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)

TRITON-CM: Evaluating Nucresiran for Cardiomyopathy

NCT: NCT07052903 · Status: RECRUITING · Phase: Phase 3 · Sponsor: Alnylam Pharmaceuticals · Started: 2025-07-02 · Est. Completion: 2032-11-30

Plain English Summary

TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy is a Phase 3 clinical trial sponsored by Alnylam Pharmaceuticals studying Transthyretin Amyloidosis With Cardiomyopathy. Tests the effectiveness of Nucresiran in reducing heart failure and other cardiovascular events in patients with transthyretin amyloidosis with cardiomyopathy. For patients with a documented diagnosis of transthyretin amyloidosis with cardiomyopathy and a history of heart failure, who meet specific health criteria. Participation involves taking Nucresiran or a placebo every 6 months for up to 5 years, with regular check-ups and monitoring. Alternative treatments include other TTR stabilizers and heart failure management, but this trial focuses on the specific benefits of Nucresiran. The trial aims to enroll 1250 participants.

Official 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

Who Can Participate

Here is what you need to know about eligibility for this trial. Eligible patients have a confirmed diagnosis of transthyretin amyloidosis with cardiomyopathy, a history of heart failure, and specific NT-proBNP levels. Patients cannot have NYHA Class IV heart failure or certain levels of polyneuropathy disability. Age range is not specified, but patients must meet specific health criteria. Health requirements include a minimum NT-proBNP level and no severe kidney impairment. This trial is studying Transthyretin Amyloidosis With Cardiomyopathy, so participants generally need a confirmed diagnosis. The trial is currently accepting new participants.

What They're Measuring

The primary outcome measures the impact of Nucresiran on reducing all-cause mortality and cardiovascular events, which can significantly improve patient quality of life. The specific primary outcome measures are: 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)). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 3, the final and most rigorous stage before seeking FDA approval. Phase 3 trials involve 300-3,000+ patients across multiple sites and compare the new treatment directly against the current standard of care. These pivotal trials generate the evidence needed for regulatory review. About 58% of Phase 3 drugs receive FDA approval. Successful Phase 3 results typically lead to a New Drug Application submission.

Why This Trial Matters

This trial addresses a critical treatment gap for patients with transthyretin amyloidosis with cardiomyopathy, offering a potential new therapy to reduce mortality and cardiovascular events. As a Phase 3 trial, positive results could directly lead to FDA approval, making this treatment available to the broader patient population. This research targets Transthyretin Amyloidosis With Cardiomyopathy, where improved treatment options are needed.

Investor Insight

The large market size and unmet need for effective treatments in transthyretin amyloidosis with cardiomyopathy suggest a high approval probability for Nucresiran. Phase 3 trials have approximately a 50-60% chance of gaining FDA approval if they reach this stage. The large enrollment target of 1250 participants suggests significant investment in this program.

Is This Trial Right for Me?

Ask your doctor if you have a confirmed diagnosis of transthyretin amyloidosis with cardiomyopathy and a history of heart failure. Participation involves regular check-ups and taking Nucresiran or a placebo every 6 months. This trial is currently recruiting participants. The trial is being conducted at 20 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

  • Study Type: INTERVENTIONAL
  • Allocation: RANDOMIZED
  • Model: PARALLEL
  • Masking: QUADRUPLE
  • Enrollment: 1,250 participants

Interventions

  • DRUG: Nucresiran — Nucresiran 300 mg administered SC q6M
  • DRUG: Sterile Normal Saline (0.9% NaCl) — Sterile Normal Saline (0.9% NaCl) administered SC once q6M

Primary Outcomes

  • 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))

Secondary Outcomes

  • 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)

Full 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

Trial Locations

  • Clinical Trial Site, La Jolla, California, United States
  • Clinical Trial Site, Washington D.C., District of Columbia, United States
  • Clinical Trial Site, Brandon, Florida, United States
  • Clinical Trial Site, Atlanta, Georgia, United States
  • Clinical Trial Site, Gainesville, Georgia, United States
  • Clinical Trial Site, Tucker, Georgia, United States
  • Clinical Trial Site, Indianapolis, Indiana, United States
  • Clinical Trial Site, Indianapolis, Indiana, United States
  • Clinical Trial Site, Kansas City, Kansas, United States
  • Clinical Trial Site, Boston, Massachusetts, United States
  • ...and 10 more locations

Frequently Asked Questions

What is clinical trial NCT07052903?

