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Phase 2 N=20 Randomized Quadruple-blind Treatment

A Study of Efficacy and Safety of Ustekinumab in Patients With Primary Biliary Cirrhosis (PBC) Who Had an Inadequate Response to Ursodeoxycholic Acid

Primary Biliary Cirrhosis

Enrolled (actual)
20
Serious AEs
5.0%
Results posted
Oct 2014
Primary outcome: Primary: Part 1: Number of Participants With Alkaline Phosphatase (ALP) Response at Week 12 — 0 participants

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
ustekinumab 90 mg (Drug); ustekinumab 45 mg (Drug); ustekinumab 180 mg (Drug); Placebo (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Janssen Research & Development, LLC
Primary completion
Jun 2013

Outcome Measures

OutcomeResultp-value
PRIMARY
Part 1: Number of Participants With Alkaline Phosphatase (ALP) Response at Week 12
SECONDARY
Part 1: Number of Participants With ALP Response at Week 28
SECONDARY
Part 1: Number of Participants With ALP Remission at Week 28
SECONDARY
Part 1: Percent Change From Baseline in ALP Concentration at Week 28
-11.25
SECONDARY
Part 1: Percent Change From Baseline in Alanine Aminotransferase, Aspartate Aminotransferase, and Bilirubin Concentration at Week 28
-9.21; -7.22; -4.61

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.

Eligibility Criteria

Inclusion Criteria

  • Have proven or are likely to have Primary Biliary Cirrhosis (PBC)
  • Be on a stable dose of ursodeoxycholic acid for at least 6 months prior to Week 0
  • Have screening alkaline phosphatase (ALP) level > 1.67 ULN (the upper limit of normal)
  • Have screening laboratory test results within protocol-specified limits
  • Have no history of latent or active tuberculosis (TB) prior to screening and no signs or symptoms suggestive of active TB upon medical history and/or physical examination.

Exclusion Criteria

  • Has history of gastrointestinal bleeding, secondary to portal hypertension, hepatic encephalopathy, or ascites requiring treatment with diuretics
  • Has a screening direct bilirubin > 1.0 mg/dL
  • Has a previous liver histology with a diagnosis of steatohepatitis or has a high risk of nonalcoholic steatohepatitis
  • Has a previous liver histology with a diagnosis of chronic autoimmune hepatitis or has a high risk of autoimmune hepatitis overlap syndrome
  • Testing positive for surface antigen (HBsAg+), regardless of the results of other hepatitis B tests
  • Have used colchicine, methotrexate (MTX), azathioprine (AZA), or systemic corticosteroids within 3 months prior to the first administration of study drug.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01389973). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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