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

Polycystic Kidney, Autosomal Dominant Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00456365
• Other study ID #: 05-0704
• Secondary ID: 2R01DK058793
• Status: Completed
• Phase: Phase 3
• First received: March 12, 2007
• Last updated: February 8, 2018
• Start date: November 2006
• Est. completion date: October 2012

Study information

• Verified date: February 2018
• Source: University of Colorado, Denver
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 110
• Est. completion date: October 2012
• Est. primary completion date: October 2012
• Accepts healthy volunteers: No
• Gender: All
• Age group: 8 Years to 22 Years

Eligibility

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

Related Conditions & MeSH terms

• Disease Progression
• Kidney Diseases
• Polycystic Kidney Diseases
• Polycystic Kidney, Autosomal Dominant

Intervention

Drug:
• pravastatin
Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years)
• Placebo
Placebo daily

Locations

Country	Name	City	State
United States	University of Colorado at Denver and Health Sciences Center	Denver	Colorado

Sponsors (2)

Lead Sponsor	Collaborator
University of Colorado, Denver	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Country where clinical trial is conducted

United States, 

References & Publications (6)

Cadnapaphornchai MA, Fick-Brosnahan GM, Duley I, Johnson AM, Strain JD, DeGroff CG, Schrier RW. Design and baseline characteristics of participants in the study of antihypertensive therapy in children and adolescents with autosomal dominant polycystic kidney disease (ADPKD). Contemp Clin Trials. 2005 Apr;26(2):211-22. — View Citation

Fick-Brosnahan GM, Belz MM, McFann KK, Johnson AM, Schrier RW. Relationship between renal volume growth and renal function in autosomal dominant polycystic kidney disease: a longitudinal study. Am J Kidney Dis. 2002 Jun;39(6):1127-34. — View Citation

Kelleher CL, McFann KK, Johnson AM, Schrier RW. Characteristics of hypertension in young adults with autosomal dominant polycystic kidney disease compared with the general U.S. population. Am J Hypertens. 2004 Nov;17(11 Pt 1):1029-34. — View Citation

Schrier RW. Optimal care of autosomal dominant polycystic kidney disease patients. Nephrology (Carlton). 2006 Apr;11(2):124-30. Review. — View Citation

Shamshirsaz AA, Reza Bekheirnia M, Kamgar M, Johnson AM, McFann K, Cadnapaphornchai M, Nobakhthaghighi N, Schrier RW. Autosomal-dominant polycystic kidney disease in infancy and childhood: progression and outcome. Kidney Int. 2005 Nov;68(5):2218-24. Erratum in: Kidney Int. 2005 Dec;68(6):2936. Shamshirsaz, Abdollah [corrected to Abdollah Shamshirsaz, Alireza]; Bekheirnia, Reza M [corrected to Reza Bekheirnia, Mir]; Haghighi, NN [corrected to Nobakhthaghighi, Niloofar]. — View Citation

Taylor M, Johnson AM, Tison M, Fain P, Schrier RW. Earlier diagnosis of autosomal dominant polycystic kidney disease: importance of family history and implications for cardiovascular and renal complications. Am J Kidney Dis. 2005 Sep;46(3):415-23. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percent of Participants Demonstrating 20% or More Increase in Total Kidney Volume	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	3 years
Secondary	Percentage Change in Total Kidney Volume Corrected for Height		3 years
Secondary	Left Ventricular Mass Index	left ventricular mass index in g/m^2 by MRI	3 years
Secondary	Urinary Albumin Excretion		3 years

External Links

•   PKD Foundation website