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Taking peoplePhase 3

Tests nucresiran against vutrisiran in hereditary transthyretin amyloidosis with polyneuropathy

Tests nucresiran, compared with vutrisiran, in adults with hereditary transthyretin amyloidosis nerve damage.

Key facts

Who runs it
Alnylam Pharmaceuticals
Where
Agios Dometios, CyprusSee the siteAlso in 21 other countries, 57 sites
Registry title
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
Status
Recruiting
Phase
Phase 3
People taking part
125 people, planned
Started
3 Feb 2026
Expected to end
12 Jun 2031
Type of study
Interventional, 2 groupsOne group gets an existing treatment, for comparison.
Treatments tested
  • Nucresiran
  • Vutrisiran
Record updated
8 October 2026

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for adults with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN), a nerve disease caused by a gene change. It compares nucresiran with vutrisiran.

Age
18 to 85 years
Sex
All sexes
Disease stage
Documented hATTR amyloidosis with polyneuropathy. Neuropathy impairment score (NIS) of 5 to 130 (inclusive). Karnofsky Performance Status (KPS) of 60% or more.
Earlier treatment
Has not had a liver transplant, and is not likely to need one during the treatment period.
Biomarker or test result
A documented TTR gene variant.

Main reasons for not taking part

  • Liver transplant, or one likely during the study
  • Other (non-hATTR) forms of amyloidosis, or amyloid in the lining of the brain (leptomeningeal amyloidosis)
  • Heart failure with NYHA class above 2
  • Liver tests (ALT or AST) above 2.5 times the upper limit of normal, or total bilirubin above 1.5 times
  • Kidney function (eGFR) of 30 mL/min/1.73m^2 or lower

The registry criteria, word for word

Show all 11 criteriaShow less

Who can take part

  • 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%

Who cannot take part

  • 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

Check if you might fit

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?

Where in Cyprus

Also in 21 other countries, 57 sites

  • The Cyprus Foundation For Muscular Dystrophy ResearchAgios Dometios, CyprusNo longer taking part

About the trial

What the trial wants to find out

The purpose of this study is to:

Read full original descriptionShow less

* 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

The 2 groups in the trial

  • Nucresiran 300 mgTreatment being tested

    Patients will be administered nucresiran 300 mg subcutaneously (SC) once every 6 months (q6M) during the Treatment Period and Treatment Extension Period

    Treatment: Nucresiran

  • Vutrisiran 25 mg followed by Nucresiran 300 mgExisting treatment, for comparison

    Patients will be administered vutrisiran 25 mg SC every 3 months (q3M) during the Treatment Period followed by nucresiran 300 mg SC q6M during the Treatment Extension Period

    Treatment: Nucresiran and Vutrisiran

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
Official title
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)
Plain-language title
Tests nucresiran against vutrisiran in hereditary transthyretin amyloidosis with polyneuropathyGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 12 November 2026.
Fit questions
Built from NCT07223203, who can take part
Record updated
8 October 2026