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TRITON-PN: A Study of Nucresiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy

This study is testing a drug called nucresiran for people with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). This condition is caused by a genetic change that leads to a protein called transthyretin (TTR) building up in nerves and other organs. Researchers want to see how well nucresiran works to improve nerve function, quality of life, and other measures, compared to an existing treatment called vutrisiran. You may be able to join if you are 18 to 85 years old, have a confirmed diagnosis of hATTR-PN with a specific gene change, and meet certain criteria for nerve impairment and physical ability. The study aims to enroll 125 participants, but its current status is unclear.

Study design
This is an interventional study comparing nucresiran to vutrisiran. It plans to enroll 125 participants.
What's involved
You would receive nucresiran every six months or vutrisiran every three months, both given as a shot under the skin. Your nerve function will be measured at the start and at Month 9.
Compensation
Not stated in the trial record.
Follow-up
Your nerve function will be measured at Month 9 after starting the study.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07223203

TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy

Recruiting
PHASE3Ages 18–85InterventionalTreatment
Alnylam Pharmaceuticals
~125 participants
Updated 2026-08-14 on ClinicalTrials.gov
What's tested:NucresiranVutrisiran

At a glance

Recruiting sites
47 of 48 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Change from Baseline in the Modified Neuropathy Impairment Score +7 (mNIS+7) Compared to the External Placebo Group from the APOLLO Study (NCT01960348) at Month 9
Measured over Baseline and Month 9

Conditions

Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy
hATTR-PN

Explore related trials

Hereditary Transthyretin Amyloidosis with PolyneuropathyTTR Gene

Where it's being run

48 sites across 25 states
Italy4
Japan4
Spain4
France3
Germany3
Taiwan3
Texas2
Australia2

Study leadership

  • Medical Director · STUDY_DIRECTOR · Alnylam Pharmaceuticals

Who to contact

Clinical Trial Information Line
[email protected]
1-877-ALNYLAM
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

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 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)
Has total bilirubin \>1.5 ULN
Has estimated glomerular filtration rate (eGFR) ≤30 mL/min/1.73m\^2
Has other known causes of sensorimotor or autonomic neuropathy

What this trial measures

  • Change from Baseline in the Modified Neuropathy Impairment Score +7 (mNIS+7) Compared to the External Placebo Group from the APOLLO Study (NCT01960348) at Month 9Baseline and Month 9

    The mNIS+7 is a composite score that measures neurologic impairment which includes the following components: physical exam of lower limbs, upper limbs and cranial nerves to assess motor strength/weakness and deep tendon reflexes, electrophysiologic measurement of large nerve fiber function, sensory testing and postural blood pressure. The mNIS+7 is scored from 0 (no impairment) to 304 points (maximum impairment). A higher score indicates a worse outcome.