TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
Key facts
- Where
- Florence, Messina, Milan, Padova, Pavia, and Rome, ItalySee the 6 sites
- Phase
- Phase 3
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 85 years old?
Source: NCT07223203, who can take part
Where in Italy
- Azienda Ospedaliero-Universitaria CareggiFlorence, ItalyNo longer taking part
- Azienda Ospedaliera Universitaria Gaetano Martino MessinaMessina, ItalyNo longer taking part
- IRCCS Foundation Istituto Neurologico Carlo BestaMilan, ItalyNo longer taking part
- Azienda Ospedaliera di PadovaPadova, ItalyNo longer taking part
- Fondazione IRCCS Policlinico San MatteoPavia, ItalyNo longer taking part
- Fondazione Policlinico Universitario Agostino Gemelli IRCCSRome, ItalyNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Alnylam Pharmaceuticals
- Trial code
- NCT07223203EU 2025-522544-40-00
- Record updated
- 8 October 2026
- Original description
The purpose of this study is to:Read full original descriptionShow less
TRITON-PN: A Phase 3, Global, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy (hATTR-PN)
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
Inclusion: 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%.
Exclusion: 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 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); Has total bilirubin >1.5 ULN; Has estimated glomerular filtration rate (eGFR) ≤30 mL/min/1.73m\^2; Has known other (non-hATTR) forms of amyloidosis or clinical evidence of leptomeningeal amyloidosis; Has other known causes of sensorimotor or autonomic neuropathy.