A Study of Efficacy and Safety of Ustekinumab in Patients With Primary Biliary Cirrhosis (PBC) Who Had an Inadequate Response to Ursodeoxycholic Acid
Condition: Primary Biliary Cirrhosis
MeSH terms: Liver Cirrhosis; Liver Cirrhosis, Biliary; Fibrosis
Study type: Interventional
Phase: Phase 2

Summary: 
      The purpose of this study is to evaluate the efficacy and safety of ustekinumab in patients
      with primary biliary cirrhosis who had an inadequate response to ursodeoxycholic acid.
    

Detailed description: 
      This is a multi-center, randomized (treatment will be assigned by chance), placebo-controlled
      (an inactive substance will be compared with the test drug to see whether the drug has a real
      effect), parallel-group (two or more groups of patients will receive different treatments)
      study that will consist of two parts. Part 1 will be an open-label (all participants will
      know the identity of the treatment) proof-of-concept study. Part 2 will be contingent on the
      results of Part 1 and will be double-blind (investigators and patients will not know what
      treatment is being given) and will evaluate the efficacy and safety of ustekinumab in
      patients with primary biliary cirrhosis (PBC) who had an inadequate response to
      ursodeoxycholic acid. The duration of participation in the study for an individual
      participant may be up to 216 weeks. Patient safety will be monitored. Part 1: ustekinumab,
      90mg subcutaneous (SC) at Weeks 0 and 4 and every 8 weeks through Week 20; Part 2: Depending
      on Part 1 results, either (ustekinumab 90mg or 45mg or placebo) or (ustekinumab 90mg or 180mg
      or placebo) SC at Weeks 0 and 4 and every 8 weeks through Week 20; Long-term Extension
      (including Part 1 and Part 2): beginning at Week 28, every 8 weeks with initially assigned
      dose until the extension dose has been selected; then every 8 weeks through Week 196 with the
      selected dose.
    

Intervention name(s): ustekinumab 90 mg; ustekinumab 45 mg; ustekinumab 180 mg; Placebo
Intervention type(s): Drug; Drug; Drug; Drug
Intervention description(s): Subcutaneous injections of ustekinumb 90 mg at Weeks 0 and 4 and every 8 weeks until the dose for the Long-Term Extension has been selected then every 8 weeks through Week 196 with the selected dose.; Subcutaneous injections of ustekinumb 45 mg at Weeks 0 and 4 and every 8 weeks until the dose for the Long-Term Extension has been selected then every 8 weeks through Week 196 with the selected dose.; Subcutaneous injections of ustekinumb 180 mg at Weeks 0 and 4 and every 8 weeks until the dose for the Long-Term Extension has been selected then every 8 weeks through Week 196 with the selected dose.; Subcutaneous injections of placebo at Weeks 0 and 4 and every 8 weeks until the dose for the Long-Term Extension has been selected then every 8 weeks through Week 196 with the selected dose.
Intervention MeSH terms: Ustekinumab
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Diseases [C] - Digestive System Diseases [C06]; primary biliary cirrhosis; ursodeoxycholic acid (UDCA); STELARA; ustekinumab