A non-interventional study to assess the long-term safety and efficacy of osilodrostat in patients with endogenous Cushing’s syndrome (LINC 6)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Drug utilisation

  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Drug interaction study

Study drug and medical condition

• Name of medicine:
  ISTURISA
• Medical condition to be studied:
  Cushing's syndrome

Population studied

• Age groups:
  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:
  100

Study design details

• Main study objective:
  The main objective of the study is to further document the long-term safety and tolerability profile of osilodrostat in routine clinical practice over a 3-year follow-up period when administered as monotherapy or in combination with other therapies in patients with endogenous Cushing's Syndrome.
• Outcomes:
  The primary outcome is the incidence of osilodrostat-related adverse events and serious adverse events during the 3 years of treatment with osilodrostat. Particular focus is on the Adverse Events of Special Interest (AESI) and Other Reportable Information (ORI). Short and long-term efficacy of osilodrostat, change in biochemical measures of disease activity, normalisation of biochemical measures of disease activity, change in cardiovascular- and metabolic-related parameters, change in physical features of the disease, changes in pituitary tumour size, changes in patient-reported outcome questionnaires, overall safety and tolerability of osilodrostat.
• Data analysis plan:
  The main goal is to further document the long-term safety and tolerability profile of osilodrostat by measuring the incidence of the safety events: o Related Adverse Events o Serious Adverse Events o Adverse Events of Special Interest o Other Reportable Information All safety summaries will show the number of AEs and the number and percentage of patients experiencing at least one event by AE category (All, related AEs, SAEs, AESI and ORI). In the secondary analyses, measurements will be presented using summary statistics, and may also be reported by looking at the change from baseline (actual and percentage) over time. For Quality of Life questionnaires, descriptive statistics including change from baseline will be presented over time. For all summary tables presented over time, time windows as defined in the SAP, will be applied.