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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

Full criteria on ClinicalTrials.gov

Sites (58)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
Clinical Trial Site
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BelgranoArgentinaRecruiting
Clinical Trial Site
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RosarioArgentinaRecruiting
Clinical Trial Site
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DarlinghurstAustraliaRecruiting
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WoolloongabbaAustraliaRecruiting
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Ribeirão PretoBrazilRecruiting
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Rio de JaneiroBrazilRecruiting
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SalvadorBrazilRecruiting
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São PauloBrazilRecruiting
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VancouverCanadaRecruiting
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BernSwitzerlandRecruiting
The Cyprus Foundation For Muscular Dystrophy Research
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Agios DometiosCyprusActive, not recruiting
Charite Universitaetsmedizin Berlin KöR
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BerlinGermanyActive, not recruiting
Universitaetsklinikum Heidelberg AöR
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HeidelbergGermanyActive, not recruiting
Universitaet Muenster
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MuensterGermanyActive, not recruiting
Hospital Universitari Vall d'Hebron
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BarcelonaSpainActive, not recruiting
Hospital Universitario Juan Ramon Jimenez
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HuelvaSpainActive, not recruiting
Bellvitge University Hospital
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L'hospitalet De LlobregatSpainActive, not recruiting
Hospital Clínico San Carlos
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MadridSpainActive, not recruiting
Hospital Son Llatzer
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PalmaSpainActive, not recruiting
Assistance Publique Hopitaux De Paris
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Creteil CedexFranceActive, not recruiting
Bicetre Hospital
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Le Kremlin Bicetre CedexFranceActive, not recruiting
Centre Hospitalier Regional De Marseille
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MarseilleFranceActive, not recruiting
Clinical Trial Site
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LondonUnited KingdomRecruiting
General Hospital of Athens - Alexandra
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AthensGreeceActive, not recruiting
University General Hospital of Heraklion
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HeraklionGreeceActive, not recruiting
Tallaght University Hospital
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Dublin 24IrelandActive, not recruiting
Azienda Ospedaliero-Universitaria Careggi
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FlorenceItalyActive, not recruiting
Azienda Ospedaliera Universitaria Gaetano Martino Messina
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MessinaItalyActive, not recruiting
IRCCS Foundation Istituto Neurologico Carlo Besta
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MilanItalyActive, not recruiting
Azienda Ospedaliera di Padova
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PadovaItalyActive, not recruiting
Fondazione IRCCS Policlinico San Matteo
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PaviaItalyActive, not recruiting
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
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RomeItalyActive, not recruiting
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Bunkyo CJapanRecruiting
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KumamotoJapanRecruiting
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Matsumoto-shiJapanRecruiting
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SuitaJapanRecruiting
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SeoulSouth KoreaRecruiting
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GuadalajaraMexicoRecruiting
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TlalpanMexicoRecruiting
Clinical Trial Site
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Kuala LumpurMalaysiaRecruiting
Unidade Local de Saúde de Santa Maria - Hopital de Santa Maria
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LisboaPortugalActive, not recruiting
Unidade Local de Saúde de Santo António - Hospital de Santo António
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PortoPortugalActive, not recruiting
Region Stockholm – SLSO
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StockholmSwedenRecruiting
Region Vaesterbotten
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UmeaSwedenRecruiting
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TaipeiTaiwanRecruiting
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Taoyuan CityTaiwanRecruiting
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AuroraUnited StatesRecruiting
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BaltimoreUnited StatesRecruiting
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BostonUnited StatesRecruiting
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Chapel HillUnited StatesRecruiting
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ChicagoUnited StatesRecruiting
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DallasUnited StatesRecruiting
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HoustonUnited StatesRecruiting
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NashvilleUnited StatesRecruiting
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New YorkUnited StatesRecruiting
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RochesterUnited StatesRecruiting
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IstanbulTurkey (Türkiye)Recruiting
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IzmirTurkey (Türkiye)Recruiting

This page is information, not medical advice. Discuss any trial with your doctor.