A Study of Vutrisiran (ALN-TTRSC02) in Patients with Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)


About this study

The purpose of this study is to evaluate the effectiveness and safety of vutrisiran (ALN-TTRSC02) in patients with hereditary transthyretin amyloidosis (hATTR amyloidosis). Participants will receive vutrisiran or the reference comparator patisiran during the Treatment Period followed by a Treatment Extension Period during which all participants in the patisiran group will switch to vutrisiran. This study will use the placebo arm of the APOLLO study (NCT01960348) as an external comparator for the co-primary and most other efficacy endpoints.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Male or female of 18 to 85 years of age (inclusive).
  • Has a diagnosis of hATTR amyloidosis with transthyretin (TTR) mutation. 
  • Has adequate neurologic impairment score (NIS).
  • Has adequate polyneuropathy disability (PND) score.
  • Has adequate Karnofsky Performance Status (KPS).

Exclusion Criteria:

  • Had a prior liver transplant or is likely to undergo liver transplantation during the study.
  • Has known other (non-hATTR) forms of amyloidosis or leptomeningeal amyloidosis.
  • Has New York Heart Association heart failure classification >2.
  • Clinically significant liver function test abnormalities.
  • Has known human immunodeficiency virus (HIV), hepatitis C virus (HCV) or hepatitis B virus (HBV) infection.
  • Received an experimental drug within 30 days of dosing.
  • Received prior TTR-lowering treatment.
  • Has other known causes of neuropathy.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status

Jacksonville, Fla.

Mayo Clinic principal investigator

Elizabeth Mauricio, M.D.

Closed for enrollment

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