Praliciguat for Focal Segmental Glomerulosclerosis

Overseen By Akebia Therapeutics

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Akebia Therapeutics

Must be taking: ACEi, ARB

Disqualifiers: Collapsing FSGS, Sickle cell, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

60 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests praliciguat, a new treatment for focal segmental glomerulosclerosis (FSGS), a kidney disease that can lead to kidney failure. The study aims to determine if praliciguat is safe and effective for this condition. Participants will receive either praliciguat or a placebo for 24 weeks, followed by praliciguat for another 24 weeks. Eligible participants must have an FSGS diagnosis confirmed by a kidney biopsy and currently take medication for high blood pressure. As a Phase 2 trial, this research measures praliciguat's effectiveness in an initial, smaller group, allowing participants to contribute to significant medical advancements.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you must be on the highest dose of ACE inhibitors or ARBs that you can tolerate. It's best to discuss your specific medications with the trial team.

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

Research shows that praliciguat is being tested for safety and effectiveness in treating focal segmental glomerulosclerosis (FSGS), a rare kidney disease. Earlier studies found no major safety concerns with praliciguat, suggesting it might be well-tolerated.

The trial has now reached a stage involving more participants to better assess the treatment's safety. Although no serious side effects have been reported so far, side effects can vary among individuals. Participants will take the drug over a period of time, allowing researchers to gather more safety data.

Prospective participants should discuss any concerns with the trial team or their doctor. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for FSGS?

Praliciguat is unique because it targets a different pathway than most current treatments for focal segmental glomerulosclerosis (FSGS), which generally focus on managing symptoms with steroids or immunosuppressants. Praliciguat works by enhancing the nitric oxide-cyclic GMP pathway, which can improve kidney function by reducing inflammation and fibrosis. Researchers are excited about this treatment because it offers a novel mechanism of action that holds the potential for better outcomes and fewer side effects compared to traditional therapies.

What evidence suggests that praliciguat might be an effective treatment for FSGS?

Research has shown that praliciguat might help treat focal segmental glomerulosclerosis (FSGS). In this trial, participants will receive either praliciguat or a placebo. Praliciguat opens blood vessels, which can lower blood pressure. Early results suggest it could also improve kidney function, crucial for people with FSGS. Past studies found praliciguat generally safe, with few side effects. While it primarily affects blood pressure, researchers believe it might also benefit kidney health. However, more research is needed to confirm its effectiveness for FSGS. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

Adults with biopsy-confirmed FSGS (a kidney disease causing scarring and protein loss in urine) can join this trial. Participants will be randomly assigned to receive either the test drug praliciguat or a placebo for 24 weeks, followed by an open-label extension where all get praliciguat.

Inclusion Criteria

UPCR ≥1 (g/g) during screening

I have been on the highest dose of ACE inhibitors or ARBs that I can tolerate for at least a month.

Estimated glomerular filtration rate ≥25 milliliters per minute per 1.73 square meters by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation

Exclusion Criteria

Collapsing FSGS in the kidney biopsy report

HbA1c >8% or non-fasting blood glucose >180 milligram/decilitre

My blood pressure is not higher than 160/100 mmHg.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive praliciguat or placebo for an initial 24-week double-blind period

24 weeks

Open-label extension

All participants receive praliciguat in an open-label extension for an additional 24 weeks

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Praliciguat

Trial Overview

The study is testing the effectiveness and safety of praliciguat compared to a placebo in treating FSGS. It's a double-blind study, meaning neither participants nor researchers know who gets the real drug during the first phase.

How Is the Trial Designed?

Group I: Praliciguat Experimental Treatment 1 Intervention

Praliciguat will be administered daily with dose escalation to a target dose during a 24-week double-blind period, followed by a 24-week open-label treatment period.

Group II: Placebo Placebo Group 1 Intervention

Matching placebo will be administered daily for 24 weeks

Who Is Running the Clinical Trial?

Akebia Therapeutics

Lead Sponsor

Trials

35

Recruited

12,400+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07268638

A Study of Praliciguat in Participants With Focal Segmental ...

This is a Phase 2, randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the efficacy and safety of praliciguat ...

2.

ir.akebia.com

ir.akebia.com/news-releases/news-release-details/akebia-announces-establishment-rare-kidney-disease-pipeline

Akebia Announces Establishment of Rare Kidney Disease ...

Praliciguat adverse events were infrequent and consistent with its known blood pressure lowering effect. Akebia filed its Investigational New ...

3.

trial.medpath.com

trial.medpath.com/clinical-trial/17466a615ebb976b/nct07268638-praliciguat-focal-segmental-glomerulosclerosis-study

A Study of Praliciguat in Participants With Focal Segmental ...

This is a Phase 2, randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the efficacy and safety of ...

4.

pharmaceutical-technology.com

pharmaceutical-technology.com/data-insights/praliciguat-akebia-therapeutics-focal-segmental-glomerulosclerosis-fsgs-likelihood-of-approval/

Praliciguat by Akebia Therapeutics for Focal Segmental ...

Praliciguat is under clinical development by Akebia Therapeutics and currently in Phase I for Focal Segmental Glomerulosclerosis (FSGS).

5.

sec.gov

sec.gov/Archives/edgar/data/1517022/000151702225000051/akb097_finalx1212025.htm

Akebia Announces Establishment of Rare Kidney Disease Pipeline

Phase 2 trial of Praliciguat, an sGC stimulator, initiated in focal segmental glomerulosclerosis (FSGS). Both trials planned to start treating subjects in 2026.

6.

theglobeandmail.com

theglobeandmail.com/investing/markets/stocks/AKBA/pressreleases/36383338/akebia-announces-establishment-of-rare-kidney-disease-pipeline/

Akebia Announces Establishment of Rare Kidney Disease ...

Praliciguat, is an oral soluble guanylate cyclase (sGC) licensed by Akebia from Cyclerion Therapeutics, Inc. in June 2021. No significant safety ...