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

Berenice Y Gitomer; Wei Wang; Diana George; Erin Coleman; Kristen L Nowak; Taylor Struemph; Melissa A Cadnapaphornchai; Nayana U Patel; Anna Jovanovich; Jelena Klawitter; Beverly Farmer; Anna Ostrow; Zhiying You; Michel Chonchol

doi:10.1016/j.cct.2023.107423

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.

Authors

Affiliations

¹ Division of Renal Diseases and Hypertension, University of Colorado Anschutz Medical Campus, 13199 East Montview Blvd., Suite 495, Aurora, CO 80045, USA. Electronic address: Berenice.gitomer@cuanschutz.edu.
² Division of Renal Diseases and Hypertension, University of Colorado Anschutz Medical Campus, 13199 East Montview Blvd., Suite 495, Aurora, CO 80045, USA. Electronic address: wei.wang@cuanschutz.edu.
³ Division of Renal Diseases and Hypertension, University of Colorado Anschutz Medical Campus, 13199 East Montview Blvd., Suite 495, Aurora, CO 80045, USA. Electronic address: diana.george@cuanschutz.edu.
⁴ Division of Renal Diseases and Hypertension, University of Colorado Anschutz Medical Campus, 13199 East Montview Blvd., Suite 495, Aurora, CO 80045, USA. Electronic address: erin.coleman@cuanschutz.edu.
⁵ Division of Renal Diseases and Hypertension, University of Colorado Anschutz Medical Campus, 13199 East Montview Blvd., Suite 495, Aurora, CO 80045, USA. Electronic address: kristen.nowak@cuanschutz.edu.
⁶ Division of Renal Diseases and Hypertension, University of Colorado Anschutz Medical Campus, 13199 East Montview Blvd., Suite 495, Aurora, CO 80045, USA. Electronic address: taylor.struemph@cuanschutz.edu.
⁷ Rocky Mountain Pediatric Kidney Center, Rocky Mountain Hospital for Children at Presbyterian/St. Luke's Medical Center, 2055 N. High St., Suite 205, Denver, CO 80205, USA.
⁸ Department of Radiology, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA. Electronic address: NaUPatel@salud.unm.edu.
⁹ Division of Renal Diseases and Hypertension, University of Colorado Anschutz Medical Campus, 13199 East Montview Blvd., Suite 495, Aurora, CO 80045, USA; VA Eastern Colorado Healthcare System, Aurora, CO, USA. Electronic address: anna.jovanovich@cuanschutz.edu.
¹⁰ Division of Renal Diseases and Hypertension, University of Colorado Anschutz Medical Campus, 13199 East Montview Blvd., Suite 495, Aurora, CO 80045, USA. Electronic address: jelena.klawitter@cuanschutz.edu.
¹¹ Division of Renal Diseases and Hypertension, University of Colorado Anschutz Medical Campus, 13199 East Montview Blvd., Suite 495, Aurora, CO 80045, USA. Electronic address: beverly.farmer@cuanschutz.edu.
¹² Division of Renal Diseases and Hypertension, University of Colorado Anschutz Medical Campus, 13199 East Montview Blvd., Suite 495, Aurora, CO 80045, USA. Electronic address: anna.ostrow@cuanschutz.edu.
¹³ Division of Renal Diseases and Hypertension, University of Colorado Anschutz Medical Campus, 13199 East Montview Blvd., Suite 495, Aurora, CO 80045, USA. Electronic address: zhiying.you@cuanschutz.edu.
¹⁴ Division of Renal Diseases and Hypertension, University of Colorado Anschutz Medical Campus, 13199 East Montview Blvd., Suite 495, Aurora, CO 80045, USA. Electronic address: michel.chonchol@cuanschutz.edu.

PMID: 38151173
DOI: 10.1016/j.cct.2023.107423

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