Vutrisiran for Amyloid Cardiomyopathy

Enrolling by invitation at 73 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Alnylam Pharmaceuticals

Must be taking: Vutrisiran, Patisiran

Disqualifiers: Other investigational studies, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 3

Duration: 36 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a drug called vutrisiran to determine its safety and effectiveness for individuals with amyloid cardiomyopathy, a type of heart disease caused by protein buildup. The trial focuses on patients who have already used vutrisiran or switched from a similar treatment, patisiran. It seeks participants from earlier vutrisiran or patisiran studies who have not discontinued treatment. As a Phase 3 trial, this represents the final step before FDA approval, providing participants an opportunity to contribute to the potential availability of a new treatment.

Do I need to stop my current medications to join the trial?

The trial protocol does not specify whether you need to stop taking your current medications. However, it seems that participants are either continuing or switching from specific related treatments, so it's best to discuss your current medications with the study team.

Is there any evidence suggesting that vutrisiran is likely to be safe for humans?

A previous study administered vutrisiran to many patients with ATTR amyloidosis for up to 58 months. The treatment was generally well-tolerated, with most participants not experiencing serious side effects. Another study showed that after one year, the safety results aligned with earlier findings, indicating no new or major safety issues over time. This suggests that vutrisiran is safe for long-term use. However, as with any treatment, some individuals might experience mild side effects. It is important to discuss any concerns with a doctor. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Vutrisiran is unique because it offers a new approach to treating amyloid cardiomyopathy by using RNA interference (RNAi) technology. Unlike traditional treatments like tafamidis, which work by stabilizing transthyretin (TTR) proteins, vutrisiran targets the TTR gene itself, reducing the production of amyloid proteins at the source. Additionally, it's administered subcutaneously once every three months, offering a more convenient dosing schedule compared to current daily treatments. Researchers are excited about vutrisiran because it has the potential to improve patient outcomes with fewer side effects and less frequent dosing.

What evidence suggests that vutrisiran might be an effective treatment for amyloid cardiomyopathy?

Research has shown that vutrisiran works well for people with amyloid cardiomyopathy (ATTR-CM). Studies have found that vutrisiran improves the heart's structure and function. It also reduces the risk of death and heart-related problems compared to a placebo. Patients taking vutrisiran experienced fewer hospital visits for heart issues. Additionally, vutrisiran helped more patients maintain or improve their physical abilities and quality of life over 30 months compared to a placebo. ^{4 5 6 7 8}

Who Is on the Research Team?

Medical Director

Principal Investigator

Alnylam Pharmaceuticals

Are You a Good Fit for This Trial?

This trial is for patients with a heart condition called Transthyretin Amyloidosis with Cardiomyopathy. It's not specified, but typically participants should be adults who meet certain health standards and have the specific medical condition being studied.

Inclusion Criteria

Patients who are participating in the patisiran parent studies ALN-TTR02-011 or ALN-TTR02-014 or who have completed the 24-month OLE Period in the vutrisiran parent study ALN-TTRSC02-003

Exclusion Criteria

Has permanently discontinued study drug administration while participating in the parent studies ALN-TTR02-011, ALN-TTR02-014, or ALN-TTRSC02-003

Future or current participation in another investigational device or drug study, scheduled to occur during this study

Has other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive vutrisiran 25 mg administered subcutaneously once every 3 months

36 months

Visits every 3 months for treatment administration

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue on extended use of vutrisiran or switch from patisiran

36 months

What Are the Treatments Tested in This Trial?

Interventions

• Vutrisiran

Trial Overview The study is testing Vutrisiran, a medication aimed at treating Transthyretin Amyloidosis with Cardiomyopathy. The trial will assess its safety and effectiveness, including how it affects the body over an extended period or when switching from another drug, patisiran.

How Is the Trial Designed?

Group I: Vutrisiran 25 mg Experimental Treatment 1 Intervention

Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M).

Who Is Running the Clinical Trial?

Alnylam Pharmaceuticals

Lead Sponsor

Trials

81

Recruited

16,100+

Dr. Yvonne Greenstreet

Alnylam Pharmaceuticals

Chief Executive Officer since 2021

MD from the University of Leeds, MBA from INSEAD

Dr. Pushkal Garg

Alnylam Pharmaceuticals

Chief Medical Officer since 2016

MD from Columbia University

Citations

1. nature.com nature.com/articles/s41591-025-03851-z

Effects of vutrisiran on cardiac structure and function in ... In conclusion, in this current population of patients with ATTR-CM, vutrisiran had beneficial impact across all domains of cardiac structure, ...

2. jacc.org jacc.org/doi/10.1016/j.jacc.2025.04.008

Vutrisiran Improves Survival and Reduces Cardiovascular ... Vutrisiran also reduced the risk of a composite of CV mortality and CV events (HR: 0.72; 95% CI: 0.55-0.94), and lowered rates of CV hospitalizations (rate ...

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39213194/

Vutrisiran in Patients with Transthyretin Amyloidosis ... Among patients with ATTR-CM, treatment with vutrisiran led to a lower risk of death from any cause and cardiovascular events than placebo ...

4. nejm.org nejm.org/doi/abs/10.1056/NEJMoa2409134

Vutrisiran in Patients with Transthyretin Amyloidosis ... Among patients with ATTR-CM, treatment with vutrisiran led to a lower risk of death from any cause and cardiovascular events than placebo and ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S0735109725057961

Impact of Vutrisiran on Functional Capacity and Quality ... Vutrisiran maintained or improved functional capacity, health status, and QOL in more patients with ATTR-CM vs placebo over 30 months.

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40848527/

Vutrisiran in Transthyretin Amyloidosis: A Pooled Safety ... In a broad population of patients with ATTR who were treated for up to 58 months, vutrisiran was well tolerated and had an acceptable safety ...

7. jacc.org jacc.org/doi/10.1016/j.jacadv.2025.102066

Vutrisiran in Transthyretin Amyloidosis: A Pooled Safety ... This pooled safety analysis aimed to evaluate the safety of vutrisiran in a large population of patients with hereditary ATTR and wild-type ATTR ...

8. investors.alnylam.com investors.alnylam.com/press-release?id=29256

Aug 31, 2025 | New Data From Landmark HELIOS-B Phase 3 ... Safety data for vutrisiran treatment through one year of the OLE period were consistent with that reported for the double-blind period. The ...