TRANSFORM

• Research type

  Research Study

• Full title

  A 24-week, Randomized, Placebo-controlled, Double-blind, Phase 2b Trial of Setanaxib in Patients with Primary Biliary Cholangitis (PBC) and Elevated Liver Stiffness

• IRAS ID

  1004094

• Contact name

  Richard Philipson

• Sponsor organisation

  Genkyotex Suisse SA

• Eudract number

  2021-001810-13

• Research summary

  This is a 52 week randomised phase 2b/3 study of an investigational (experimental) medication called Setanaxib. The primary purpose of this study is to review the safety and effectiveness of Setanaxib in participants with primary biliary cholangitis (PBC). There is an unmet medical need in PBC patients with reported inadequate responses to the current approved treatment options such as ursodeoxycholic acid (UDCA) and more recently another medicine, obeticholic acid (OCA). Previous studies have shown that Setanaxib has improved symptoms of PBC and this study is designed to confirm these findings in the hope of meeting this unmet medical need.

  Up to approximately 318 participants are expected to participate in this study at 150-200 study sites globally.

  This study will consist of 4 parts: Screening Period, Main Phase, Extension Phase and Follow up Phase.
  Screening Phase – expected to last 4 weeks. Participants will undergo a series of tests to confirm if they are eligible for participation in the Main phase of the study.
  Main Phase – expected to last 52 weeks (1 year). Participants will be randomised to receive either Setanaxib or a placebo.
  Extension Phase – expected to last 52 weeks (1 year). All participants will receive Setanaxib regardless of what treatment they received in the Main Phase.
  Follow up Phase – consisting of 2 visits over a 12 week (3 month) period.
  Participation in this study is expected to be up to 120 weeks (2 years 4 months) in total.

  Lay summary of study results: The lay summary is not currently available.
  As per European requirements, the results of the clinical trial will be uploaded in the EudraCT database (https://gbr01.safelinks.protection.outlook.com/?url=https%3A%2F%2Fwww.clinicaltrialsregister.eu%2Fctr-search%2Fsearch%3Fquery%3D2021-001810-13&data=05%7C02%7Ccoventryandwarwick.rec%40hra.nhs.uk%7C7b159fd56321460a4b0908dd1dc46615%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638699451540832268%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=RF0lOFZPpC4VSJCxuCC1L4lthRgZ8W3PWSMHkMNeHKc%3D&reserved=0) by June 2025.

• REC name

  West Midlands - Coventry & Warwickshire Research Ethics Committee

• REC reference

  21/WM/0250

• Date of REC Opinion

  6 Dec 2021

• REC opinion

  Further Information Favourable Opinion