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.