Multi-part, double blind study to assess safety, tolerability and effi

• Research type

  Research Study

• Full title

  A multi-part, randomized, double-blind, placebo-controlled study to assess the safety, tolerability and efficacy of LJN452 in patients with primary biliary cirrhosis

• IRAS ID

  186282

• Contact name

  David Jones

• Contact email

  david.jones@ncl.ac.uk

• Sponsor organisation

  Novartis Pharmaceuticals UK Ltd

• Eudract number

  2015-001590-41

• Duration of Study in the UK

  2 years, 2 months, 13 days

• Research summary

  This is a Phase II double-blind, placebo controlled, dose escalation study of LJN452 in patients suffering from primary biliary cirrhosis (PBC). It is not anticipated that patients will enjoy any therapeutic benefit from being on the trial. It is designed to evaluate the tolerability, efficacy and safety of LJN452 in this patient group. The study will last 4 weeks and is divided into two parts: Part 1 will involve approximately 5 cohorts consisting of 15 patients each, all of whom will be on standard of care therapy at the time of enrolment. These patients will receive up to 5 different dose levels of LJN452 on a 2:1 ratio which means that 10 patients per cohort will receive LJN452 and 5 patients will receive placebo. The active drug cohorts are broken down as follows:

  Cohort 1:
   LJN452 0.03 mg daily for 28 days (n=10) (given as 3 x LJN452 0.01 mg)
   LJN452 Placebo daily for 28 days (n=5)

  Cohort 2:
   LJN452 TBD mg daily for 28 days (n=10)
   LJN452 Placebo daily for 28 days (n=5)

  Cohorts 3-5 (if required) may receive different doses:
   LJN452 TBD mg daily for 28 days (n=10)
   LJN452 Placebo daily for 28 days (n=5)

  Part 2 will consist of 20 patients who are not receiving standard of care treatment at time of enrolment and this group will receive a 1:1 randomisation ratio which means that 10 patients per cohort will receive LJN452 for 28 days and 10 patients will receive placebo for 28 days. Due to the placebo arm of the study some patients in Part 2 will, for a time, receive neither UDCA nor LJN452, however previous studies have shown that withholding treatment for a short period was not associated with any detriment in the patients' condition. Part 2 will commence once Part I has finished.

• REC name

  North East - Newcastle & North Tyneside 1 Research Ethics Committee

• REC reference

  15/NE/0374

• Date of REC Opinion

  19 Feb 2016

• REC opinion

  Further Information Favourable Opinion