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

This study has been completed.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
University of Colorado, Denver
ClinicalTrials.gov Identifier:
NCT00456365
First received: March 12, 2007
Last updated: December 12, 2012
Last verified: December 2012
  Purpose

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 Intervention Phase
Polycystic Kidney, Autosomal Dominant
Drug: pravastatin
Drug: Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Effect of Statin Therapy on Disease Progression in Autosomal Dominant Polycystic Kidney Disease

Resource links provided by NLM:


Further study details as provided by University of Colorado, Denver:

Primary Outcome Measures:
  • Total renal volume [ Time Frame: 3 years ] [ Designated as safety issue: No ]
  • Left ventricular mass index [ Time Frame: 3 years ] [ Designated as safety issue: No ]
  • Urinary albumin excretion [ Time Frame: 3 years ] [ Designated as safety issue: No ]
  • Endothelial-dependent vasodilation [ Time Frame: 3 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change in primary outcome measures with respect to LDL cholesterol [ Time Frame: 3 years ] [ Designated as safety issue: No ]
  • Change in primary outcome measures with respect to blood pressure [ Time Frame: 3 years ] [ Designated as safety issue: No ]

Enrollment: 112
Study Start Date: November 2006
Study Completion Date: October 2012
Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
Pravastatin
Drug: pravastatin
Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years)
Placebo Comparator: 2
Placebo
Drug: Placebo
Placebo daily

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 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.

  Eligibility

Ages Eligible for Study:   8 Years to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age 8-21 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: 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
Investigators
Principal Investigator: Melissa A Cadnapaphornchai, MD University of Colorado, Denver
Principal Investigator: Robert W Schrier, MD University of Colorado, Denver
  More Information

Additional Information:
Publications:

Responsible Party: University of Colorado, Denver
ClinicalTrials.gov Identifier: NCT00456365     History of Changes
Other Study ID Numbers: 05-0704, 2R01DK058793
Study First Received: March 12, 2007
Last Updated: December 12, 2012
Health Authority: United States: Institutional Review Board

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
Kidney Diseases, Cystic
Disease Attributes
Pathologic Processes
Pravastatin
Anticholesteremic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Lipid Regulating Agents
Therapeutic Uses
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Enzyme Inhibitors

ClinicalTrials.gov processed this record on April 16, 2014