Prospective observational study to monitor and assess the safety of Onpattro® patisiran-LNP in a real-world cohort of hATTR amyloidosis patients

Study type

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

  Disease epidemiology

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  PATISIRAN
• Medical condition to be studied:
  Familial amyloidosis

  Hereditary neuropathic amyloidosis

  Amyloidosis

  Acquired ATTR amyloidosis

  Cardiac amyloidosis
• Additional medical condition(s):
  Hereditary transthyretin-mediated (hATTR) amyloidosis

Population studied

• Age groups:
  Preterm newborn infants (0 – 27 days)

  Term newborn infants (0 – 27 days)

  Infants and toddlers (28 days – 23 months)

  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  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:
  300

Study design details

• Main study objective:
  The main objective of this study is to characterise the safety of patisiran-LNP under real-world conditions, including determining and comparing the incidence of selected events (eg, hepatic) in hATTR amyloidosis patients exposed to patisiran-LNP.
• Outcomes:
  The primary outcome of interest is the incidence of selected events of interest. These events include: hepatic events, cardiovascular events, renal events and ocular events, as well as infusion-¬related reactions (including hypersensitivity reactions). ; • The incidence of selected events in sub-populations (eg, patients administered home infusions, patients with prior liver transplant, hepatic impairment, and renal impairment) . ; • Epidemiological and clinical characteristics of hATTR amyloidosis patients, and patients treated with patisiran-LNP in a real-world setting. ; • Pregnancy outcomes and selected infant outcomes.
• Data analysis plan:
  Epidemiological cohort techniques will be used to analyse the occurrence of safety events by exposure status. Incidence rates will be calculated in patient-years with respect to both exposure status and observational time. Relative risk numbers (incidence rate ratios) will be derived for composite endpoints of selected safety events of interest. To account for varying exposure windows and varying observational time, the proportion of patients experiencing an event may also be estimated using time-to-event methodology.