Statin therapy in patients with early-stage autosomal dominant polycystic kidney disease: Design and baseline characteristics

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.