TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Launched by ALNYLAM PHARMACEUTICALS · Jun 30, 2025

Keywords

ClinConnect Summary

This clinical trial, called TRITON-CM, is studying a new medicine called nucresiran for people who have a condition called transthyretin amyloidosis with cardiomyopathy. This condition happens when abnormal proteins build up in the heart, making it harder for the heart to work properly. The study aims to see if nucresiran can help reduce the risk of death and serious heart problems compared to a placebo (a treatment with no active medicine). The researchers will also look at how the medicine affects patients’ overall health and quality of life, based on what the patients report.

People eligible for this study are adults diagnosed with transthyretin amyloidosis affecting the heart, including both inherited and non-inherited forms. They must have a history of heart failure, including at least one hospital stay because of it, and meet certain blood test levels related to heart stress. Participants may already be taking other heart or amyloidosis medicines. However, those with very advanced heart failure or severe nerve problems, or who have kidney problems or have previously used similar treatments, cannot join. If you take part, you can expect to be closely monitored and receive either the new medicine or a placebo, with the goal of understanding how well nucresiran works and how it might improve heart health and daily living.

Gender

ALL

Eligibility criteria

• • Inclusion Criteria
• • Has documented diagnosis of ATTR amyloidosis with cardiomyopathy including those with hereditary ATTR (hATTR) or wild-type ATTR (wATTR). amyloidosis.
• • Has medical history of heart failure (HF) with at least 1 prior hospitalization for HF.
• • Has screening N-terminal prohormone B-type natriuretic peptide (NT-proBNP) \>300 ng/L and \<8500 ng/L; In patients with permanent or persistent atrial fibrillation, screening NT-proBNP \>600 ng/L and \<8500 ng/L.
• • Patients may be receiving approved TTR stabilizers for ATTR amyloidosis (eg, tafamidis, acoramidis) and may be receiving background therapy for HF at the discretion of the Investigator.
• • Exclusion Criteria
• • New York Heart Association (NYHA) Class IV HF; or NYHA Class III heart failure AND ATTR Amyloidosis Disease Stage 3.
• • Has a polyneuropathy disability (PND) Score IIIa, IIIb, or IV.
• • Has an estimated glomerular filtration rate eGFR of \<30 mL/min/1.73m\^2 at screening.
• • Has received prior or currently receiving TTR-lowering therapy