BridgeMD
TRITON-PN: A Phase 3 Study to Evaluate the Efficacy and Safety of Nucresiran in Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy
EU CTIS ID: 2025-522544-40-00
What this study is testing
To determine the efficacy of nucresiran in patients with hATTR-PN by evaluating the effect on neurologic impairment
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Male or female age 18 (or age of legal consent) to 85 years, inclusive, at the time of initial informed consent.
- 2. Have a diagnosis of hATTR amyloidosis with polyneuropathy with a documented TTR gene variant
- 3. Have a neuropathy impairment score (NIS) of 5 to 130 (inclusive; this criterion must be met at the Baseline Visit 2)
- 4. Have a Polyneuropathy Disability (PND) score of ≤3b (this criterion must be met at the Baseline Visit 2)
- 5. Have a Karnofsky Performance Status (KPS) of ≥60%Informed Consent
- 6. Patient is able to understand and is willing and able to comply with the study requirements and to provide written informed consent.
You likely can't join if
- 1. All exclusion criteria can be found in the protocol (Section 4.2). 1. 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
- 18. Has uncontrolled clinically significant cardiac arrhythmia or unstable angina
- 19. Has known type 1 diabetes
- 10. Received prior or currently receiving TTR-lowering therapy
- 20. Has had type 2 diabetes mellitus for ≥5 years
- 21. Has untreated hypo- or hyperthyroidism
See the full eligibility criteria
Who can join
- 1. Male or female age 18 (or age of legal consent) to 85 years, inclusive, at the time of initial informed consent.
- 2. Have a diagnosis of hATTR amyloidosis with polyneuropathy with a documented TTR gene variant
- 3. Have a neuropathy impairment score (NIS) of 5 to 130 (inclusive; this criterion must be met at the Baseline Visit 2)
- 4. Have a Polyneuropathy Disability (PND) score of ≤3b (this criterion must be met at the Baseline Visit 2)
- 5. Have a Karnofsky Performance Status (KPS) of ≥60%Informed Consent
- 6. Patient is able to understand and is willing and able to comply with the study requirements and to provide written informed consent.
What rules you out
- 1. All exclusion criteria can be found in the protocol (Section 4.2). 1. 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
- 18. Has uncontrolled clinically significant cardiac arrhythmia or unstable angina
- 19. Has known type 1 diabetes
- 10. Received prior or currently receiving TTR-lowering therapy
- 20. Has had type 2 diabetes mellitus for ≥5 years
- 21. Has untreated hypo- or hyperthyroidism
- 22. Has had a major surgery within the past 3 months or has a major surgery planned during the study through Month 18
- 23. Has an active infection requiring systemic antiviral, antiparasitic, or antimicrobial therapy that will not be completed prior to dosing (Day 1)
- 24. Has other medical conditions or comorbidities which, in the opinion of the Investigator, could interfere with study compliance or data interpretation
- 25. Has a history of multiple drug allergies or history of allergic reactions to any component of or excipient in the study drug
- 26. Has a history of intolerance to SC injection(s)
- 7. Estimated glomerular filtration rate (eGFR) ≤30 mL/min/1.73m2 (calculation will be based on the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] equation; refer to Section 10.1)
- 11. Is currently taking tafamidis, acoramidis, doxycycline, or tauroursodeoxycholic acid; if previously on any of these agents, must have completed a 14-day washout prior to dosing (Day 1)
- 12. Is currently taking diflunisal; if previously on this agent, must have completed a 3-day washout prior to dosing (Day 1)
- 2. Has known other (non-hATTR) forms of amyloidosis or clinical evidence of leptomeningeal amyloidosis
- 3. Has a New York Heart Association (NYHA) heart failure classification >2
- 4. Has any of the following laboratory parameter assessments at screening: a. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2.5 upper limit of normal (ULN) reference range b. Total bilirubin >1.5 ULN (>2 ULN in patients with Gilbert's Syndrome) c. International normalized ratio (INR) >2.0
- 5. Platelet count <50,000/μL
- 6. Absolute neutrophil count <1500 cells/mm³
- 27. Has a life expectancy of <2 years based on Investigator's clinical judgement
- 28. Is not willing to comply with the contraceptive requirements during the study period as described in Section 5.9.1
- 29. Female patient who is pregnant or breastfeeding
- 8. Has vitamin B12 levels below the lower limit of normal
- 30. Unwilling or unable to limit alcohol consumption throughout the course of the study
- 31. History of alcohol use disorder within the last 12 months before screening in the opinion of the Investigator
- 32. History of illicit substance use within the past 5 years that in the opinion of the Investigator would interfere with compliance with study procedures or follow-up visits
- 9. Has known human immunodeficiency virus infection; or evidence of acute or chronic hepatitis C virus or hepatitis B virus infection
- 13. Anticipated to start an approved TTR stabilizing therapy (eg, tafamidis or acoramidis) within the first 18 months following randomization
- 14. Received prior anti-TTR antibody treatment (ie, “TTR depleters”)
- 15. Received an investigational agent within the last 30 days (or 5 half-lives of the investigational drug, whichever is longer), or are currently in follow-up of another clinical study. Any agent that has received health agency authorization (including for emergency use) by local or regional authorities is not considered investigational
- 16. Has other known causes of sensorimotor or autonomic neuropathy (eg, autoimmune disease, monoclonal gammopathy) that the treating physician believes to be contributing to the neuropathy
- 17. Had acute coronary syndrome within the past 3 months
The study team makes the final eligibility decision.
Where it's taking place
- Mexico
- Japan
- Taiwan
- Malaysia
- Brazil
- Argentina
- Canada
- Switzerland
- Australia
- United Kingdom
- Korea, Republic of
- United States
- Turkey
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Mexico; Japan; Taiwan; Malaysia; Brazil; Argentina and 7 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.