Evuzamitide in PET/CT to Measure Potential Therapeutic Response in ATTR

NCT05635045 | Completed Phase 2 Results FDA Drug

• 1 other identifier: AAAU0561
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

This was a single center, prospective cohort study that is evaluating the ability of 124I-evuzamitide PET scanning to detect potential therapeutic changes in subjects under treatment for ATTR after one year had elapsed since their original 124I-evuzamitide PET scan. Ten previously scanned subjects re-consented to undergo another 124I-evuzamitide PET scan. Demographic, clinical and phenotypic data were collected to characterize potential changes since their previous scans.

Conditions

Transthyretin Amyloidosis

Keywords

124I-evuzamitide; Positron Emission Tomography (PET); ATTR-CM

Outcome Measures

Primary Outcomes (3)

• Median of Uptake of Evuzamitide as Measured by Volume of Interest in Milliliters

  Volumes of interest (VOIs) were manually traced on PET emission images fused with CT images to define the left ventricular (LV) and right ventricular (RV) contours, including cavity blood pool. Blood pool activity concentration was measured in a 10-mm-diameter left atrial spherical VOI. For 124I-evuzamitide, we used a threshold of mean + 2 standard deviations of blood pool activity concentration.

  Up to one month after imaging

• Median of Change in Evuzamitide Uptake as Measured by Percentage Change in Injected Dose (%ID)

  Median percentage change in injected dose (%ID), calculated as VOI mean activity concentration x VOI volume/injected activity.

  Up to one month after imaging

• Median of Uptake of Evuzamitide as Measured by Cardiac Amyloid Activity (CAA) in Grams

  Cardiac amyloid activity (CAA) in grams is measured as VOI mean standardized uptake value times VOI volume. Standardized uptake value was defined as mean or maximal VOI activity concentration/(injected activity/bodyweight), while target-to-background ratio was considered VOI mean activity concentration/blood pool mean activity concentration. VOI volume was manually traced on PET emission images fused with CT images.

  Up to one month after imaging

Study Arms (4)

Stabilizer Only

ACTIVE COMPARATOR

Subjects treated with tafamidis selected to re-scan to determine the change over time in Evuzamitide uptake

Drug: I 124-Evuzamitide

Silencer Only

ACTIVE COMPARATOR

Subjects treated with vutrisiran selected to re-scan to determine the change over time in Evuzamitide uptake

Drug: I 124-Evuzamitide

Stabilizer and Silencer (tafamidis + patisiran)

ACTIVE COMPARATOR

Subjects treated with tafamidis and patisiran selected to re-scan to determine the change over time in Evuzamitide uptake

Drug: I 124-Evuzamitide

Stabilizer and Silencer (vutrisiran + diflunisal)

ACTIVE COMPARATOR

Subjects treated with vutrisiran and diflunisal selected to re-scan to determine the change over time in Evuzamitide uptake

Drug: I 124-Evuzamitide

Interventions

I 124-Evuzamitide DRUG

Amyloid reactive protein used as imaging agent in whole body SPECT-CT imaging

Also known as: Attralus AT-01

Silencer Only; Stabilizer Only; Stabilizer and Silencer (tafamidis + patisiran); Stabilizer and Silencer (vutrisiran + diflunisal)

Eligibility Criteria

• Age: 50 Years - 105 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements.
• Were enrolled in the initial pilot using 124I-Evuzamitide, on ATTR stabilizer and/or silencer therapy. Able to understand and sign the informed consent document after the nature of the study has been fully explained.
• TTR genotype shown to be either Val122Ile or wild type.

You may not qualify if:

• Must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements.
• Were enrolled in the initial pilot using 124I-Evuzamitide, on ATTR stabilizer and/or silencer therapy. Able to understand and sign the informed consent document after the nature of the study has been fully explained.
• TTR genotype shown to be either Val122Ile or wild type.
• The presence of any of the following excludes eligibility for enrollment in this study:
• Primary amyloidosis (AL) or secondary amyloidosis (AA).
• Active malignancy or non-amyloid disease with expected survival of less than 1 year.
• Heart failure, in the opinion of the investigator, primarily caused by something other than amyloidosis.
• Ventricular assist device.
• Impairment from stroke, injury or other medical disorder that precludes participation in the study.
• Disabling dementia or other mental or behavioral disease.
• Enrollment in a clinical trial not approved for co-enrollment.
• Continuous intravenous inotropic therapy.
• Inability or unwillingness to comply with the study requirements.
• Chronic kidney disease requiring hemodialysis or peritoneal dialysis.
• Patients taking heparin, or heparin derivatives (e.g. low molecular weight heparins) for anticoagulation.

Sponsors & Collaborators

• Columbia University: lead
• Attralus, Inc.: collaborator

Study Sites (1)

Columbia University Irving Medical Center

New York, New York, 10032, United States

Location

Related Publications (1)

• Smiley DA, Einstein AJ, O'Gorman KJ, Santana D, Teruya S, Chan N, Nalbandian A, Poterucha TJ, Helmke ST, Mintz A, Goldner K, Sekulic M, Mirabal A, Cuomo MO, Guadalupe S, De Los Santos J, Paulino ME, Mateo KA, Rodriguez CM, Jimenez M, Wardhere A, Bampatsias D, Castillo M, Peng B, Maurer MS. Early Detection of Transthyretin Cardiac Amyloidosis Using 124I-Evuzamitide Positron Emission Tomography/Computed Tomography. JACC Cardiovasc Imaging. 2025 Jul;18(7):799-811. doi: 10.1016/j.jcmg.2025.01.018. Epub 2025 May 28.

  PMID: 40439628 DERIVED

MeSH Terms

Conditions

Amyloidosis, Hereditary, Transthyretin-Related

Limitations and Caveats

Material problems with one dose of agent prevented one subject's measures from being analyzed.

Results Point of Contact

• Title: Mathew Maurer
• Organization: Columbia University Medical Center

msm10@cumc.columbia.edu

2123058274

Study Officials

• Mathew Maurer, MD

  Columbia University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Arnold and Arlene Goldstein Professor of Medicine

Study Record Dates

• First Submitted: November 23, 2022
• First Posted: December 2, 2022
• Study Start: July 8, 2022
• Primary Completion: May 7, 2024
• Study Completion: June 12, 2024
• Last Updated: July 18, 2025
• Results First Posted: July 18, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)