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Polycystic Kidney, Autosomal DominantPhase 3NCT00456365

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

A Phase 3 study testing pravastatin in people with Polycystic Kidney, Autosomal Dominant, run by University of Colorado, Denver at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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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). Read more about this study

Who is it for?
Ages 8 to 22 years
Conditions the study is for
Polycystic Kidney, Autosomal Dominant
What is being tested?
pravastatin
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
110 participants
Where
1 siteUnited States
Study dates
Nov 2006to Oct 2012
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 8 to 22 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Age 8-22 years
  • Autosomal dominant polycystic kidney disease
  • Normal kidney function

Who cannot join

  • 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

Trial locations and contacts

1 site in 1 country.

United States1 site

  • University of Colorado at Denver and Health Sciences Center

    Denver, Colorado, 80262

Other trials in United States

About this study

Official title: 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).

Read the 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 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.

Being tested
pravastatin, Placebo
Keywords
Autosomal dominant polycystic kidney disease, Cystic kidney disease, High blood pressure, Statin
Medical subject headings
Polycystic Kidney, Autosomal Dominant, Kidney Diseases, Cystic, Hypertension

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • pravastatinDrug

    Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years)

  • PlaceboDrug

    Placebo daily

Study groups in NCT00456365
Study groupRoleWhat they receive
PravastatinExperimentalPravastatin
Receives: Drug: pravastatin
PlaceboPlacebo comparatorPlacebo
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

    Measured: 3 years

Secondary outcomes (3)

  1. Percentage Change in Total Kidney Volume Corrected for Height

    Measured: 3 years

  2. Left Ventricular Mass Index

    left ventricular mass index in g/m^2 by MRI

    Measured: 3 years

  3. Urinary Albumin Excretion

    Measured: 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Apr 2007 (estimated)
  2. Study startNov 2006
  3. Primary completionOct 2012
  4. Study completionOct 2012

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
110 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00456365
Sponsor's protocol ID
05-0704
Other IDs
2R01DK058793
Status last verified
Feb 2018
First submitted
12 Mar 2007
Last updated
9 Mar 2018
Data monitoring committee
Yes
Individual data sharing
No

Publications

  • 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.

    PubMed 16669974

  • 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.

    PubMed 16221221

  • 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.

    PubMed 16129202

  • 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.

    PubMed 15837441

  • 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.

    PubMed 12046022

  • 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.

    PubMed 15533729

  • 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.

    PubMed 39356039

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing pravastatin in people with Polycystic Kidney, Autosomal Dominant, run by University of Colorado, Denver at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 9 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 9 Mar 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percent of Participants Demonstrating 20% or More Increase in Total Kidney Volume (time frame: 3 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00456365, which also lists 7 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Mar 2018.

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