ConTTRibute: A Global Observational Multicenter Long-Term Study of Patients with Transthyretin (TTR)-Mediated Amyloidosis (ATTR amyloidosis)

Study type

• Study topic:
  Disease /health condition
• Study type:
  Non-interventional study

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine, other:
  Onpattro, Amvuttra
• Study drug International non-proprietary name (INN) or common name:
  PATISIRAN

  VUTRISIRAN SODIUM
• Anatomical Therapeutic Chemical (ATC) code:
  (N07XX12) patisiran

  patisiran

  (N07XX18) vutrisiran

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

Population studied

• Short description of the study population:
  Patients with a diagnosis of ATTR amyloidosis, hereditary or wild type, and pre-symptomatic carriers with a known disease-causing TTR variant will be eligible for the study.

Study design details

• Study design:
  This is a prospective, global, multicenter, long-term observational study designed to document the clinical outcomes of patients with hATTR amyloidosis, or wtATTR amyloidosis, and the safety of patisiran and vutrisiran when used in patients with hATTR amyloidosis.
• Outcomes:
  The outcomes of interest are incidence of adverse events (AEs), selected events of interest, health care provider reported outcomes and patient-reported outcomes.
• Data analysis plan:
  No statistical inferences will be drawn from the study. Descriptive statistics will be used to meet the objectives of the study.