What side effects are associated with this type of intervention?

Treatments for amyloidosis, particularly those involving radiolabeled compounds like 124I-evuzamitide, can cause side effects such as radiation exposure risks, allergic reactions, and injection site reactions. Broader treatments, including anti-amyloid antibodies, may lead to amyloid-related imaging abnormalities (ARIA), headaches, falls, diarrhea, confusion, and hypersensitivity reactions.

What prior FDA approvals exist?

FDA-approved treatments for amyloidosis primarily focus on reducing amyloid production and managing symptoms. These include chemotherapy agents like melphalan, corticosteroids such as dexamethasone, and proteasome inhibitors like bortezomib. Monoclonal antibodies such as daratumumab have also been approved for treating light chain (AL) amyloidosis. While the context does not mention specific FDA-approved radiolabeled compounds for amyloid visualization, ongoing studies like the 124I-evuzamitide PET scan aim to enhance diagnostic capabilities and therapeutic monitoring in amyloidosis.

What other types of research exist?

Promising alternatives to 124I-evuzamitide PET Scan for amyloidosis patients include 18F-florbetapir, 18F-flutemetamol, and 11C-PiB (Pittsburgh Compound B) PET scans. These radiolabeled compounds are used for amyloid imaging and have shown efficacy in detecting amyloid deposits in various clinical settings.

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Evuzamitide Imaging for Amyloidosis

Phase 2

Waitlist Available

Led By Mathew Maurer, MD

Research Sponsored by Columbia University

Eligibility Criteria Checklist

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

Must have

TTR genotype shown to be either Val122Ile or wild type

Left ventricular septal OR inferolateral wall thickness ≥12 mm with echocardiographic features of ATTR-CM (low tissue doppler velocities, preserved apical strain, elevated E/E') or CMR features of an infiltrative cardiomyopathy (increased wall thickness with delayed enhancement or difficulty nulling of the myocardium)

Must not have

Active malignancy or non-amyloid disease with expected survival of less than 1 year

Chronic kidney disease requiring hemodialysis or peritoneal dialysis

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 1 month after scanning

Awards & highlights

No Placebo-Only Group

Summary

This trial uses a special imaging technique to look for changes in the hearts of patients with a specific heart condition after treatment. The scan helps doctors see harmful protein deposits in the heart, which can indicate if the treatment is working.

Who is the study for?

This trial is for adults with thickened heart walls and signs of cardiac amyloidosis, who can consent to the study. It's not for those with primary or secondary amyloidosis unrelated to transthyretin, pregnant women, patients on certain blood thinners, or anyone with a life expectancy under one year due to other diseases.

What is being tested?

The trial tests I 124-Evuzamitide as a PET scan imaging agent in detecting cardiac and extra-cardiac amyloid deposits in patients suspected of having ATTR-CM but have inconclusive standard scans. It aims to improve diagnosis accuracy for specific genetic variants associated with this condition.

What are the potential side effects?

Potential side effects are not explicitly listed here; however, as with any PET scan procedure using radiotracers like I 124-Evuzamitide, there may be risks related to radiation exposure and possible allergic reactions to the tracer.

Eligibility Criteria

Inclusion Criteria

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

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My genetic test shows I have the Val122Ile mutation or no mutation.

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My heart has thick walls and signs of a specific type of heart disease.

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I have a grade 1 Tc99-PYP scan with signs of heart amyloidosis or confirmed by biopsy.

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My heart pumps well (EF>40%).

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I have ATTR-CM and signs of amyloidosis outside my heart, like nerve issues or carpal tunnel.

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My tests show amyloid deposits in my heart or other tissues.

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My heart condition allows me to perform daily activities with slight or moderate limitations.

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I had a Tc99-PYP scan for heart issues, but it didn't confirm the condition.

Exclusion Criteria

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

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I have a serious illness with a life expectancy of less than 1 year.

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I am on dialysis for chronic kidney disease.

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I am currently on heparin or similar medications for blood thinning.

