Setanaxib for Primary Biliary Cholangitis

(TRANSFORM Trial)

Recruiting at 127 trial locations

Overseen By Stefan Carlsson

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Genkyotex Suisse SA

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

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

76 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

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.

Inclusion Criteria

Serum ALP ≥1.67×ULN at Screening

Definite or probable PBC diagnosis as demonstrated by the presence of ≥2 of the following 3 diagnostic factors:

For the purposes of this trial, women of childbearing potential are defined as 'Fertile, following menarche and until becoming postmenopausal unless permanently sterile. Permanent sterilization methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy.'

Exclusion Criteria

My bilirubin levels are high, but my albumin levels are normal.

I have had a liver transplant, am waiting for one, or have severe liver disease.

Presence of any laboratory abnormality or condition that, in the opinion of the Investigator, could interfere with or compromise a participant's treatment, assessment, or compliance with the protocol and/or study procedures

Timeline

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Treatment

Participants receive setanaxib or placebo for a 24-week double-blind treatment period

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Setanaxib

Trial Overview 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.

Participant Groups

Group I: Setanaxib 1600 mg/day Experimental Treatment 1 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/day Experimental Treatment 1 Intervention

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

Group III: Placebo Placebo Group 1 Intervention

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

Who Is Running the Clinical Trial?

Genkyotex Suisse SA

Lead Sponsor

Trials

2

Recruited

130+

Calliditas Therapeutics Suisse SA

Lead Sponsor

Trials

2

Recruited

130+