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RNAi Therapeutics

Vutrisiran for Transthyretin Amyloidosis with Cardiomyopathy

Phase 3
Waitlist Available
Research Sponsored by Alnylam Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Has medical history of heart failure (HF) with at least 1 prior hospitalization for HF OR clinical evidence of HF
Has a documented diagnosis of transthyretin (ATTR) amyloidosis with cardiomyopathy, classified as either hereditary ATTR (hATTR) amyloidosis with cardiomyopathy or wild-type ATTR (wtATTR) amyloidosis with cardiomyopathy meeting pre-specified diagnostic criteria
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 30-36 months
Awards & highlights

Study Summary

This trial will test if vutrisiran, given every 3 months, is better than placebo at treating ATTR amyloidosis with heart problems.

Who is the study for?
This trial is for patients with a confirmed diagnosis of transthyretin (ATTR) amyloidosis with cardiomyopathy, which includes inherited or wild-type forms. Participants should have a history of heart failure, evidenced by hospitalization or clinical signs. Those with primary or leptomeningeal amyloidosis, severe polyneuropathy, advanced heart failure, very low kidney function, prior TTR treatments, or other types of cardiomyopathies cannot join.Check my eligibility
What is being tested?
The study tests the effectiveness and safety of Vutrisiran given as an injection under the skin every three months compared to a placebo (saline solution). The goal is to see if Vutrisiran can help patients with ATTR amyloidosis affecting the heart.See study design
What are the potential side effects?
Potential side effects from Vutrisiran may include injection site reactions such as redness and pain, liver issues like elevated liver enzymes indicating damage or inflammation, and possible allergic reactions among others.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been hospitalized for heart failure before or show signs of it.
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I have been diagnosed with a type of heart disease caused by transthyretin amyloidosis.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~30-36 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 30-36 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits)
Secondary outcome measures
All-cause Mortality
Change from Baseline in 6-Minute Walk Test (6-MWT) at Month 30
Change from Baseline in Global Longitudinal Strain by Echocardiographic Assessment at Month 30
+5 more

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: Vutrisiran 25 mgExperimental Treatment1 Intervention
Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) during the double-blind period.
Group II: PlaceboPlacebo Group1 Intervention
Participants will receive placebo during the double-blind period.

Find a Location

Who is running the clinical trial?

Alnylam PharmaceuticalsLead Sponsor
71 Previous Clinical Trials
13,663 Total Patients Enrolled
5 Trials studying Transthyretin Amyloidosis
5,713 Patients Enrolled for Transthyretin Amyloidosis
Medical DirectorStudy DirectorAlnylam Pharmaceuticals
2,766 Previous Clinical Trials
8,060,138 Total Patients Enrolled
2 Trials studying Transthyretin Amyloidosis
188 Patients Enrolled for Transthyretin Amyloidosis

Media Library

Vutrisiran (RNAi Therapeutics) Clinical Trial Eligibility Overview. Trial Name: NCT04153149 — Phase 3
Transthyretin Amyloidosis Research Study Groups: Placebo, Vutrisiran 25 mg
Transthyretin Amyloidosis Clinical Trial 2023: Vutrisiran Highlights & Side Effects. Trial Name: NCT04153149 — Phase 3
Vutrisiran (RNAi Therapeutics) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04153149 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this experiment allow octogenarians as volunteers?

"According to the study's eligibility requirements, patients between 18 and 85 years old may apply. There are 246 similar studies for individuals outside of this age range."

Answered by AI

Which type of person would be best suited for this clinical trial?

"Patients with amyloid neuropathies who come from families with a history of the condition are eligible for this trial if they are between 18 and 85 years old. A total of 655 patients can take part in this study."

Answered by AI

Is Vutrisiran a new medication or have their been other studies with this drug?

"There are currently two Vutrisiran clinical trials underway, both in Phase 3. The majority of the 158 total trial sites for this treatment are located in Houston, Texas."

Answered by AI

What is the uniqueness of this trial in relation to others?

"As of now, there are 2 ongoing studies for Vutrisiran in 24 different cities and 39 nations. The first trial was completed in 2019 by Alnylam Pharmaceuticals. That initial study had 164 participants and finished Phase 3 drug approval. In the years since, 17 more similar studies have been conducted."

Answered by AI

Are there any research facilities in North America testing this medication?

"Right now, medical professionals are looking for patients to participate in this clinical trial from 17 different hospitals. If you choose to partake in the study, it would be best to select a location near you so that travelling isn't too much of an inconvenience. The locations recruiting patients include Boston, Quebec City and Toronto as well as 14 other cities."

Answered by AI

When might Vutrisiran be available for public use?

"Vutrisiran is rated as a 3 in terms of safety by our team at Power. This is due to this being a Phase 3 trial, which provides some evidence for efficacy and multiple rounds of supporting data for safety."

Answered by AI
~123 spots leftby Mar 2025