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I am on a continuous IV medication to strengthen my heart.

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I have been diagnosed with primary or secondary amyloidosis.

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I do not have any impairments from stroke, injury, or other conditions that would stop me from joining the study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 1 month after scanning

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 1 month after scanning

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 1 month after scanning for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

COV in subjects who are allele carriers with non-diagnostic PYP scans

COV in subjects with ATTR-CM

COV in subjects with heart failure and grade 1 PYP scans

Awards & Highlights

No Placebo-Only Group

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

Trial Design

3Treatment groups

Experimental Treatment

Group I: Cohort 3Experimental Treatment1 Intervention

Subjects with ATTR-CM from either ATTRwt or Val122Ile variant who have biopsy proven evidence of extra-cardiac TTR amyloidosis or clinical suspicion of extracardiac disease, including but not limited to peripheral neuropathy, carpal tunnel syndrome, spinal stenosis will be administered single dose evuzamitide \<1mCi.. 1. ATTR-CM defined by the following 1. Amyloid deposits in cardiac or non-cardiac tissue confirmed by Congo Red (or equivalent) staining OR technetium scintigraphy with 99m Tc-pyrophosphate with Grade 2 or 3 cardiac uptake in the absence of abnormal light chains ratio, 2. End-diastolic interventricular septum thickness of \> 12 mm on previous echocardiogram 2. TTR genotype shown to be either Val122Ile or wild type.

Group II: Cohort 2Experimental Treatment1 Intervention

Subjects with TTR variant such as Phe64Leu, late onset Val30Met, etc.) that are associated with cardiac amyloidosis but have PYP scans not diagnostic of ATTR-CM will be administered single dose evuzamitide \<1mCi. 1. Tc99-PYP scan performed for clinical suspicion of ATTR-CM that is not diagnostic of ATTR-CM 2. No evidence of monoclonal proteins by assessment of serum kappa and lambda free light chain ratio and immunofixation of serum and urine. 3. Left ventricular septal OR inferolateral wall thickness ≥12 mm with echocardiographic features of ATTR-CM (low tissue doppler velocities, preserved apical strain, elevated E/E') or CMR features of an infiltrative cardiomyopathy (increased wall thickness with delayed enhancement or difficulty nulling of the myocardium)

Group III: Cohort 1Experimental Treatment1 Intervention

Subjects with grade 1 Tc99-PYP scans who have clinical features suggestive of ATTR-CM or have grade 1 Tc99-PYP scans but endomyocardial biopsy evidence of TTR cardiac amyloidosis will be administered single dose evuzamitide \<1mCi. 1. Heart failure with a preserved ejection fraction (EF\>40%) 2. Grade 1 Tc99-PYP scan performed for clinical suspicion of ATTR-CM 3. No evidence of monoclonal proteins by assessment of serum kappa and lambda free light chain ratio and immunofixation of serum and urine. 4. Left ventricular septal OR inferolateral wall thickness ≥12 mm

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

I 124-Evuzamitide

2022

Completed Phase 2

~30

0 / 14Eligibility 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

Common treatments for Amyloidosis include immunotherapy, radiolabeled compounds for amyloid visualization, and stem cell transplantation. Immunotherapy targets proteins involved in amyloid binding and aggregation, potentially reducing amyloid accumulation. Radiolabeled compounds like 124I-evuzamitide are used in PET scans to visualize amyloid deposits, aiding in diagnosis and monitoring treatment efficacy. Stem cell transplantation aims to treat underlying plasma cell dyscrasias, particularly in AL amyloidosis, by replacing diseased cells with healthy ones. These treatments are crucial as they help manage amyloid burden, improve organ function, and enhance patient outcomes.

Efficacy of chronic BACE1 inhibition in PS2APP mice depends on the regional Aβ deposition rate and plaque burden at treatment initiation.Anti-apoE immunotherapy inhibits amyloid accumulation in a transgenic mouse model of Aβ amyloidosis.A clinico-epidemiological study of macular amyloidosis from north India.