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.

Abstract

Background: Autosomal dominant polycystic kidney disease (ADPKD) is characterized by the development and continued growth of multiple cysts in the kidneys leading to ultimate loss of kidney function in most patients. Currently, tolvaptan is the only agency approved therapy to slow kidney disease advancement in patients with faster progressing disease underscoring the need for additional ADPKD therapies suitable for all patients. We previously showed that pravastatin slowed kidney disease progression in children and young adults with ADPKD. However, the intervention has not been tested in an adult cohort.

Aims: The aim of the study is to conduct a single center, randomized, placebo-controlled double-blinded clinical trial to determine the efficacy of pravastatin on slowing kidney disease progression in adult patients with early stage ADPKD.

Methods: One hundred and fifty adult patients with ADPKD and eGFR ≥60 ml/min/1.73m2 will be enrolled in the study and randomized to receive 40 mg/day pravastatin or placebo for a period of 2-years.

Outcomes: The primary outcome of the trial is change in total kidney volume assessed by magnetic resonance imaging (MRI). Secondary outcomes include change in kidney function by iothalamate GFR and renal blood flow and markers of inflammation and oxidative stress.

Conclusion: This study will assess the kidney therapeutic benefits of pravastatin in adult patients with ADPKD. The recruitment goal of 150 subjects was attained and the study is ongoing.

Registration: This study is registered on Clinicaltrials.gov # NCT03273413.

Keywords: Kidney function; Polycystic kidney disease; Statin; Total kidney volume.

Publication types

  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Adult
  • Child
  • Disease Progression
  • Double-Blind Method
  • Glomerular Filtration Rate
  • Humans
  • Hydroxymethylglutaryl-CoA Reductase Inhibitors* / therapeutic use
  • Polycystic Kidney, Autosomal Dominant* / complications
  • Polycystic Kidney, Autosomal Dominant* / diagnostic imaging
  • Polycystic Kidney, Autosomal Dominant* / drug therapy
  • Pravastatin / therapeutic use
  • Young Adult

Substances

  • Hydroxymethylglutaryl-CoA Reductase Inhibitors
  • Pravastatin

Associated data

  • ClinicalTrials.gov/NCT03273413