Prospective Observational Study to Monitor and Assess the Safety of Amvuttra® [Vutrisiran] in a Real-World Cohort of hATTR Amyloidosis Patients

Study type

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

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  AMVUTTRA
• Study drug International non-proprietary name (INN) or common name:
  VUTRISIRAN
• Anatomical Therapeutic Chemical (ATC) code:
  (N07XX18) vutrisiran

  vutrisiran
• Medical condition to be studied:
  Acquired ATTR amyloidosis
• Additional medical condition(s):
  Transthyretin-mediated amyloidosis, Hereditary transthyretin-mediated amyloidosis

Population studied

• Short description of the study population:
  Not yet known, study will enroll hATTR patients, who will be treated per standard of care.
• 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)
• Special population of interest:
  Hepatic impaired

  Pregnant women
• Estimated number of subjects:
  300

Study design details

• Main study objective:
  Primary aim = long-term(>2 years) safety of vutrisiran under real-world conditions including determining & comparing incidence of selected events of interest (clinical consequences of vitamin A deficiency, including delayed symptoms &hypersensitivity reactions) in hATTR amyloidosis participants exposed to vutrisiran as compared to other medications (except patisiran) treating hATTR amyloidosis.
• Outcomes:
  1. Number of Participants With Adverse Events (AEs) due toClinical Consequences of Vitamin A Deficiency, IncludingDelayed Symptoms ; 2. Number of Participants With AEs due to HypersensitivityReactions, Number of Hepatic AEs in Participants With Moderate/Severe Hepatic Impairment; Pregnancy Outcomes (Live/Preterm Births, Spontaneous Abortions, Stillbirths & Elective/Therapeutic Abortions); Infant Outcomes, Congenital Malformations at Birth & Lactation & Infant Follow-up Data Through First Year of Life; Epidemiological &Clinical Characteristics of Patients Treated With Vutrisiran in Real-world
• Data analysis plan:
  Descriptive statistics for continuous variables including the number of observations, mean, standard deviation, median, interquartile range, and minimum/maximum; the associated 95% confidence interval (CI) will be provided, as appropriate. ; Categorical variables will be summarised as frequency and proportion of the relevant population. ; Summary statistics will be presented for the full study population and separately by subgroups (eg, exposure status, liver transplant status, hepatic impairment status, occurrence of specified safety events of interest) where appropriate.