TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy

Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18-85
SponsorAlnylam Pharmaceuticals

About this trial

The purpose of this study is to:

* Determine the efficacy of nucresiran in patients with hATTR-PN by evaluating the effect on neurologic impairment, quality of life, nutritional status, disability, and gait speed * Demonstrate superiority of nucresiran compared to in-study vutrisiran with respect to serum transthyretin (TTR) levels

Eligibility criteria

Qualifiers

Has documented diagnosis of hATTR-PN

Has a diagnosis of hATTR amyloidosis with polyneuropathy with a documented TTR gene variant

Has a neuropathy impairment score (NIS) of 5 to 130 (inclusive)

Has a Karnofsky Performance Status (KPS) of ≥60%

Disqualifiers

Has had a liver transplant or is likely, in the opinion of the Investigator, to undergo liver transplantation during the Treatment Period of the study

Has known other (non-hATTR) forms of amyloidosis or clinical evidence of leptomeningeal amyloidosis

Has a New York Heart Association (NYHA) heart failure classification >2

Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2.5 upper limit of normal (ULN)

Trial design

Treatments tested in this trial

  • Nucresiran
  • Vutrisiran

Treatment groups

125 Participants
are divided into 2 treatment groups

Sponsors and collaborators