Avacostar - A Post Authorization Safety Study (PASS) to Evaluate the Incidence of Safety Events of Interest in Patients Treated With Avacopan for ANCAassociated Vasculitis (AAV)

Study type

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

  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  TAVNEOS
• Medical condition to be studied:
  Anti-neutrophil cytoplasmic antibody positive vasculitis

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:
  500

Study design details

• Main study objective:
  To evaluate the incidence of defined MESIs in patients with AAV commencing avacopan.
• Outcomes:
  - To evaluate the incidence of defined MESIs. MESIs include liver injury,cardiac safety, serious infection, and malignancy. Incidence rates of AEs, AEs leading to discontinuation of therapy, SAEs, ADRs SADRs, change in recorded laboratory assessments over time, time to first flare, and change in VDI score over time for the avacopan group. Incidence rates of AEs, MESIs, SAEs, change in lab results over time, time to first flare, and change in VDI score over time for the non-avacopan group. Refer to protocol for more.
• Data analysis plan:
  Statistical analyses will be exploratory in nature. All variables will be analysed descriptively with appropriate statistical methods: categorical variables by frequency tables (absolute and relative frequencies) and continuous variables by descriptive statistics (i.e. number of patients, mean, standard deviation, minimum, median, quartiles, and maximum). Continuous variables will be summarised by absolute value and changes from baseline per analysis time point, if applicable. 95% confidence intervals will be provided where appropriate. Comparisons between treatment groups will be performed using appropriate methods to adjust for potential bias. Outcomes of statistical comparisons will be interpreted with caution. All statistical issues including calculated variables, handling of missing data, and the format and content of tables will be detailed in the Statistical Analysis Plan (SAP). The SAP will be finalised before study database lock.