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| ID | Type | Description | Link |
|---|---|---|---|
| 2R01DK058793 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | NIH |
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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 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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Pravastatin | Experimental | Pravastatin |
|
| Placebo | Placebo Comparator | Placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| pravastatin | Drug | Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years) |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage Change in Total Kidney Volume Corrected for Height | 3 years | |
| Left Ventricular Mass Index | left ventricular mass index in g/m^2 by MRI | 3 years |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Melissa A Cadnapaphornchai, MD | University of Colorado, Denver | Principal Investigator |
| Robert W Schrier, MD | University of Colorado, Denver | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Colorado at Denver and Health Sciences Center | Denver | Colorado | 80262 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 16669974 | Background | Schrier RW. Optimal care of autosomal dominant polycystic kidney disease patients. Nephrology (Carlton). 2006 Apr;11(2):124-30. doi: 10.1111/j.1440-1797.2006.00535.x. | |
| 16221221 | Background | 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. doi: 10.1111/j.1523-1755.2005.00678.x. |
| Label | URL |
|---|---|
| PKD Foundation website | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | Pravastatin | Pravastatin pravastatin: Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years) |
| FG001 | Placebo | Placebo Placebo: Placebo daily |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
A total of 111 participants were screened with 110 enrolled in this clinical trial, including 56 randomized to pravastatin and 54 randomized to placebo.
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| ID | Title | Description |
|---|---|---|
| BG000 | Pravastatin | Pravastatin pravastatin: Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years) |
| BG001 | Placebo | Placebo Placebo: Placebo daily |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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 | Intention to treat | Posted | Number | percentage of participants | 3 years |
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Pravastatin | Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years) |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Melissa Cadnapaphornchai, MD | PKD Research Group, University of Colorado Anschutz Medical Campus | 3037241690 | pkd@ucdenver.edu |
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| ID | Term |
|---|---|
| D016891 | Polycystic Kidney, Autosomal Dominant |
| D052177 | Kidney Diseases, Cystic |
| D006973 | Hypertension |
| ID | Term |
|---|---|
| D007690 | Polycystic Kidney Diseases |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
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| ID | Term |
|---|---|
| D017035 | Pravastatin |
| ID | Term |
|---|---|
| D009281 | Naphthalenes |
| D011084 | Polycyclic Aromatic Hydrocarbons |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
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| Placebo | Drug | Placebo daily |
|
| Urinary Albumin Excretion | 3 years |
| 16129202 | Background | 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. doi: 10.1053/j.ajkd.2005.05.029. |
| 15837441 | Background | 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. doi: 10.1016/j.cct.2005.01.001. |
| 12046022 | Background | 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. doi: 10.1053/ajkd.2002.33379. |
| 15533729 | Background | 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. doi: 10.1016/j.amjhyper.2004.06.020. |
| 39356039 | Derived | St Pierre K, Cashmore BA, Bolignano D, Zoccali C, Ruospo M, Craig JC, Strippoli GF, Mallett AJ, Green SC, Tunnicliffe DJ. Interventions for preventing the progression of autosomal dominant polycystic kidney disease. Cochrane Database Syst Rev. 2024 Oct 2;10(10):CD010294. doi: 10.1002/14651858.CD010294.pub3. |
| Pregnancy |
|
| oncologic diagnosis |
|
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Total kidney volume corrected for height | Total kidney volume corrected for height (HtTKV, ml/m) | Mean | Standard Deviation | ml/m |
|
| Urinary albumin excretion | Urinary albumin excretion in mcg/min | Mean | Standard Deviation | mcg/min |
|
| Left ventricular mass index | Left ventricular mass index in g/m^2 by MRI | Mean | Standard Deviation | g/m^2 |
|
| Total cholesterol | Mean | Standard Deviation | mg/dL |
|
| LDL cholesterol | Mean | Standard Deviation | mg/dL |
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
| Secondary | Percentage Change in Total Kidney Volume Corrected for Height | ITT | Posted | Mean | Standard Deviation | Percent change | 3 years |
|
|
|
|
| Secondary | Left Ventricular Mass Index | left ventricular mass index in g/m^2 by MRI | Posted | Mean | Standard Deviation | g/m^2 | 3 years |
|
|
|
|
| Secondary | Urinary Albumin Excretion | Posted | Mean | Standard Deviation | mcg/min | 3 years |
|
|
|
|
| 0 |
| 56 |
| 0 |
| 56 |
| EG001 | Placebo | Placebo daily | 0 | 54 | 0 | 54 |
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| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000072661 | Ciliopathies |
| D030342 | Genetic Diseases, Inborn |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D011083 | Polycyclic Compounds |