Post-authorization safety study of iptacopan in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) using data from the non-interventional IPIG PNH Registry

Study type

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

  Safety study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine, other:
  Fabhalta
• Study drug International non-proprietary name (INN) or common name:
  IPTACOPAN
• Anatomical Therapeutic Chemical (ATC) code:
  (L04AJ08) iptacopan

  iptacopan
• Medical condition to be studied:
  Paroxysmal nocturnal haemoglobinuria

Population studied

• Short description of the study population:
  Adult patients with paroxysmal nocturnal haemoglobinuria treated with iptacopan.
• Age groups:
  In utero

  Adult and elderly population (≥18 years)

  Adults (18 to < 65 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Elderly (≥ 65 years)
• Estimated number of subjects:
  200

Study design details

• Study design:
  A multinational non-interventional descriptive single-arm cohort study based on secondary analysis of the data collected within the IPIG PNH Registry on iptacopan-treated patients.
• Main study objective:
  The primary objective of the study is to describe the risk of infections caused by encapsulated bacteria in patients with PNH treated with iptacopan in routine clinical practice.
• Comparators:
  None
• Outcomes:
  ⦁ Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae); ⦁ Serious infections caused by encapsulated bacteria; ⦁ All serious infections ; ⦁ Proportion of patients with vaccinations against encapsulated bacteria at the start of iptacopan treatment and at each subsequent visit; ⦁ Breakthrough hemolysis ; ⦁ Serious hemolysis following discontinuation of iptacopan (within 14 days from discontinuation) ; ⦁ Major adverse vascular events including thrombotic events ; ⦁ Malignancies; ⦁ Hyperlipidemia; ⦁ Thrombocytopenia; ⦁ Serious adverse events; ⦁ All-cause mortality; ⦁ Pregnancy-related outcomes
• Data analysis plan:
  The analysis in this study will be focused on the while-on-treatment and the hypothetical risk estimands. The treatment policy estimand strategy will not be implemented in this study because subjects discontinuing iptacopan are expected to start another PNH therapy. ; ; Risk will be evaluated in terms of frequency (counts and percentages), cumulative incidence (event probability as a function of time), incidence rates (number of patients with events per 100 patient-years) and occurrence rates (number of episodes per 100 patient-years). Incidence and occurrence rates will be calculated by year of treatment and for the entire duration of the study.; ; Subgroup and sensitivity analyses will be performed as described in the study Statistical Analysis Plan.

Documents

Study, other information

05.01.0301 Feasibility Documentation - Registry Based Study Feasibility Assessment _ 11-Jan-2024_Redacted.pdf

05.01.0301 Feasibility Documentation - Registry Evaluation and Quality Standards Tool (REQueST) _ 10-Sep-2023_Redacted.pdf