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

150 Participants Needed

Trial Summary

What is the purpose of this trial?

This study plans to learn if pravastatin is helpful in slowing down the progression of kidney disease in adults with autosomal dominant polycystic kidney disease (ADPKD). Pravastatin has been approved by the Food and Drug Administration (FDA) for adults for treatment of hyperlipidemia (high cholesterol levels). The investigators are using pravastatin in this study as an investigational drug for treatment of ADPKD.

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 data supports the effectiveness of the drug pravastatin for treating polycystic kidney disease?

Pravastatin is effective in lowering cholesterol levels, as it reduces LDL cholesterol by 15-40% and increases HDL cholesterol by 2-20%, which may indirectly benefit kidney health by improving overall cardiovascular health. ^{1 2 3 4 5}

Is pravastatin generally safe for humans?

Pravastatin is generally well tolerated with few adverse effects reported in clinical trials, making it a safe option for reducing cholesterol levels in humans. ^{1 6 7 8 9}

How does the drug pravastatin differ from other treatments for polycystic kidney disease?

Pravastatin is unique because it is primarily used to lower cholesterol by inhibiting an enzyme involved in cholesterol production, which may also have beneficial effects on kidney function. Unlike other treatments for polycystic kidney disease, pravastatin's potential benefits in this condition are being explored due to its effects on reducing cholesterol and possibly improving kidney health. ^{1 4 9 10 11}

Research Team

Michel Chonchol, MD

Principal Investigator

University of Colorado, Denver

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Pravastatin: a new drug for the treatment of hypercholesterolemia. [2013]

2. Germany pubmed.ncbi.nlm.nih.gov

Clinical pharmacology of pravastatin, a selective inhibitor of HMG-CoA reductase. [2018]

3. Israel pubmed.ncbi.nlm.nih.gov

Efficacy and safety of pravastatin once daily in primary moderate hypercholesterolemia: the Israeli experience. [2013]

4. United States pubmed.ncbi.nlm.nih.gov

The effect of pravastatin on plasma lipoprotein and apolipoprotein levels in primary hypercholesterolemia. The Southeastern Michigan Collaborative Group. [2013]

5. United States pubmed.ncbi.nlm.nih.gov

Comparison of the safety, tolerability, and pharmacokinetic profile of a single oral dose of pitavastatin 4 mg in adult subjects with severe renal impairment not on hemodialysis versus healthy adult subjects. [2012]

6. United States pubmed.ncbi.nlm.nih.gov

Pitavastatin calcium: clinical review of a new antihyperlipidemic medication. [2022]

7. United States pubmed.ncbi.nlm.nih.gov

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

8. New Zealand pubmed.ncbi.nlm.nih.gov

Formulation and Evaluation of Pravastatin Sodium-Loaded PLGA Nanoparticles: In vitro-in vivo Studies Assessment. [2023]

9. Germany pubmed.ncbi.nlm.nih.gov

The pharmacokinetics of pravastatin in patients on chronic hemodialysis. [2019]

10. England pubmed.ncbi.nlm.nih.gov

Randomized comparison of pitavastatin and pravastatin treatment on the reduction of urinary albumin in patients with type 2 diabetic nephropathy. [2022]

11. United States pubmed.ncbi.nlm.nih.gov

Effects of pravastatin on plasma lipid concentrations in poloxamer 407-induced hyperlipidemic rats. [2013]