What side effects are associated with this type of intervention?

Common treatments for Transthyretin Amyloid Cardiomyopathy (ATTR-CM) that reduce TTR protein production, such as antisense oligonucleotides (e.g., inotersen) and small interfering RNA (e.g., patisiran), have known side effects. Inotersen can cause thrombocytopenia (low platelet count) and glomerulonephritis (kidney inflammation), necessitating regular monitoring of platelet counts and kidney function. Patisiran may lead to infusion-related reactions, including flushing, back pain, and nausea. Both treatments require careful monitoring to manage these potential adverse effects effectively.

What prior FDA approvals exist?

The FDA has approved several treatments for Transthyretin Amyloid Cardiomyopathy (ATTR-CM) that target the reduction of transthyretin (TTR) protein production. Notably, Patisiran and Inotersen are RNA-targeted therapies similar to Eplontersen. Patisiran is a small interfering RNA (siRNA) that reduces TTR production by targeting its mRNA, while Inotersen is an antisense oligonucleotide that also reduces TTR protein levels. Both drugs have shown efficacy in reducing amyloid deposits and improving clinical outcomes in patients with hereditary ATTR amyloidosis.

What other types of research exist?

Promising alternatives to Eplontersen for ATTR-CM patients include Patisiran and Inotersen. Patisiran is an RNA interference (RNAi) therapeutic that reduces TTR protein production and has shown efficacy in treating hereditary ATTR amyloidosis with polyneuropathy. Inotersen is another antisense oligonucleotide that targets TTR mRNA to reduce TTR protein levels and is approved for hereditary ATTR amyloidosis with polyneuropathy. Both therapies have demonstrated potential benefits in clinical trials and may be considered for ATTR-CM treatment in 2024.

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Antisense Oligonucleotide

Eplontersen for Transthyretin Amyloid Cardiomyopathy

Phase 3

Recruiting

Research Sponsored by Ionis Pharmaceuticals, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Summary

This trial tests the safety of eplontersen, a medication given periodically, in patients with a specific heart condition (ATTR-CM). The drug aims to reduce harmful proteins to improve heart health.

Who is the study for?

This trial is for adults with a heart condition called ATTR-CM who have completed a previous eplontersen study or were part of the ISIS 420915-CS101 study and judged suitable by the investigator. Participants must be willing to take vitamin A as required and not have any new or worsening conditions that could affect their participation.

What is being tested?

The trial is testing the long-term safety of continued doses of a drug named Eplontersen in patients with Transthyretin-Mediated Amyloid Cardiomyopathy, which affects the heart. It aims to understand how well people tolerate this medication over an extended period.

What are the potential side effects?

While specific side effects are not listed here, participants will be monitored for any adverse reactions to Eplontersen during their extended treatment to ensure its ongoing safety and tolerability.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

Pivotal Trial

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

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: EplontersenExperimental Treatment1 Intervention

Eplontersen will be administered once every month by sub-cutaneous (SC) injection for up to 36 months or 6 months after eplontersen is approved and available in the site's country, whichever occurs first.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Eplontersen

2020

Completed Phase 3

~200

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Transthyretin Amyloid Cardiomyopathy (ATTR-CM) include RNA-targeted therapies such as antisense oligonucleotides (e.g., inotersen) and small interfering RNA (siRNA) formulations (e.g., patisiran). These treatments work by reducing the production of transthyretin (TTR) protein in the liver, thereby decreasing the availability of misfolded TTR monomers that form amyloid deposits in the heart. This reduction in amyloid deposits can help stabilize or improve cardiac function, alleviate symptoms, and potentially improve quality of life for patients with ATTR-CM. These mechanisms are crucial as they target the root cause of the disease, offering a more effective approach compared to symptomatic treatments.

Hereditary Transthyretin Amyloidosis: Clinical Presentation and Management Updates.Revertant Phenomenon in DMD and LGMD2I and Its Therapeutic Implications: A Review of Study Under Mentorship of Terrence Partridge.Persistent dystrophin protein restoration 90 days after a course of intraperitoneally administered naked 2'OMePS AON and ZM2 NP-AON complexes in mdx mice.