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