What side effects are associated with this type of intervention?

Common treatments for Amyloid Cardiomyopathy that stabilize transthyretin protein, such as tafamidis and diflunisal, generally have a favorable safety profile but can cause side effects like gastrointestinal issues (nausea, diarrhea), fatigue, and urinary tract infections. RNA silencing therapies like patisiran and inotersen may lead to infusion-related reactions, peripheral edema, and in some cases, thrombocytopenia and liver enzyme elevations. These side effects are generally manageable with appropriate medical supervision.

What prior FDA approvals exist?

The FDA has approved tafamidis (marketed as Vyndaqel and Vyndamax) for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). Tafamidis works by stabilizing the transthyretin protein, thereby preventing its misfolding and subsequent amyloid deposition in tissues. This mechanism is similar to that of Acoramidis (AG10), which is currently under investigation. Tafamidis has been shown to delay disease progression and improve survival in patients with ATTR-CM.

What other types of research exist?

Promising alternatives to Acoramidis (AG10) for Amyloid Cardiomyopathy patients include Tafamidis, which stabilizes transthyretin tetramers and slows disease progression, and monoclonal antibodies like 2T5C9 and 2G9C, which target transthyretin amyloid fibrils. Additionally, ongoing research into anti-amyloidosis nanomedicines and retinoids like acitretin, which enhance ADAM10 gene expression, may offer novel therapeutic approaches.

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Transthyretin Stabilizer

Acoramidis for Amyloid Cardiomyopathy

Phase 3

Waitlist Available

Research Sponsored by Eidos Therapeutics, a BridgeBio company

Eligibility Criteria Checklist

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

Must have

Be older than 18 years old

Must not have

At the end of Study AG10-301 or at Day 1 of Study AG10-304 (or any time during the study), participant is on prohibited medication

Has confirmed diagnosis of light-chain (AL) amyloidosis at any time during Study AG10-301

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 60 months

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing the safety and effectiveness of acoramidis in people with a heart condition called ATTR-CM. The drug works by preventing harmful protein clumps in the heart. Participants must have completed a previous study and cannot use other specific treatments during this trial.

Who is the study for?

This trial is for people who finished the AG10-301 study, understand and can sign consent forms, and agree to use effective birth control. It's not for those with certain medical conditions or sensitivities, recent heart issues or strokes, kidney problems, other clinical trials within 30 days, substance abuse history, psychiatric conditions that affect compliance, mechanical heart devices or transplants.

What is being tested?

The trial tests Acoramidis (AG10) in patients with Transthyretin Amyloid Cardiomyopathy who completed a previous Phase 3 trial. It's an open-label extension meaning everyone knows they're getting AG10 and it focuses on long-term safety monitoring of the drug.

What are the potential side effects?

While specific side effects are not listed here, participants will be monitored for any adverse reactions due to Acoramidis (AG10), which could include typical drug-related side effects such as allergic reactions or issues related to its impact on amyloid cardiomyopathy.

Eligibility Criteria

Exclusion Criteria

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

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I am not taking any medications that are not allowed in the study.

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I have been diagnosed with AL amyloidosis.

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I have had a heart or liver transplant, or I am on the heart transplant list.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 60 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~60 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 60 months for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Incidence of treatment-emergent Adverse Events [Safety and Tolerability]

Secondary study objectives

Evaluate all-cause mortality and cardiovascular mortality

Evaluate the effect of acoramidis on health-related quality of life Kansas City Cardiomyopathy Questionnaire

Evaluate the effect of acoramidis on the 6-minute walk test (6MWT)

+1 more

Awards & Highlights

No Placebo-Only Group

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

Pivotal Trial

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: AG10Experimental Treatment1 Intervention

Open-label study all participants will receive AG10 during this study.

0 / 3Eligibility criteria met

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 Amyloid Cardiomyopathy include TTR stabilizers like Acoramidis (AG10) and Tafamidis, which work by binding to the TTR protein and preventing its misfolding and aggregation into amyloid fibrils. Additionally, RNA silencing therapies such as Patisiran and Inotersen reduce the production of TTR protein by targeting its mRNA, thereby decreasing the availability of misfolded monomers. Gene editing approaches, like those using CRISPR-Cas9, aim to knock out the TTR gene to prevent the production of amyloidogenic proteins altogether. These treatments are vital for patients as they address the underlying cause of amyloid deposition, thereby slowing disease progression and improving quality of life.

Inhibition of amyloid formation of amyloid β (1-42), amylin and insulin by 1,5-diazacyclooctanes, a spermine-acrolein conjugate.Amylin and Secretases in the Pathology and Treatment of Alzheimer's Disease.In vitro and in vivo models for anti-amyloidosis nanomedicines.