NCT07223203RecruitingPhase 3
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
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)
Source: ClinicalTrials.gov · Last updated 8 Oct 2026
Brief summary (registry, in English)
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
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 125 (estimated)
- First submitted
- 29 Oct 2025
- Start date
- 3 Feb 2026
- Primary completion date
- 27 Dec 2027
- Lead sponsor
- Alnylam Pharmaceuticals
- Diseases
- Amyloidosis
- NCT number
- NCT07223203
- EU CT number
- 2025-522544-40-00
What you commit to
- Study type: Interventional · Phase 3
- Planned participants: 125
- From 3 Feb 2026 to the primary completion date, 27 Dec 2027
- 58 sites in 22 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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
Other illness
- Has a New York Heart Association (NYHA) heart failure classification \>2
Lab values
- 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
Other
- Has known other (non-hATTR) forms of amyloidosis or clinical evidence of leptomeningeal amyloidosis
- Has other known causes of sensorimotor or autonomic neuropathy
Who the study is looking for
- 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%
Age in the registry: 18 years to 85 years · Sex: All · Does not accept healthy volunteers
Sites (58)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Clinical Trial Site Coordinator of this site? Confirm its status | Belgrano | Argentina | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Rosario | Argentina | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Darlinghurst | Australia | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Woolloongabba | Australia | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Ribeirão Preto | Brazil | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Rio de Janeiro | Brazil | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Salvador | Brazil | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | São Paulo | Brazil | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Vancouver | Canada | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Bern | Switzerland | Recruiting |
| The Cyprus Foundation For Muscular Dystrophy Research Coordinator of this site? Confirm its status | Agios Dometios | Cyprus | Active, not recruiting |
| Charite Universitaetsmedizin Berlin KöR Coordinator of this site? Confirm its status | Berlin | Germany | Active, not recruiting |
| Universitaetsklinikum Heidelberg AöR Coordinator of this site? Confirm its status | Heidelberg | Germany | Active, not recruiting |
| Universitaet Muenster Coordinator of this site? Confirm its status | Muenster | Germany | Active, not recruiting |
| Hospital Universitari Vall d'Hebron Coordinator of this site? Confirm its status | Barcelona | Spain | Active, not recruiting |
| Hospital Universitario Juan Ramon Jimenez Coordinator of this site? Confirm its status | Huelva | Spain | Active, not recruiting |
| Bellvitge University Hospital Coordinator of this site? Confirm its status | L'hospitalet De Llobregat | Spain | Active, not recruiting |
| Hospital Clínico San Carlos Coordinator of this site? Confirm its status | Madrid | Spain | Active, not recruiting |
| Hospital Son Llatzer Coordinator of this site? Confirm its status | Palma | Spain | Active, not recruiting |
| Assistance Publique Hopitaux De Paris Coordinator of this site? Confirm its status | Creteil Cedex | France | Active, not recruiting |
| Bicetre Hospital Coordinator of this site? Confirm its status | Le Kremlin Bicetre Cedex | France | Active, not recruiting |
| Centre Hospitalier Regional De Marseille Coordinator of this site? Confirm its status | Marseille | France | Active, not recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | London | United Kingdom | Recruiting |
| General Hospital of Athens - Alexandra Coordinator of this site? Confirm its status | Athens | Greece | Active, not recruiting |
| University General Hospital of Heraklion Coordinator of this site? Confirm its status | Heraklion | Greece | Active, not recruiting |
| Tallaght University Hospital Coordinator of this site? Confirm its status | Dublin 24 | Ireland | Active, not recruiting |
| Azienda Ospedaliero-Universitaria Careggi Coordinator of this site? Confirm its status | Florence | Italy | Active, not recruiting |
| Azienda Ospedaliera Universitaria Gaetano Martino Messina Coordinator of this site? Confirm its status | Messina | Italy | Active, not recruiting |
| IRCCS Foundation Istituto Neurologico Carlo Besta Coordinator of this site? Confirm its status | Milan | Italy | Active, not recruiting |
| Azienda Ospedaliera di Padova Coordinator of this site? Confirm its status | Padova | Italy | Active, not recruiting |
| Fondazione IRCCS Policlinico San Matteo Coordinator of this site? Confirm its status | Pavia | Italy | Active, not recruiting |
| Fondazione Policlinico Universitario Agostino Gemelli IRCCS Coordinator of this site? Confirm its status | Rome | Italy | Active, not recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Bunkyo C | Japan | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Kumamoto | Japan | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Matsumoto-shi | Japan | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Suita | Japan | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Seoul | South Korea | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Guadalajara | Mexico | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Tlalpan | Mexico | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Kuala Lumpur | Malaysia | Recruiting |
| Unidade Local de Saúde de Santa Maria - Hopital de Santa Maria Coordinator of this site? Confirm its status | Lisboa | Portugal | Active, not recruiting |
| Unidade Local de Saúde de Santo António - Hospital de Santo António Coordinator of this site? Confirm its status | Porto | Portugal | Active, not recruiting |
| Region Stockholm – SLSO Coordinator of this site? Confirm its status | Stockholm | Sweden | Recruiting |
| Region Vaesterbotten Coordinator of this site? Confirm its status | Umea | Sweden | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Taipei | Taiwan | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Taoyuan City | Taiwan | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Aurora | United States | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Baltimore | United States | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Boston | United States | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Chapel Hill | United States | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Chicago | United States | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Dallas | United States | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Houston | United States | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Nashville | United States | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | New York | United States | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Rochester | United States | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Istanbul | Turkey (Türkiye) | Recruiting |
| Clinical Trial Site Coordinator of this site? Confirm its status | Izmir | Turkey (Türkiye) | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.