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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

March 12, 2007
December 12, 2012
November 2006
October 2012   (final data collection date for primary outcome measure)
  • 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 ]
  • Total renal volume
  • Left ventricular mass index
  • Urinary albumin excretion
  • Endothelial-dependent vasodilation
Complete list of historical versions of study NCT00456365 on ClinicalTrials.gov Archive Site
  • 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 ]
  • Change in primary outcome measures with respect to LDL cholesterol
  • Change in primary outcome measures with respect to blood pressure
Not Provided
Not Provided
 
Effect of Statin Therapy on Disease Progression in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Effect of Statin Therapy on Disease Progression in Autosomal Dominant Polycystic Kidney Disease

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).

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.

Interventional
Phase 3
Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Polycystic Kidney, Autosomal Dominant
  • Drug: pravastatin
    Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years)
  • Drug: Placebo
    Placebo daily
  • Experimental: 1
    Pravastatin
    Intervention: Drug: pravastatin
  • Placebo Comparator: 2
    Placebo
    Intervention: Drug: Placebo

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
112
October 2012
October 2012   (final data collection date for primary outcome measure)

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
Both
8 Years to 21 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00456365
05-0704, 2R01DK058793
Yes
University of Colorado, Denver
University of Colorado, Denver
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Principal Investigator: Melissa A Cadnapaphornchai, MD University of Colorado, Denver
Principal Investigator: Robert W Schrier, MD University of Colorado, Denver
University of Colorado, Denver
December 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP