A 9-year, Multicentre, Non-interventional, Post-authorisation Safety Study for Patients Prescribed JINARC® for Autosomal Dominant Polycystic Kidney Disease (JINARC PASS)

Study type

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

  Drug utilisation

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  JINARC

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)
• Special population of interest:
  Hepatic impaired

  Immunocompromised

  Pregnant women

  Renal impaired
• Estimated number of subjects:
  3000

Study design details

• Main study objective:
  To characterise and quantify the identified risk of idiosyncratic liver injury in JINARC treated patients with ADPKD in routine clinical practice.
• Outcomes:
  The incidence of patients with acceptable levels of transaminases at screening who experience an elevation of transaminase (alanine aminotransferase ALT or aspartate aminotransferase AST) ≥ 3x upper limit of normal (ULN), or an AE consistent with hepatotoxicity. • The incidence of patients who meet Hy’s laboratory criteria).• The number and percentage of patients - skin neoplasms (noting in particular basal cell carcinoma)- glaucoma• The frequency of all AEs Additional exploratory endpoints will also be assessed.
• Data analysis plan:
  Overall and stratified (eg, age, sex, race/ethnicity, country) data summaries will be presented, where possible. The main safety analyses will be evaluating the risk of serious hepatotoxicity, rare adverse outcomes (glaucoma and basal cell carcinoma), risk with long-term use, the frequency and outcome of pregnancies, the extent of off-label use, and use and risk in patients over the age of 50 years.