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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

Must not have

Has known primary amyloidosis or leptomeningeal amyloidosis

Has other non-TTR cardiomyopathy, hypertensive cardiomyopathy, cardiomyopathy due to valvular heart disease, or cardiomyopathy due to ischemic heart disease

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to month 36

Awards & highlights

Pivotal Trial

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have been diagnosed with primary or leptomeningeal amyloidosis.

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I have a heart condition not caused by TTR amyloidosis.

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I have severe nerve damage affecting my daily activities.

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I have been treated before to lower TTR levels.

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I have the most severe form of heart failure.

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I have severe heart failure and am considered high risk.

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My kidney function is severely reduced.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to month 36

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to month 36

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to month 36 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Composite Endpoint of All-Cause Mortality and Recurrent CV Events (CV Hospitalizations and Urgent HF Visits) in the Vutrisiran Monotherapy Subgroup

Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) in the Overall Population

Secondary study objectives

Change from Baseline in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) in the Overall Population and Vutrisiran Monotherapy Subgroup

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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 (DB) period. After the DB period, participants enter the open-label randomized treatment extension (RTE) period to receive vutrisiran 25 mg SC q3M or vutrisiran 50 mg SC every 6 months (q6M). After implementation of Amendment 4, participants from the DB period will enter the open-label treatment extension (OLE) period to receive vutrisiran 25 mg SC q3M and participants who were previously receiving vutrisiran 50 mg q6M in the RTE period, will be transitioned to vutrisiran 25 mg q3M in the OLE period.

Group II: PlaceboPlacebo Group2 Interventions

Participants will receive placebo during the double-blind period. After the DB period, participants enter the RTE period to receive vutrisiran 25 mg SC q3M or vutrisiran 50 mg SC q6M. After implementation of Amendment 4, participants from the DB period will enter the OLE period to receive vutrisiran 25 mg SC q3M and participants who were previously receiving vutrisiran 50 mg q6M in the RTE period, will be transitioned to vutrisiran 25 mg q3M in the OLE period.

0 / 9Eligibility criteria met