VOICE

• Research type

  Research Study

• Full title

  Observational Registry of Cystic Fibrosis Patients in Europe

• IRAS ID

  193655

• Contact name

  Siobhan Carr

• Contact email

  s.carr@rbht.nhs.uk

• Sponsor organisation

  Vertex Pharmaceuticals Incorporated

• Duration of Study in the UK

  5 years, 0 months, 0 days

• Research summary

  New medications have been produced to target different mutations in CF, however, although several registries have been previously conducted, on patients with Cystic Fibrosis, none have involved encounter based data collection.

  The purpose of this study is to describe the clinical management of cystic fibrosis patients who are homozygous for the F508del-CFTR mutation, define risk factors for rapid clinical deterioration and to monitor the effectiveness of new therapeutic interventions.

  This is a 5 year, multi-centre, retrospective and prospective, non-interventional registry study of patients with Cystic Fibrosis (CF) homozygous for the F508del-CFTR mutation.

  4000 patients will be participating in the study within Europe.

  Patients with CF homozygous for the F508del-CFTR mutation, will be followed for 5 years as part of routine care. No additional procedures to those performed as part of routine care will be performed.

• REC name

  East Midlands - Nottingham 2 Research Ethics Committee

• REC reference

  16/EM/0331

• Date of REC Opinion

  26 Jul 2016

• REC opinion

  Unfavourable Opinion