An Open-label Observational Safety Study of Colobreathe® (colistimethate sodium dry powder for inhalation) Compared with Other Inhaled Anti-pseudomonal Antibiotics in Cystic Fibrosis Patients Using Cystic Fibrosis Registries

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  COLOBREATHE
• Study drug International non-proprietary name (INN) or common name:
  COLISTIMETHATE SODIUM

Population studied

• Short description of the study population:
  Patients enrolled in the UK cystic fibrosis (CF) registry database who were prescribed Colobreathe (treated group) and matched patients in the CF registry database not treated with Colobreathe but taking other inhaled antibiotic treatments (Comparator-treated).
• Age groups:
  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  11400

Study design details

• Main study objective:
  The primary objective is to evaluate the long-term safety of Colobreathe used in patients with cystic fibrosis with P. aeruginosa infection of the lungs, compared with other inhaled anti-pseudomonal antibiotics.
• Outcomes:
  Annual rates of all adverse events (AEs) reported for the Colobreathe-treated and matched, comparator-treated patients, 1. Reasons for discontinuing treatment2. AEs of special interest (cough/productive cough, chest discomfort/chest pain, wheezing/bronchospasm, dysphonia, dyspnoea, lower respiratory tract infection, and taste abnormality dysgeusia) in the first 90 days of treatment3. Off-label use4. PA exacerbations
• Data analysis plan:
  At each interim (6-month) data review, descriptive analyses will be conducted to describe the demographic features of patients, incidence rates of AEs, and the frequency and reasons for discontinuing treatment.Interim analyses will include comparative analyses. A negative binomial model will be used to compare the annualized rate of any AEs between Colobreathe-treated and comparator-treated groups. Model estimates will be adjusted for propensity score.