Statin Therapy for Polycystic Kidney Disease

Overseen By Diana George

Age: 18 - 65

Sex: Any

Trial Phase: Phase 4

Sponsor: University of Colorado, Denver

Must not be taking: Tolvaptan, Fibrates, Niacin, others

Disqualifiers: Diabetes, Hypertension, Renal disease, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 2 years

What You Need to Know Before You Apply

What is the purpose of this trial?

The trial aims to determine if pravastatin, a drug typically used to lower cholesterol, can slow kidney disease in individuals with autosomal dominant polycystic kidney disease (ADPKD). Participants will receive either pravastatin or a placebo (a pill with no active medicine) to assess pravastatin's effectiveness for this condition. Suitable candidates should have ADPKD, a total kidney volume over 500 mL, and well-controlled blood pressure. As a Phase 4 trial, this research seeks to understand how this FDA-approved treatment can benefit more patients.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications, including tolvaptan, gemfibrozil, other fibrates, niacin, clarithromycin, or cyclosporine. If you are on any of these, you would need to discontinue them to participate.

What is the safety track record for pravastatin?

Research shows that pravastatin, a medication approved for treating high cholesterol, is under study for potential benefits in polycystic kidney disease (ADPKD). Studies have found that adults usually tolerate pravastatin well. It is widely used and known to be safe for managing cholesterol levels. While specific evidence does not yet confirm pravastatin's effect on kidney health in adults with ADPKD, it has reduced inflammation in younger patients. This suggests it could be safe and might benefit adults with ADPKD. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Pravastatin is unique because it is primarily a cholesterol-lowering medication, but researchers are exploring its potential to slow the progression of polycystic kidney disease (PKD). Unlike the standard treatments for PKD, which mainly aim to manage symptoms or delay the need for dialysis, pravastatin may offer a new way to target the underlying disease process. Researchers are excited about pravastatin because it could alter the course of PKD by reducing inflammation and slowing cyst growth, providing a new hope for patients beyond the current options.

What evidence suggests that pravastatin might be an effective treatment for ADPKD?

This trial will compare pravastatin with a placebo to evaluate its effects on adults with autosomal dominant polycystic kidney disease (ADPKD). Research has shown that pravastatin does not significantly help adults with ADPKD in slowing kidney growth or improving kidney function. Several studies found no major differences between those taking pravastatin and those taking a placebo. However, earlier research indicated that pravastatin was effective in slowing kidney disease progression in younger people with ADPKD. While pravastatin is approved for lowering cholesterol, its effect on ADPKD in adults remains unclear. Therefore, the potential benefit of pravastatin for kidney disease in adults with ADPKD remains unproven based on current evidence. ^{1 3 5 6 7}

Who Is on the Research Team?

Michel Chonchol, MD

Principal Investigator

University of Colorado, Denver

Are You a Good Fit for This Trial?

This trial is for adults with early stage autosomal dominant polycystic kidney disease (ADPKD). Participants should have a kidney volume over 500 mL, good kidney function (GFR ≥60), and controlled blood pressure. It's not for those recently hospitalized, pregnant or breastfeeding women, tobacco users, people with alcohol issues, uncontrolled high blood pressure, diabetes, certain cholesterol disorders or sensitivities to statins.

Inclusion Criteria

Your kidney function is not too low.

I have been diagnosed with polycystic kidney disease.

My kidneys are larger than normal.

Exclusion Criteria

You cannot have an MRI due to having certain implants, a pacemaker, or feeling very anxious in small spaces.

Pregnant or breast feeding

You currently smoke or use tobacco products.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 40 mg tablets of pravastatin or placebo every day for 6 weeks, followed by continuation for 2 years if well tolerated

2 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Placebo
• Pravastatin

Trial Overview

The study is testing if pravastatin can slow down the progression of ADPKD compared to a placebo. Pravastatin is usually used for high cholesterol but here it's being investigated specifically for ADPKD. Patients will be randomly assigned to either receive pravastatin or a placebo.

How Is the Trial Designed?

Group I: Pravastatin Active Control 1 Intervention

Participants will receive 40 mg tablets of pravastatin everyday for 6 weeks. If well tolerated, participants will continue taking 40 mg dose of pravastatin everyday for 2 years.

Group II: Placebo Placebo Group 1 Intervention

Participants will receive inactive 40 mg tablets of placebo everyday for 6 weeks. If well tolerated, participants will continue taking inactive 40 mg dose of placebo everyday for 2 years.

Who Is Running the Clinical Trial?

University of Colorado, Denver

Lead Sponsor

Trials

1,842

Recruited

3,028,000+

Published Research Related to This Trial

Pravastatin is an effective HMG-CoA reductase inhibitor that significantly lowers total cholesterol by 15-30% and LDL cholesterol by 15-40% in patients with hypercholesterolemia, including those with diabetes, when taken in doses of 10-40 mg/day.

It is generally well tolerated with fewer reported adverse effects compared to similar medications like lovastatin, suggesting it may be a safer option for lipid-lowering therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pravastatin: a new drug for the treatment of hypercholesterolemia. Jungnickel, PW., Cantral, KA., Maloley, PA.[2013]

Pitavastatin is effective in reducing low-density lipoprotein (LDL) levels similarly to other statins like simvastatin and atorvastatin, making it a viable option for treating hypercholesterolemia and combined dyslipidemia.

Despite its efficacy, pitavastatin has not demonstrated improved clinical outcomes in high-risk patients compared to cheaper alternatives, and its higher cost may limit its use in practice.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pitavastatin: a new 3-hydroxy-3-methylglutaryl coenzyme a reductase inhibitor for the treatment of hyperlipidemia. Baker, WL., Datta, R.[2012]

Citations

1.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC4011448/

Effect of Pravastatin on Total Kidney Volume, Left ...

Conclusions. Pravastatin is an effective agent to slow progression of structural kidney disease in children and young adults with ADPKD. These findings support ...

2.

kidney-international.org

kidney-international.org/article/S0085-2538(25)00780-X/abstract

A randomized controlled trial evaluated the effect of ...

Among adult patients with ADPKD and preserved kidney function, pravastatin did not slow the increase in HtTKV and had no effect on slowing the ...

3.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/41077128/

A randomized controlled trial evaluated the effect of ...

Conclusions: Among adult patients with ADPKD and preserved kidney function, pravastatin did not slow the increase in HtTKV and had no effect on ...

4.

sciencedirect.com

sciencedirect.com/science/article/pii/S1551714423003464

Statin therapy in patients with early-stage autosomal ...

However, there is no evidence that pravastatin therapy may impact kidney outcomes in adult patients with ADPKD. Indeed, a post-hoc analysis of data from the ...

5.

docwirenews.com

docwirenews.com/post/effects-of-pravastatin-on-kidney-outcomes-in-adults-with-adpkd

Effects of Pravastatin on Kidney Outcomes in Adults With ...

No significant differences were observed in HtTKV changes, kidney function, or renal blood flow between patients in the pravastatin and placebo ...

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04284657

Pravastatin and Alkali Therapy in Patients With Autosomal ...

Cholesterol-lowering agents called statins have shown promise in the treatment of younger ADPKD patients, reducing inflammation and progression as assessed by ...

7.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC9459055/

A Systematic Review of Reported Outcomes in ADPKD Studies

Most ADPKD studies report outcomes concerning kidney function, change in total kidney volume (TKV), change in creatinine clearance, and the development of ESKD.