Ustekinumab in Subjects with Primary Biliary Cirrhosis

• Research type

  Research Study

• Full title

  A Phase 2, Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel-group Study Evaluating the Efficacy and Safety of Ustekinumab in Subjects with Primary Biliary Cirrhosis Who had an Inadequate Response to Ursodeoxycholic Acid (UDCA)

• IRAS ID

  81423

• Sponsor organisation

  Janssen-Cilag International N.V.

• Eudract number

  2011-000554-31

• ISRCTN Number

  N/A

• Clinicaltrials.gov Identifier

  N/A

• Research summary

  Ustekinumab is an investigational drug that is being tested in patients with Primary Biliary Cirrhosis (PBC). It is given as a small injection just under the skin. PBC is an autoimmune disease of the liver where the bile ducts are slowly destroyed. Bile builds up in the liver and over time damages the tissue, leading to loss of liver function. Ustekinumab is one of a group of drugs known as monoclonal antibodies. Ustekinumab has been shown to interact with certain substances (interleukins) that control the body??s immune response. It may reduce the body??s immune response in autoimmune diseases such as PBC. This study aims to discover how safe and effective Ustekinumab is in treating patients with PBC who have not responded to previous treatment with ursodeoxycholic acid. The study consists of two parts. Subjects in Europe will only participate in Part 2. In Part 1, PBC patients will receive medical tests and 4 injections of the same dose of ustekinumab, over 36 weeks. In Part 2, PBC patients will receive medical tests and 4 injections of ustekinumab over 36 weeks, at one of 3 doses based on the results of the first part. Part 2 of the study is placebo controlled, meaning that subjects may receive ustekinumab or placebo (a dummy substance with identical appearance but without effective drug). Neither the subject nor the doctor will know which they are receiving. Subjects have a 2 in 3 chance of receiving active drug. Subjects completing Part 1 or Part 2 may then be invited to receive ustekinumab for a further 188 weeks. Overall, subjects will be in the trial for up to 224 weeks and visit the centre 31 times. Blood, urine and, where possible, liver biopsy samples will be taken. A pharmaceutical company is funding this study.

• REC name

  East Midlands - Nottingham 2 Research Ethics Committee

• REC reference

  11/EM/0256

• Date of REC Opinion

  21 Sep 2011

• REC opinion

  Further Information Favourable Opinion