborator:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Information provided by (Responsible Party):
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).

Condition or disease Intervention/treatment Phase
Polycystic Kidney, Autosomal Dominant Drug: pravastatin Drug: Placebo Phase 3

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 Type : Interventional  (Clinical Trial)
Actual Enrollment : 110 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Effect of Statin Therapy on Disease Progression in Autosomal Dominant Polycystic Kidney Disease
Study Start Date : November 2006
Actual Primary Completion Date : October 2012
Actual Study Completion Date : October 2012

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: Pravastatin
Pravastatin
Drug: pravastatin
Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years)
Placebo Comparator: Placebo
Placebo
Drug: Placebo
Placebo daily



Primary Outcome Measures :
  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 Outcome Measures :
  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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years to 22 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00456365


Locations
United States, Colorado
University of Colorado at Denver and Health Sciences Center
Denver, Colorado, United States, 80262
Sponsors and Collaborators
University of Colorado, Denver
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Investigators
Principal Investigator: Melissa A Cadnapaphornchai, MD University of Colorado, Denver
Principal Investigator: Robert W Schrier, MD University of Colorado, Denver

Additional Information:
Publications:

Responsible Party: University of Colorado, Denver
ClinicalTrials.gov Identifier: NCT00456365     History of Changes
Other Study ID Numbers: 05-0704
2R01DK058793 ( U.S. NIH Grant/Contract )
First Posted: April 4, 2007    Key Record Dates
Results First Posted: March 9, 2018
Last Update Posted: March 9, 2018
Last Verified: February 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by University of Colorado, Denver:
Autosomal dominant polycystic kidney disease
Cystic kidney disease
High blood pressure
Statin

Additional relevant MeSH terms:
Polycystic Kidney Diseases
Polycystic Kidney, Autosomal Dominant
Kidney Diseases
Disease Progression
Urologic Diseases
Disease Attributes
Pathologic Processes
Kidney Diseases, Cystic
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Pravastatin
Anticholesteremic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Enzyme Inhibitors
Lipid Regulating Agents