Health TrialsbyHealthPanel

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

ConditionTransthyretin Amyloidosis With Cardiomyopathy
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18-85
SponsorAlnylam Pharmaceuticals

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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/1.73m² at screening.

Has received prior or currently receiving TTR-lowering therapy

Trial design

Design model

Parallel

Treatments tested in this trial

  • Nucresiran

    Drug

    Nucresiran 300 mg administered SC q6M

  • Sterile Normal Saline (0.9% NaCl)

    Drug

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

Treatment groups

1,750 Participants
are divided into 2 treatment groups
Group A: PlaceboPlacebo comparator 2 interventions
Group B: Nucresiran 300 mgExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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

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 frame
Baseline to end of double-blind period (estimated 32 months, maximum 5 years)

Secondary outcomes

1

Time to first CV event (CV hospitalizations and urgent HF visits) or all-cause mortality

Time frame
Baseline to end of double-blind period (estimated 32 months, maximum 5 years)
2

All-cause mortality

Time frame
Baseline to end of double-blind period (estimated 32 months, maximum 5 years)
3

Recurrent CV events (CV hospitalizations and urgent HF visits)

Time frame
Baseline to end of double-blind period (estimated 32 months, maximum 5 years)
4

Change from baseline in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS)

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.

Time frame
Baseline to Month 30

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Alnylam Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov
Apply now