TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
- Summary
- The purpose of this study is to: * Evaluate the efficacy of nucresiran compared to placebo on reducing all-cause mortality and cardiovascular (CV) events * Evaluate the efficacy of nucresiran compared to placebo on additional assessments of CV events and/or death * Evaluate the efficacy of nucresiran compared to placebo on patient-reported health status and health-related quality of life
- Short title
- TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
- Official title
- TRITON-CM: A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Transthyretin-Mediated Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
- Control arm
- Placebo
- Registry reports results
- Listing at source
- Listed
- Recruitment status
- Recruiting
- Phase
- Phase 3
- Study type
- Interventional
- Completion date
- 2032-11-30
- First posted
- 2025-07-08
- Registry last updated
- 2026-08-14
- Primary completion date
- 2030-05-28
- Start date
- 2025-07-02
- Participants
- 1,750
- Participant count basis
- Estimated
- Planned participants
- 1,750
- Protocol amendments
- Number of sites
- Data monitoring committee
- Registry sponsor category
- Industry
- Lead sponsor
- Alnylam Pharmaceuticals
- Source registry
- ClinicalTrials.gov
- Registry ID
- NCT07052903
- ClinicalTrials.gov ID
- NCT07052903
- Registry record
- Open registry record
- Allocation
- Randomised
- Model
- Parallel groups
- Purpose
- Treatment
- Masking
- Quadruple masking
- Masked roles
- Participants, Care providers, Investigators, Outcome assessors
Composite outcome of all-cause mortality and recurrent cardiovascular [CV] events (CV hospitalizations and urgent heart failure [HF] visits)
Timeframe: Baseline to end of double-blind period (estimated 32 months, maximum 5 years)
All-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits) will be compared between treatment groups using an Andersen-Gill model.
Time to first CV event (CV hospitalizations and urgent HF visits) or all-cause mortality
Timeframe: Baseline to end of double-blind period (estimated 32 months, maximum 5 years)
All-cause mortality
Timeframe: Baseline to end of double-blind period (estimated 32 months, maximum 5 years)
Recurrent CV events (CV hospitalizations and urgent HF visits)
Timeframe: Baseline to end of double-blind period (estimated 32 months, maximum 5 years)
Change from baseline in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS)
Timeframe: Baseline to Month 30
The KCCQ is a 23-item self-administered questionnaire quantifying 6 domains (symptoms, physical function, quality of life, social limitation, self-efficacy, and symptom stability) and 2 summary scores (clinical and overall summary \[OS\]). Scores are transformed to a range of 0-100, in which higher scores reflect better health status.
- Sex
- All
- Age groups
- Adults, Older adults
- Minimum age
- 18 Years
- Maximum age
- 85 Years
- Healthy volunteers
- No
- 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 or signs and symptoms that require treatment with a diuretic.
- 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.
- Has 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
- Clinical Trial SiteLa Jolla, United StatesRecruiting
- Clinical Trial SiteLaguna Hills, United StatesRecruiting
- Clinical Trial SiteStanford, United StatesRecruiting
- Clinical Trial SiteNorwich, United StatesRecruiting
- Clinical Trial SiteWashington D.C., United StatesRecruiting
- Clinical Trial SiteBrandon, United StatesRecruiting
- Clinical Trial SiteMiami, United StatesRecruiting
- Clinical Trial SiteAtlanta, United StatesRecruiting
- Clinical Trial SiteGainesville, United StatesRecruiting
- Clinical Trial SiteTucker, United StatesRecruiting
- Clinical Trial SiteChicago, United StatesRecruiting
- Clinical Trial SiteEvanston, United StatesRecruiting
- Clinical Trial SiteIndianapolis, United StatesRecruiting
- Clinical Trial SiteIndianapolis, United StatesRecruiting
- Clinical Trial SiteKansas City, United StatesRecruiting
- Clinical Trial SiteBaltimore, United StatesRecruiting
- Clinical Trial SiteBoston, United StatesRecruiting
- Clinical Trial SiteBoston, United StatesRecruiting
- Clinical Trial SiteDetroit, United StatesRecruiting
- Clinical Trial SiteRochester, United StatesRecruiting
- Clinical Trial SiteSt Louis, United StatesRecruiting
- Clinical Trial SiteManhasset, United StatesRecruiting
- Clinical Trial SiteNew York, United StatesRecruiting
- Clinical Trial SiteNew York, United StatesRecruiting
- Clinical Trial SiteNew York, United StatesRecruiting
And 226 more, listed on the interactive page.
Other registry identifiers
- 2024-519917-72-00