What side effects are associated with this type of intervention?

Common treatments for Transthyretin Amyloid Cardiomyopathy (ATTR-CM), such as monoclonal antibodies and small molecules designed to stabilize transthyretin or reduce amyloid deposition, have known side effects. Monoclonal antibodies can cause infusion-related reactions like fever, chills, rash, and fatigue. Small molecules like tafamidis may lead to diarrhea, urinary tract infections, and abdominal pain. Both treatment types may also pose risks of liver function abnormalities and cardiovascular events.

What prior FDA approvals exist?

The FDA has approved several treatments for Transthyretin Amyloid Cardiomyopathy (ATTR-CM). Tafamidis (Vyndaqel) is a small molecule that stabilizes the transthyretin protein, preventing it from misfolding and forming amyloid deposits. Patisiran (Onpattro) and Inotersen (Tegsedi) are RNA-targeted therapies that reduce the production of transthyretin, thereby decreasing amyloid deposition. These treatments aim to manage the progression of ATTR-CM by either stabilizing the transthyretin protein or reducing its synthesis, similar to the investigational agent ALXN2220.

What other types of research exist?

Tafamidis is a promising alternative for ATTR-CM patients. It has shown efficacy in delaying neurological progression in both Val30Met and non-Val30Met genotypes of transthyretin familial amyloid polyneuropathy. Additionally, other potential treatments include repurposed oncology drugs and emerging tau-targeting therapies, though these are less advanced.

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Monoclonal Antibodies

ALXN2220 for ATTR-CM (DepleTTR-CM Trial)

Phase 3

Recruiting

Research Sponsored by Alexion Pharmaceuticals, Inc.

Eligibility Criteria Checklist

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

Must have

Treatment with a loop diuretic for at least 30 days prior to Screening

History of heart failure NYHA Class II-IV at Screening

Must not have

Known leptomeningeal amyloidosis

Uncontrolled clinically significant cardiac arrhythmia, per Investigator's assessment

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

Summary

This trial is testing a new drug called ALXN2220 to help adults with a heart condition known as ATTR-CM. The goal is to see if the drug can lower the risk of death and serious heart issues. Researchers are comparing the outcomes between those who receive the drug and those who do not.

Who is the study for?

This trial is for adults with ATTR-CM, a type of heart disease caused by amyloid buildup. Participants should have symptoms and evidence of the condition but not be on other treatments for it. Those with other types of cardiomyopathy or who can't follow the study protocol are excluded.

What is being tested?

The study tests ALXN2220's effectiveness against a placebo in treating ATTR-CM. It measures which group has fewer deaths and cardiovascular events over time to see if ALXN2220 makes a difference.

What are the potential side effects?

Possible side effects aren't specified here, but generally, new treatments like ALXN2220 could cause unexpected reactions ranging from mild discomfort to more serious conditions that would be closely monitored.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been on a water pill for at least 30 days.

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I have a history of moderate to severe heart failure.

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My heart condition is confirmed as ATTR-CM, either wild-type or variant.

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 leptomeningeal amyloidosis.

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My heart rhythm is stable and not causing health issues.

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I have been diagnosed with a type of amyloidosis.

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I am on dialysis or my kidney function is very low.

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I have severe nerve damage that severely limits my daily activities.

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.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: ALXN2220Experimental Treatment1 Intervention

Participants will receive weight-based dose of ALXN2220 via intravenous (IV) infusion every 4 weeks (q4w) for at least 24 months up to a maximum of 48 months.

Group II: PlaceboPlacebo Group1 Intervention

Participants will receive placebo via IV infusion q4w for at least 24 months up to a maximum of 48 months.

0 / 8Eligibility 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 Transthyretin Amyloid Cardiomyopathy (ATTR-CM) include TTR stabilizers, RNA-targeted therapies, and investigational agents like monoclonal antibodies. TTR stabilizers, such as tafamidis, work by binding to the TTR protein and preventing its dissociation into monomers, which can misfold and form amyloid deposits. RNA-targeted therapies, like patisiran and inotersen, reduce the production of TTR by interfering with its synthesis in the liver. Investigational agents, including monoclonal antibodies, aim to enhance the clearance of amyloid deposits or inhibit their formation. These treatments are vital for ATTR-CM patients as they address the root cause of amyloid deposition, thereby potentially slowing disease progression and improving cardiac function.

Diagnosis and therapeutic approaches to transthyretin amyloidosis.