What side effects are associated with this type of intervention?

Common treatments for Primary Biliary Cirrhosis (PBC) include ursodeoxycholic acid (UDCA), which can cause side effects such as weight gain, hair thinning, and diarrhea. Statins, used to manage hypercholesterolemia in PBC patients, may lead to muscle pain, liver enzyme abnormalities, and, in rare cases, liver toxicity. Setanaxib, a Selective NADPH Oxidase Inhibitor, is being studied for its effects on PBC, but specific side effects are not well-documented yet. However, potential side effects could include gastrointestinal disturbances and liver enzyme changes, similar to other treatments in this category.

What prior FDA approvals exist?

The FDA has approved several treatments for Primary Biliary Cirrhosis (PBC). Ursodeoxycholic acid (UDCA) was the first drug approved and remains a standard treatment, improving liver function tests and delaying disease progression. Obeticholic acid (OCA) is another FDA-approved drug for PBC, particularly for patients who have an inadequate response to UDCA. OCA works as a farnesoid X receptor (FXR) agonist, which is different from the mechanism of Setanaxib, a Selective NADPH Oxidase Inhibitor. While Setanaxib is being studied for its potential to reduce liver stiffness and improve alkaline phosphatase levels, it represents a novel approach compared to existing treatments.

What other types of research exist?

Promising novel alternatives to Setanaxib for Primary Biliary Cirrhosis patients include picroside II, which has hepatoprotective effects, and platycodin D, which reduces hepatic injury and fibrosis. Additionally, liver-selective nitric oxide donors like V-PYRRO/NO may offer protective benefits against liver toxicity.

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FXR Agonist

Setanaxib for Primary Biliary Cholangitis (TRANSFORM Trial)

Phase 2

Waitlist Available

Research Sponsored by Genkyotex Suisse SA

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up screening (day -28) and week 24

Summary

This trial is testing setanaxib, a drug that may help lower liver enzyme levels, in people with a liver condition called PBC who don't respond well to the usual treatment. The goal is to see if setanaxib can improve their liver function by reducing enzyme levels. Setanaxib is being tested for its potential to improve liver function in PBC patients who do not respond well to standard treatments.

Who is the study for?

Adults diagnosed with primary biliary cholangitis (PBC) who have an inadequate response or intolerance to ursodeoxycholic acid, and a liver stiffness of ≥8.8 kPa. Participants must not be pregnant, should use effective contraception, and cannot have certain conditions like high bilirubin levels, significant kidney dysfunction, other liver diseases, recent infections requiring antibiotics, or any history of hypersensitivity to setanaxib.

What is being tested?

The trial is testing the effectiveness of setanaxib on patients with PBC compared to a placebo. The main goal is to see if there's an improvement in liver function after 52 weeks for those taking setanaxib who previously didn't respond well or couldn't tolerate standard treatment.

What are the potential side effects?

While specific side effects for setanaxib are not listed here, common side effects may include gastrointestinal symptoms such as diarrhea and abdominal pain (as noted in UDCA intolerance), potential allergic reactions due to drug sensitivity, and general medication-related risks like fatigue or headaches.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ screening (day -28) and week 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~screening (day -28) and week 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and screening (day -28) and week 24 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Change from Baseline in Fatigue

Change from Baseline in PGIC Pruritus

Change from Baseline in PGIS Pruritus

+7 more

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Setanaxib 1600 mg/dayExperimental Treatment1 Intervention

Participants will be administered setanaxib at a dose of 1600 mg/day for the 24-week double-blind treatment period.

Group II: Setanaxib 1200 mg/dayExperimental Treatment1 Intervention

Participants will be administered setanaxib at a dose of 1200 mg/day for the 24-week double-blind treatment period.

Group III: PlaceboPlacebo Group1 Intervention

Participants will be administered a placebo for the 24-week double-blind treatment period.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Setanaxib

2022

Completed Phase 2

~140

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Primary Biliary Cirrhosis (PBC) include ursodeoxycholic acid (UDCA) and obeticholic acid (OCA). UDCA works by reducing the concentration of toxic bile acids, thereby decreasing liver inflammation and damage. OCA, a farnesoid X receptor agonist, helps to reduce bile acid synthesis and increase bile flow. These mechanisms are crucial for PBC patients as they help to slow disease progression and alleviate symptoms. Setanaxib, a selective NADPH oxidase inhibitor, is being studied for its potential to reduce oxidative stress and inflammation in PBC, offering a novel approach to managing the disease.