This is a a phase 2, multi-center, double-blind, placebo-controlled randomized clinical trial to evaluate the safety, tolerability, and preliminary efficacy of bempedoic acid (BA) in patients with ADPKD at high risk for disease progression and eGFR≥35 ml/min/1.73m2, with or without concurrent tolvaptan use.
BEAT-PKD
Bempedoic Acid Therapy for Polycystic Kidney Disease (BEAT-PKD) Randomized Clinical Trial
Polycystic kidney disease
All genders
18-60
Recruiting now
Overview
Principal Investigator: Dana Miskulin, MD, MS
Contact Us
Shuaib Mohammed
Study details
Inclusion Criteria
- Diagnosis of ADPKD
- Mayo Imaging Classification 1C,1D,1E or 1B with documented eGFR decline >3ml/min/1.73m2/year over at least 2 years with no other known causes
- Age 18-60 years with eGFR≥35 ml/min/1.73m2
Exclusion Criteria
- Proteinuria >500mg/g Cr or active hepatitis/abnormal liver function tests (LFTs)
- Serum uric acid >10mg/dL or ≥1 acute gout flare in the prior year
- Current use of simvastatin >20mg or pravastatin >40 mg daily, or active coronary artery disease.
Study Requirements
Participants in the BEAT-PKD study will be enrolled for 24 months and attend approximately 13 research visits, including screening, baseline, and follow-up visits every 3 months. Study procedures include blood draws for safety labs, metabolic panels, uric acid, lipid profiles, and biomarker analysis, as well as urine collections for pregnancy testing, biomarker analysis, and long-term biobanking. MRI scans will be performed at baseline, 12 months, and 24 months to assess kidney and liver volumes and visceral fat. Participants will also complete ADPKD-specific quality-of-life questionnaires and undergo pill counts and adverse event monitoring to assess drug tolerability and safety.