NCT07052903 is a Phase 3 INTERVENTIONAL study titled "TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy." It is currently recruiting and is sponsored by Alnylam Pharmaceuticals. The trial targets enrollment of 1250 participants.

What conditions does NCT07052903 study?

This trial investigates treatments for Transthyretin Amyloidosis With Cardiomyopathy. The primary condition under study is Transthyretin Amyloidosis With Cardiomyopathy.

What treatments are being tested in NCT07052903?

The interventions being studied include: Nucresiran (DRUG), Sterile Normal Saline (0.9% NaCl) (DRUG). Nucresiran 300 mg administered SC q6M

What does Phase 3 mean for NCT07052903?

Phase 3 trials are large-scale studies involving 300-3,000+ patients that compare the new treatment against existing standard treatments. Positive Phase 3 results are typically required for FDA approval.

What is the current status of NCT07052903?

This trial is currently "Recruiting." It started on 2025-07-02. The estimated completion date is 2032-11-30.

Who is sponsoring NCT07052903?

NCT07052903 is sponsored by Alnylam Pharmaceuticals. The sponsor is responsible for funding, designing, and overseeing the clinical trial.

How many people can participate in NCT07052903?

The trial aims to enroll 1250 participants. The trial is currently recruiting and accepting new participants.

How is NCT07052903 designed?

This is a interventional study, uses randomized allocation, follows a parallel design, employs quadruple masking. Masking means some participants and/or investigators do not know which treatment group a participant is in, which helps reduce bias.

What are the primary outcomes being measured in NCT07052903?

The primary outcome measures are: 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)). These are the main endpoints researchers use to determine whether the treatment is effective.

Where is NCT07052903 being conducted?

This trial is being conducted at 20 sites, including La Jolla, California; Washington D.C., District of Columbia; Brandon, Florida; Atlanta, Georgia and 16 more sites (United States).

Where can I find official information about NCT07052903?

The official record for NCT07052903 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT07052903. This government database provides the most up-to-date and detailed information about the trial.

What is NCT07052903 testing in simple terms?

Tests the effectiveness of Nucresiran in reducing heart failure and other cardiovascular events in patients with transthyretin amyloidosis with cardiomyopathy. For patients with a documented diagnosis of transthyretin amyloidosis with cardiomyopathy and a history of heart failure, who meet specific health criteria.

Why is this trial significant?

This trial addresses a critical treatment gap for patients with transthyretin amyloidosis with cardiomyopathy, offering a potential new therapy to reduce mortality and cardiovascular events. As a Phase 3 trial, positive results could lead directly to regulatory approval and new treatment options for patients.

What are the potential risks of participating in NCT07052903?

Key risks include potential side effects from the medication, such as injection site reactions and flu-like symptoms. Monitor for any changes in heart function and report any new symptoms to your healthcare provider. As with any clinical trial, participants are closely monitored and can withdraw at any time.

Should I consider participating in NCT07052903?

Ask your doctor if you have a confirmed diagnosis of transthyretin amyloidosis with cardiomyopathy and a history of heart failure. Participation involves regular check-ups and taking Nucresiran or a placebo every 6 months. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.

What does NCT07052903 signal from an investment perspective?

The large market size and unmet need for effective treatments in transthyretin amyloidosis with cardiomyopathy suggest a high approval probability for Nucresiran. This is a Phase 3 trial, which is the final pivotal stage before potential regulatory submission.

What happens if the treatment in this trial doesn't work?

Participation involves taking Nucresiran or a placebo every 6 months for up to 5 years, with regular check-ups and monitoring. Participants in clinical trials always have the right to withdraw and pursue alternative treatments. The study team will help transition patients to other available options.

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This analysis is AI-generated and does not constitute medical advice. Always consult your healthcare provider before making decisions about clinical trial participation.