Study to Assess Safety & Efficacy of GKT137831 in Patients With Primary Biliary Cholangitis Receiving Ursodiol.
Condition: Primary Biliary Cirrhosis
MeSH terms: Cholangitis; Liver Cirrhosis, Biliary
Study type: Interventional
Phase: Phase 2

Summary: 
      The purpose of this study is to assess the safety and efficacy of GKT13783 in patients with
      Primary Biliary Cholangitis (PBC) who are taking a stable dose of ursodeoxycholic acid (UDCA)
      treatment, and have persistently high levels of a liver enzyme called Alkaline Phosphatase
      (ALP).
    

Detailed description: 
      Primary biliary cholangitis (PBC) is a disease of the liver. It is caused a sustained attack
      by the body's immune system on the bile ducts (canals) inside the liver. This continuous
      assault leads to their gradual destruction and eventual disappearance. This results in
      obstruction to the flow of bile which gets worse with disease progression. Once the bile duct
      injury has been established, the disease progresses due to ongoing obstruction of bile flow,
      inflammation and scarring of the liver tissue(fibrosis). The liver eventually fails.

      This research is looking into whether the study drug is better than a dummy drug when given
      to patients with PBC. This trial will monitor the patients taking part with regular blood
      tests and ultrasound liver scans before, during, and at the end of the trial. These measures
      will allow for the ongoing assessment of liver function, and liver stiffness. It is hoped
      that in patients in whom the study drug is beneficial, the liver function or stiffness may
      progress at a slower pace, or may even improve during or at the end of the trial. Liver
      injury, inflammation and fibrosis Participants will be randomly assigned to 1 of 3 treatment
      groups (active drug once daily, active drug twice daily or placebo). This is a double blinded
      study so neither the participants nor the staff responsible for their care will know which
      group they have been assigned to. During the treatment period, participants will take 4
      capsules orally at home in the morning and 4 capsules in the evening for 24 weeks.

      Participants will be in the trial for 32 weeks in total (about 8 months) and will attend
      approximately 8 clinic visits.
    

Intervention name(s): GKT137831; Placebo oral capsule
Intervention type(s): Drug; Drug
Intervention description(s): GKT137831 100mg capsules. To be taken as part of two dose arms which are 400mg twice daily or 400mg once daily.; Matching capsules.
Other intervention name(s): setanaxib; Placebo
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: This will be a double-blind, randomized, placebo-controlled, multicenter, parallel group phase 2 trial. A total of 102 subjects will be randomized and allocated to placebo or one of the 2 active treatment arms, according to a 1:1:1 randomization ratio, stratified at study entry by disease severity defined as baseline serum gamma glutamyl transferase (GGT) < 2.5 x ULN or ≥ 2.5 x ULN). Accordingly, approximately 34 subjects will be allocated to each of the 3 treatment arms.
Masking: Triple (Participant, Care Provider, Investigator)
Primary purpose: Treatment