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)...
Date First Received: March 12, 2007
Last Updated: December 17, 2007
Verified by: University of Colorado at Denver and Health Sciences Center, December 2007
Clinical Trial Phase: Phase 3 | Start Date: November 2006
Overall Status: Recruiting
Estimated Enrollment: 100
Brief Summary
Official Title: “Effect of Statin Therapy on Disease Progression in Autosomal Dominant Polycystic Kidney Disease”
Condition Keyword(s):
Intervention(s):
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).
Study Type: Interventional
Study Design: Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Parallel Assignment, Efficacy Study
Detailed Clinical Trial 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.
Intervention(s) in this Clinical Trial
- Drug: pravastatin
- Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years)
- Drug: Placebo
- Placebo daily
Arms, Groups and Cohorts in this Clinical Trial
- Experimental: 1
- Pravastatin
- Placebo Comparator: 2
- Placebo
Outcome Measures for this Clinical Trial
Primary Measures
- Total renal volume
- Time Frame: 3 years
Safety Issue?: No
- Time Frame: 3 years
- Left ventricular mass index
- Time Frame: 3 years
Safety Issue?: No
- Time Frame: 3 years
- Urinary albumin excretion
- Time Frame: 3 years
Safety Issue?: No
- Time Frame: 3 years
- Endothelial-dependent vasodilation
- Time Frame: 3 years
Safety Issue?: No
- Time Frame: 3 years
Secondary Measures
- Change in primary outcome measures with respect to LDL cholesterol
- Time Frame: 3 years
Safety Issue?: No
- Time Frame: 3 years
- Change in primary outcome measures with respect to blood pressure
- Time Frame: 3 years
Safety Issue?: No
- Time Frame: 3 years
Criteria for Participation in this Clinical Trial
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
Gender Eligibility for this Clinical Trial: Both
Minimum Age for this Clinical Trial: 8 Years
Maximum Age for this Clinical Trial: 21 Years
Are Healthy Volunteers Accepted for this Clinical Trial?: No
Clinical Trial Sponsor Information
Lead Sponsor: University of Colorado at Denver and Health Sciences Center
Overall Clinical Trial Officials and Contacts
Melissa A Cadnapaphornchai, MD Principal Investigator University of Colorado at Denver and Health Sciences Center
Overall Contact: Melissa A Cadnapaphornchai, MD 303-724-1690 pkd.nurse@uchsc.edu
Related Publications
References
Schrier RW. Optimal care of autosomal dominant polycystic kidney disease patients. Nephrology (Carlton). 2006 Apr;11(2):124-30. Review.
Abdollah Shamshirsaz A, 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].
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.
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.
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.
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.
Additional Information
Information obtained from ClinicalTrials.gov on November 20, 2008
Link to the current ClinicalTrials.gov record. http://clinicaltrials.gov/show/NCT00456365
Study ID Number: 2R01 DK058793-06-A1
ClinicalTrials.gov Identifier: NCT00456365
Health Authority: United States: Institutional Review Board
Clinical Trials Authorship and Review
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