Clinical Trial
Study Details
Status
RECRUITING
Study ID #
NCT07052903
Study Start
Primary Completion
Study Completion
Enrollment
1250
Study Type
Interventional
Phase
Phase 3
Interventions
Sterile Normal Saline (0.9% NaCl)

Sterile Normal Saline (0.9% NaCl) administered SC once q6M

Primary Outcomes
Measure
Composite outcome of all-cause mortality and recurrent cardiovascular [CV] events (CV hospitalizations and urgent heart failure [HF] visits)
Description

All-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits) will be compared between treatment groups using an Andersen-Gill model.

Timeframe
Baseline to end of double-blind period (estimated 32 months, maximum 5 years)
Secondary Outcomes
Measure
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)
Measure
All-cause mortality
Timeframe
Baseline to end of double-blind period (estimated 32 months, maximum 5 years)
Measure
Recurrent CV events (CV hospitalizations and urgent HF visits)
Timeframe
Baseline to end of double-blind period (estimated 32 months, maximum 5 years)
Measure
Change from baseline in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS)
Description

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.

Timeframe
Baseline to Month 30
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

Conditions
Transthyretin Amyloidosis With Cardiomyopathy
Study Contact
Name
Clinical Trial Information Line
Role
Contact
Phone
1-877-ALNYLAM
Email
clinicaltrials@alnylam.com
Name
Clinical Trial Information Line
Role
Contact
Phone
1-877-256-9526
Email
clinicaltrials@alnylam.com
Locations
Facility

Clinical Trial Site
Washington D.C., DC 20010
United States

Facility

Clinical Trial Site
Brandon, FL 33511
United States

Facility

Clinical Trial Site
Cumming, GA 30041
United States

Facility

Clinical Trial Site
Gainesville, GA 30501
United States

Facility

Clinical Trial Site
Boston, MA 02118
United States

Facility

Clinical Trial Site
Philadelphia, PA 19104
United States

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 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.

Exclusion Criteria

* 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

Eligibility Minimum Age
18 Years
Eligibility Maximum Age
85 Years
Sexes Eligible for Study
ALL
Eligibility Std Ages
Adult
Older Adult