A prospective, observational study on the correlations between change in lung function and change in cough and dyspnoea in patients with connective tissue disease-associated progressive fibrosing INTErstitial luNg diseaSE (CTD associated PF-ILD) treated with nintedanib (INTENSE)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)

Study drug and medical condition

• Name of medicine:
  OFEV
• Medical condition to be studied:
  Pulmonary fibrosis
• Additional medical condition(s):
  Connective tissue disease

Population studied

• Age groups:
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)
• Estimated number of subjects:
  120

Study design details

• Main study objective:
  The main objective of the study is to investigate the correlation between changes from baseline at Month 24 in FVC % pred. and changes from baseline at Month 24 in dyspnoea score points and cough score points as measured with the L-PF questionnaire over 24 months of nintedanib treatment in patients with CTD suffering from chronic fibrosing ILD with a progressive phenotype (excluding IPF).
• Outcomes:
  • The correlation between changes from baseline at Month 24 in FVC % pred. and changes from baseline at Month 24 in dyspnoea symptom score points • The correlation between changes from baseline at Month 24 in FVC % pred. and changes from baseline at Month 24 in cough symptom score points, • The correlation between change from baseline at Month 24 in FVC mL and change from baseline at Month 24 in dyspnoea symptom score • The correlation between change from baseline at Month 24 in FVC mL and change from baseline at Month 24 in cough symptom score • The correlation between baseline FVC % pred and change in dyspnoea symptom score from baseline at Month 24
• Data analysis plan:
  Continuous characteristics will be presented in the form of mean values (SD) and/ or median values (IQR, expressed in the form 25th– 75th percentile), depending on the fulfilment of the normality assumption for their distribution. The normality assumption will be examined via graphical means (PP – plot, QQ – plot, and Histograms) alongside the statistical tests of Kolmogorov-Smirnov and Shapiro-Wilk, depending on the available valid sample size available for every variable examined. Categorical characteristics will be presented in the form of absolute (N) and relative frequencies (%). For the analysis of the primary outcome, the Pearson and/or the Spearman correlation coefficient (depending on the fulfilment of the normality assumption of their distribution) will be calculated, along with their 95% confidence intervals and the two-sided p-values. The respective null hypothesis will be rejected in case if p ≤ 0.025 (Bonferroni correction for mult