A new clinical trial evaluating the efficacy of nucresiran in treating transthyretin amyloid cardiomyopathy (ATTR-CM) is set to launch soon, according to study information published on clinicaltrials.gov.
Nucresiran is an investigational gene-silencing drug that operates through a mechanism known as RNA interference (RNAi). Genes contain instructions for making proteins, and these instructions are carried by messenger RNA (mRNA). RNAi-based therapies like nucresiran target and degrade specific mRNA molecules, thereby preventing the synthesis of disease-causing proteins.
Read more about ATTR-CM therapies
In ATTR-CM, defective transthyretin (TTR) is produced in the liver and accumulates in the heart, leading to cardiac dysfunction. Nucresiran inhibits TTR production at the mRNA level. According to its developer, the drug offers a more sustained reduction in TTR levels, allowing for less frequent dosing compared to other RNAi therapies.
The TRITON-CM trial will evaluate the impact of nucresiran on all-cause and cardiovascular mortality, the incidence of cardiovascular events and hospitalizations and patient-reported health status and quality of life.
The randomized, placebo-controlled study will enroll over 1,200 patients with confirmed ATTR-CM. Participants will be assigned to receive either 300 mg of nucresiran or a placebo (normal saline), administered subcutaneously every six months. The study is double-blinded, meaning neither the participants nor the researchers will know which treatment each participant is receiving until the study concludes.
The study will compare several specific outcome measures between patients receiving the drug and those receiving placebo, including rates of death from any cause, rate of cardiovascular mortality, rate of cardiovascular events, hospitalization frequency and changes in the Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OS), a validated, 23-item self-administered tool that evaluates physical function, symptoms, social limitation, quality of life, and self-efficacy in patients with cardiomyopathy.
The trial will be conducted at major centers in Cumming, Georgia, and Boston, Massachusetts. The estimated study completion date is November 2032.
Sign up here to get the latest news, perspectives, and information about ATTR-CM sent directly to your inbox. Registration is free and only takes a minute
