Bempedoic Acid for Polycystic Kidney Disease

(BEAT-PKD Trial)

Not yet recruiting at 2 trial locations

Overseen By Charalett Diggs, RN, MSN

Age: 18 - 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Kenneth Hallows

Must not be taking: Bempedoic acid, Simvastatin, Pravastatin

Disqualifiers: Diabetes, Active coronary artery disease, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 3

Duration: 24 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test whether bempedoic acid, a drug typically used to lower cholesterol, can help treat autosomal dominant polycystic kidney disease (ADPKD). ADPKD causes cysts to grow in the kidneys, leading to kidney failure. The trial will determine if bempedoic acid can safely slow the disease's progression. Participants should have rapidly progressing ADPKD and a kidney function measurement (eGFR) of 35 or greater. The study will compare bempedoic acid to a placebo to evaluate its safety and potential effectiveness. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it does allow participants who are on a stable dose of Tolvaptan to join. If you are taking simvastatin or pravastatin above certain doses, you may need to adjust those medications.

Is there any evidence suggesting that bempedoic acid is likely to be safe for humans?

Research has shown that bempedoic acid, a drug approved by the FDA to lower cholesterol, is generally safe. In a study involving nearly 14,000 patients who couldn't take statins (another cholesterol-lowering drug), bempedoic acid was well-tolerated, and no major safety issues emerged. Even when tested in people with kidney problems, although drug levels in their bodies increased, no new safety concerns appeared.

These results suggest that bempedoic acid is usually safe for people. However, since this trial specifically examines its use for polycystic kidney disease, researchers continue to monitor for any new side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for polycystic kidney disease?

Unlike the standard treatments for polycystic kidney disease, such as blood pressure medications, pain relievers, or lifestyle changes, bempedoic acid targets a different pathway in the body. Bempedoic acid is unique because it works by inhibiting ATP citrate lyase, an enzyme involved in cholesterol synthesis, which may reduce cyst growth and kidney damage. Researchers are excited about this novel approach because it offers a potential new way to slow down the progression of the disease, providing hope for improved management of this chronic condition.

What evidence suggests that bempedoic acid might be an effective treatment for polycystic kidney disease?

Research has shown that bempedoic acid (BA) may help treat polycystic kidney disease. Studies have found that BA can slow cyst growth in lab tests by activating an enzyme called AMPK, which helps prevent growth and fluid buildup in kidney cysts. In animal studies, BA reduced the severity of polycystic kidney disease. This trial will compare the effects of BA alone to a placebo. The FDA has already approved BA for lowering cholesterol, indicating it is generally safe for use. ^{1 2 6 7 8}

Who Is on the Research Team?

Dana Miskulin, MD

Principal Investigator

Tufts University

Kenneth R Hallows, MD, PhD, FASN

Principal Investigator

University of Vermont

Stephen Seliger, MD

Principal Investigator

University of Maryland

Are You a Good Fit for This Trial?

This trial is for individuals with rapidly progressive Polycystic Kidney Disease (ADPKD) who have a kidney function score (estimated glomerular filtration rate) of 35 or higher. It includes those on or off a stable dose of Tolvaptan, and participants will be recruited from specific PKD clinics.

Inclusion Criteria

ADPKD patients as defined by Pei-Ravine criteria

Estimated glomerular filtration rate (eGFR) ≥35 ml/min/1.73m2 by CKD-Epi equation utilizing creatinine

Fluent English-speaking

Exclusion Criteria

I have diabetes or my fasting glucose level is above 125 mg/dL.

Proteinuria >500 mg/day

I am currently taking bempedoic acid or have had an allergic reaction to it.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either bempedoic acid or placebo for two years to assess safety, tolerability, and preliminary efficacy

24 months

In-person visits at baseline, 3, 6, 12, 18, and 24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Bempedoic Acid

Trial Overview

The trial is testing Bempedoic Acid, an FDA-approved cholesterol-lowering drug, to see if it can slow down ADPKD progression by activating certain enzymes in the body. This phase 2 study compares Bempedoic Acid against a placebo over two years.

How Is the Trial Designed?

Group I: Over-encapsulated bempedoic acid 180 mg p.o. once daily Active Control 1 Intervention

Group II: Matching over-encapsulated placebo pill given p.o. once daily Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Kenneth Hallows

Lead Sponsor

University of Southern California

Collaborator

Trials

956

Recruited

1,609,000+

University of Maryland, Baltimore County

Collaborator

Trials

13

Recruited

2,400+

Tufts Medical Center

Collaborator

Trials

264

Recruited

264,000+

Biomedical Research Institute of New Mexico

Collaborator

Trials

11

Recruited

2,300+

University of Pittsburgh

Collaborator

Trials

1,820

Recruited

16,360,000+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07282821

Bempedoic Acid Therapy for Polycystic Kidney Disease ...

Tolvaptan (Tol), the only FDA-approved drug for treatment of ADPKD, has some benefit in slowing kidney disease progression, but tolvaptan causes ...

2.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC9730828/

Beneficial effects of bempedoic acid treatment in polycystic ...

We conclude that BA and ACLY inhibition inhibited cyst growth in vitro, and BA decreased ADPKD severity in vivo. Combining BA with tolvaptan further improved ...

3.

c-path.org

c-path.org/emerging-therapies-in-autosomal-dominant-polycystic-kidney-disease/

Emerging Therapies in Autosomal Dominant Polycystic ...

Metabolic reprogramming is targeted by agents such as metformin, bempedoic acid, GLP-1 receptor agonists, and structured dietary interventions.

4.

journals.lww.com

journals.lww.com/kidney360/abstract/9900/emerging_therapies_in_autosomal_dominant.826.aspx

Emerging Therapies in Autosomal Dominant Polycystic ...

Metabolic reprogramming is targeted by agents such as metformin, bempedoic acid, GLP-1 receptor agonists, and structured dietary interventions.

5.

frontiersin.org

frontiersin.org/journals/molecular-biosciences/articles/10.3389/fmolb.2023.1126055/full

Metabolism-based approaches for autosomal dominant ...

Bempedoic acid was generally well-tolerated following a single oral dose in subjects with renal impairment (Amore et al., 2022).

6.

thelancet.com

thelancet.com/journals/landia/article/PIIS2213-8587(24)00063-9/fulltext

Evaluating the effect of bempedoic acid on kidney function

There are extensive published data on the safety of bempedoic acid from the CLEAR Outcomes trial, in which 13 970 statin-intolerant patients at high ...

7.

jacc.org

jacc.org/doi/10.1016/S0735-1097%2822%2902450-0

SAFETY AND EFFICACY OF BEMPEDOIC ACID IN ...

Bempedoic acid (BA) is an ATP citrate lyase inhibitor that lowers LDL-C. We evaluated the safety and efficacy of BA in patients with Stage 2 or ...

8.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC8932715/

Pharmacokinetics of bempedoic acid in patients with renal ...

Bempedoic acid exposures in subjects with renal impairment were increased up to approximately two‐fold with no safety signals identified.