Safety and efficacy study of patients with primary Sjögren’s Syndrome

• Research type

  Research Study

• Full title

  A randomised, double blind, placebo controlled multicentre Phase 2 dose ranging study to assess the safety and efficacy of multiple VAY736 doses administered subcutaneously in patients with moderate to severe primary Sjögren’s syndrome

• IRAS ID

  222956

• Contact name

  Robert Moots

• Contact email

  R.J.Moots@liverpool.ac.uk

• Sponsor organisation

  Novartis Pharma AG

• Eudract number

  2016-003292-22

• Duration of Study in the UK

  2 years, 5 months, 14 days

• Research summary

  Research Summary

  Sjögren's Syndrome (SS) is the UK’s second most common autoimmune rheumatic disease, yet the condition remains under recognised and frequently under treated. The effects of SS can be widespread; the moisture-producing glands of the body are affected and many organ systems may be involved as well.
  
  Primary Sjögren’s Syndrome (pSS) does not command a high profile within the medical profession and the general public is largely unaware of the problems faced by sufferers. Currently, treatment for patients with pSS is limited to remedies that provide only symptomatic relief for dryness of for example the eyes and mouth.
  Steroids, a class of powerful anti-inflammatory medicines, do not work for most patients with pSS and no treatment exists to relieve severe, disabling fatigue.

  VAY736, the drug currently being tested is called a monoclonal antibody. This drug is able to recognise, target and stick to specific proteins on specific cells. VAY736 binds to a protein on B cells (a group of white blood cells). These cells play a role in the development of pSS.

  This study aims to find out if VAY736 is safe and has beneficial effects in people who have moderate to severe pSS. Eligible patients will initially be randomly allocated to one of four treatment arms:
  Treatment arm 1: 5mg
  Treatment arm 2: 50mg
  Treatment arm 3: 300mg
  Treatment arm 4: placebo
  The treatment period will last for 24 weeks and after this, for a subset of patients up to 52 weeks:
  Treatment arm 5: Previous placebo(arm 4) group will receive VAY736 for 28 weeks
  Treatment arm 6: VAY736 for 28 weeks
  Treatment arm 7: placebo for 28 weeks

  A post treatment safety follow up period for all patients will last for a minimum of 20 weeks.

  Approximately 180 patients will be recruited worldwide, with 11 patients being recruited in the UK, from up to 5 sites.

  Summary of Results

  This study met its primary objective. The dose of 300 mg monthly was identified as providing the most patient benefit in lowering disease activity. The safety profile of ianalumab obtained in this Phase 2b trial is favorable and consistent with the previous experience. This study demonstrates a clinically significant treatment benefit to patients with Sjögren’s syndrome, a B-cell-associated autoimmune disease of high unmet need. Further studies are needed in Phase 3 to further evaluate these treatment benefits.

• REC name

  North East - Tyne & Wear South Research Ethics Committee

• REC reference

  17/NE/0096

• Date of REC Opinion

  8 May 2017

• REC opinion

  Further Information Favourable Opinion