Statin Therapy in Patients With Early Stage ADPKD

NCT03273413 | Active Not Recruiting Phase 4 DMC FDA Drug

• 1 other identifier: 17-0678
• Study Type: interventional
• Target: 150
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

ADPKD; Autosomal Dominant Polycystic Kidney

Keywords

Pravastatin; Statin

Outcome Measures

Primary Outcomes (1)

• Change in Total Kidney Volume

  Total kidney volume as assessed by renal MRI, at baseline and after 2 years of treatment

  Baseline, 2 years

Secondary Outcomes (5)

• Change in Renal Blood Flow

  Baseline, 2 years

• Change in Kidney Function

  Baseline, 2 years

• Change in Circulating Inflammatory Markers

  Baseline, 2 years

• Change in Circulating Markers of Oxidative Stress

  Baseline, 2 years

• Change in Urinary Epithelial Cells

  Baseline, 2 years

Other Outcomes (1)

• Change in Blood Vessel Stiffness

  Baseline, 2 years

Study Arms (2)

Placebo

PLACEBO COMPARATOR

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.

Drug: Placebo

Pravastatin

ACTIVE COMPARATOR

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.

Drug: Pravastatin

Interventions

Pravastatin DRUG

Anti-inflammatory, anti-oxidative stress, and anti-proliferative therapy

Pravastatin

Placebo DRUG

Inactive tablet

Placebo

Eligibility Criteria

• Age: 25 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Diagnosis of ADPKD
• Total kidney volume \>500 mL
• Estimated glomerular filtration rate (GFR) ≥60 mL/min/1.73m\^2
• Controlled blood pressure \<140/80 mmHg

You may not qualify if:

• Uncontrolled hypertension
• Diabetes mellitus
• Renal disease, renal cancer, single kidney, recent renal surgery, or acute kidney injury
• Unstable angina
• Coronary artery disease
• Prior ischemic stroke
• Other clinical indication for a statin
• History of hospitalizations within the last 3 months
• Hepatic impairment or liver function abnormalities
• Secondary hypercholesterolemia or hypocholesterolemia
• Use of tolvaptan, gemfibrozil, other fibrates, niacin, clarithromycin, or cyclosporine
• Hypersensitivity to statins
• Immunosuppressive therapy within the last year
• Clinical contraindication for an MRI (i.e. implants, pacemaker, claustrophobia)
• Hypersensitivity to iodine

Sponsors & Collaborators

• University of Colorado, Denver: lead

Study Sites (1)

University of Colorado Denver Anschutz Medical Campus

Denver, Colorado, 80045, United States

Location

Related Publications (4)

• Cadnapaphornchai MA, George DM, McFann K, Wang W, Gitomer B, Strain JD, Schrier RW. Effect of pravastatin on total kidney volume, left ventricular mass index, and microalbuminuria in pediatric autosomal dominant polycystic kidney disease. Clin J Am Soc Nephrol. 2014 May;9(5):889-96. doi: 10.2215/CJN.08350813. Epub 2014 Apr 10.

  PMID: 24721893 BACKGROUND

• Gitomer BY, Ostrow A, Wang W, George D, Coleman E, Nowak KL, Cadnapaphornchai MA, Patel NU, Steele C, Jovanovich A, Klawitter J, Farmer B, You Z, Chonchol M. A randomized controlled trial evaluated the effect of pravastatin on kidney disease outcomes in adult patients with early-stage autosomal dominant polycystic kidney disease. Kidney Int. 2025 Oct 10:S0085-2538(25)00780-X. doi: 10.1016/j.kint.2025.08.037. Online ahead of print.

  PMID: 41077128 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.

  PMID: 39356039 DERIVED

• Gitomer BY, Wang W, George D, Coleman E, Nowak KL, Struemph T, Cadnapaphornchai MA, Patel NU, Jovanovich A, Klawitter J, Farmer B, Ostrow A, You Z, Chonchol M. Statin therapy in patients with early-stage autosomal dominant polycystic kidney disease: Design and baseline characteristics. Contemp Clin Trials. 2024 Feb;137:107423. doi: 10.1016/j.cct.2023.107423. Epub 2023 Dec 25.

  PMID: 38151173 DERIVED

MeSH Terms

Conditions

Polycystic Kidney, Autosomal Dominant

Interventions

Pravastatin

Condition Hierarchy (Ancestors)

Polycystic Kidney Diseases; Kidney Diseases, Cystic; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ciliopathies; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Naphthalenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds

Study Officials

• Michel Chonchol, MD

  University of Colorado, Denver

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Randomized, double-blind, placebo-controlled, parallel study design.

Study Record Dates

• First Submitted: September 1, 2017
• First Posted: September 6, 2017
• Study Start: August 31, 2017
• Primary Completion: March 25, 2024
• Study Completion: September 30, 2025
• Last Updated: May 8, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)