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New potentiator moves into UK patient trials

UK CF clinics enrolling adult trial patients with G551D mutation.

A cystic fibrosis (CF) potentiator called ‘GLPG1837’, the first in a series of CF modulator compounds in the development pipeline from Belgian biotechnology firm Galapagos NV is now entering patient trials with enrolment starting in March 2016 at four UK sites.

Lab data for GLPG1837 demonstrated superior activity relative to ivacaftor in people with the G551D genotype, and now an adult trial, called Saphira 1, will trial three doses of GLPG1837 with the aim of evaluating the safety and tolerability of the drug, along with assessing changes in sweat chloride levels as a marker of CFTR ion channel function.

The target patient population for the study is:
  • Male or female
  • At least 18 years of age 
  • Cystic fibrosis with the G551D mutation 
  • Weigh at least 40 Kg (6 St., 5 Ib)
If you participate you would need to:
  • Be in the study for a maximum of eight to nine weeks, during which time you will visit the clinic seven times for various study assessments
  • Receive three different doses of the study treatment over a four-week period (two tablets, twice a day).  There is no placebo. 
  • Keep a daily diary of your medication and side effects
If you are currently taking Kalydeco (Ivacaftor) you will need to stop taking this medication for approximately five-and-a-half weeks during the study so that the true effect of the study drug can be assessed.

If you are interested in joining the Saphira 1 study, the following UK CF centres are participating:

  • Queen Elizabeth University Hospital, Glasgow, Dr Gordon MacGregor; get in touch.
  • Royal Brompton Hospital, London, Prof Jane Davies; get in touch
  • Liverpool Heart and Chest Hospital, Dr James Greenwood; get in touch.
  • The Medicines Evaluation Unit, Manchester, Dr Alex Horsley; get in touch.