Reducing Exacerbations in People with PCD using Erdosteine and Azithromycin Therapy

Repeat Study

REDUCING EXACERBATIONS IN PEOPLE WITH PCD USING ERDOSTEINE AND AZITHROMYCIN THERAPY

Study Title

Improving outcomes of children and young adults with primary ciliary dyskinesia: a multi-centre, double-blind, double dummy, 2×2 partial factorial, randomised controlled trial.

What is study about:

The study is investigating if 12 months of Azithromycin and or Erdosteine can reduce exacerbations in children with PCD.

Main Aims of Study:

The main aims are to find out if 12 months of treatment with azithromycin (3 times/week) and/or Erdosteine (2 times/day)

  • Reduces the number of exacerbations (flare ups) in a 12-month period.
  • Improves clinical outcomes (Quality of Life (QoL), number of hospitalisations) and reduces non-scheduled doctor visits) by 12 months.
  • Reduces treatment costs.
  • Affects bacterial growth and antibiotic resistance in the respiratory tract (lungs and back of the nose) We are also looking at whether:
  • The microscopic structure of cilia in people with PCD affects the outcomes of the aims above.
  • Whole exome sequencing is useful in PCD for identifying PCD gene mutations. (Whole exome sequencing is a relatively new way of looking at genes).