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

Not Applicable

Recruiting

• Conditions: Transthyretin Amyloidosis With Cardiomyopathy
• Interventions: Drug: Nucresiran; Drug: Sterile Normal Saline (0.9% NaCl)

• Registration Number: NCT07052903
• Lead Sponsor: Alnylam Pharmaceuticals

Brief 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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-06-30
• Study First Submitted QC: 2025-06-30
• Status Verified: 2025-07
• Study Start: 2025-07-02
• Study First Posted: 2025-07-08
• Last Update Submitted: 2025-07-11
• Last Update Posted: 2025-07-15
• Primary Completion: 2030-05-28
• Study Completion: 2032-11-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 1250

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Nucresiran	Participants will receive placebo administered subcutaneously (SC) once every 6 months (q6M) during the double-blind (DB) period, followed by nucresiran 300 mg administered SC q6M during the open-label extension (OLE) period.
Placebo	Sterile Normal Saline (0.9% NaCl)	Participants will receive placebo administered subcutaneously (SC) once every 6 months (q6M) during the double-blind (DB) period, followed by nucresiran 300 mg administered SC q6M during the open-label extension (OLE) period.
Nucresiran 300 mg	Nucresiran	Participants will receive nucresiran 300 mg administered SC Q6M during the DB period, followed by nucresiran 300 mg administered SC q6M during the OLE period.

Primary Outcome Measures

Name	Time	Method
Composite outcome of all-cause mortality and recurrent cardiovascular [CV] events (CV hospitalizations and urgent heart failure [HF] visits)	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.

Secondary Outcome Measures

Name	Time	Method
Time to first CV event (CV hospitalizations and urgent HF visits) or all-cause mortality	Baseline to end of double-blind period (estimated 32 months, maximum 5 years)
All-cause mortality	Baseline to end of double-blind period (estimated 32 months, maximum 5 years)
Recurrent CV events (CV hospitalizations and urgent HF visits)	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)	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.

Trial Locations

Locations (1)

Clinical Trial Site; 🇺🇸 Boston, Massachusetts, United States