Transthyretin AmyloidosisPhase 2NCT05635045

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

A Phase 2 study testing I 124-Evuzamitide in people with Transthyretin Amyloidosis, run by Columbia University at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 50 to 105 yearsHealthy volunteers can take part
Conditions the study is for: Transthyretin Amyloidosis
What is being tested?: I 124-Evuzamitide
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 10 participants
Where: 1 siteUnited States
Study dates: 8 Jul 2022to 12 Jun 2024
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 50 to 105 years
All sexes
Healthy volunteers accepted
Adult, Older adult

Who may be able to join

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.

Who cannot join

Overall Inclusion Criteria:
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).

Show all 17Show fewer

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.
Other reason that would make the subject inappropriate for entry into this study.

Trial locations and contacts

1 site in 1 country.

United States1 site

Columbia University Irving Medical Center
New York, New York, 10032

Other trials in United States

About this study

Official title: A Follow-Up Study to Monitor Therapeutic Response in Transthyretin Cardiac Amyloidosis Using Amyloid Reactive Peptide 124I-evuzamitide (AT01) PET/CT

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.

Read the detailed description

Transthyretin cardiac amyloidosis (ATTR-CA) causes progressive heart disease that is often overlooked. It harms the heart muscle because the unstable, unfurled amyloid proteins fold up into large pieces that get caught in between layers of heart tissue, causing amyloid deposits. The earlier it is detected, the better for the patient.

There is a need to improve the early diagnosis of this disease because echocardiography (sonograms of the heart) and cardiac MRI are not useful enough for this. There is an X-ray of the heart using a compound called PYP that can detect amyloid deposits earlier than ultrasound images or clinical signs, but it's not clear how early it does so. Also, it can't detect amyloid deposits outside the heart, which causes lots of pain and suffering in people with this disease.

In the first phase of this study the radiation compound, evuzamitide, was shown to detect amyloidosis in the heart of selected subjects. So, the investigators used it to detect potential therapeutic changes in 10 subjects undergoing treatment for Transthyretin Amyloidosis.

Changes in the uptake of evuzamitide were compared between subjects treated on stabilizer only, subjects treated on silencer only and subjects treated with two different combinations of stabilizer plus silencer

Conditions: Transthyretin Amyloidosis
Being tested: I 124-Evuzamitide
Keywords: 124I-evuzamitide, Positron Emission Tomography (PET), ATTR-CM
Medical subject headings: Amyloidosis, Hereditary, Transthyretin-Related

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

I 124-EvuzamitideDrug
Also called Attralus AT-01
Amyloid reactive protein used as imaging agent in whole body SPECT-CT imaging

Study groups in NCT05635045
Study group	Role	What they receive
Stabilizer Only	Active comparator	Subjects treated with tafamidis selected to re-scan to determine the change over time in Evuzamitide uptake Receives: 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 Receives: 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 Receives: 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 Receives: Drug: I 124-Evuzamitide

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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.
Measured: 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.
Measured: Up to one month after imaging

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

8 Jul 202212 Jun 2024

First posted2 Dec 2022
Study start8 Jul 2022
Primary completion7 May 2024
Study completion12 Jun 2024

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Transthyretin AmyloidosisStudies of the same condition, open and under way.
More trials run by Columbia UniversityThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Diagnosis
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 10 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05635045
Sponsor's protocol ID: AAAU0561
Status last verified: Jul 2025
First submitted: 23 Nov 2022
Last updated: 18 Jul 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

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.
PubMed 40439628

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingObservational study
A Retrospective Observational Study of Epidemiology and Outcomes in Transthyretin Amyloid Cardiomyopathy in Germany
ATTR-CM (Transthyretin Amyloid Cardiomyopathy)
Active, not recruitingPhase 3
EPIC-ATTR: A Study to Evaluate the Effect of Eplontersen on the Transthyretin Reduction and Long-term Safety in Chinese Subjects With Transthyretin Amyloid Cardiomyopathy
Transthyretin Amyloid Cardiomyopathy
RecruitingObservational study
Essen Amyloidosis Registry
Amyloidosis; Systemic, Amyloidosis Cardiac
Active, not recruitingPhase 3
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Neuromuscular Disease, Neuromuscular Diseases (NMD)
RecruitingPhase 3
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

All Transthyretin Amyloidosis trials

Study at a glance

From the registry record, last updated by the sponsor on 18 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Follow-Up Study to Monitor Therapeutic Response in Transthyretin Cardiac Amyloidosis Using Amyloid Reactive Peptide 124I-evuzamitide (AT01) PET/CT
ClinicalTrials.gov ID: NCT05635045
Acronym / sponsor ID: AAAU0561
Conditions: Transthyretin Amyloidosis
Keywords: 124I-evuzamitide, Positron Emission Tomography (PET), ATTR-CM
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 10 participants (actual)
Lead sponsor: Columbia UniversityUniversity, hospital or other
Collaborators: Attralus, Inc.
Study start: 8 Jul 2022 (actual)
Primary completion: 7 May 2024 (actual)
Study completion: 12 Jun 2024 (actual)
First posted: 2 Dec 2022 (actual)
Results: Posted 18 Jul 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Diagnosis
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 4

Arm	Type	Receives
Stabilizer Only	Active comparator	Drug: I 124-EvuzamitideSubjects treated with tafamidis selected to re-scan to determine the change over time in Evuzamitide uptake
Silencer Only	Active comparator	Drug: I 124-EvuzamitideSubjects treated with vutrisiran selected to re-scan to determine the change over time in Evuzamitide uptake
Stabilizer and Silencer (tafamidis + patisiran)	Active comparator	Drug: I 124-EvuzamitideSubjects treated with tafamidis and patisiran selected to re-scan to determine the change over time in Evuzamitide uptake
Stabilizer and Silencer (vutrisiran + diflunisal)	Active comparator	Drug: I 124-EvuzamitideSubjects treated with vutrisiran and diflunisal selected to re-scan to determine the change over time in Evuzamitide uptake

Who may qualify

Ages: 50 to 105 years
Sex: All
Healthy volunteers: Accepted
Age groups: Adult, Older adult

Key inclusion criteria (3 listed)

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.

Key exclusion criteria (17 listed)

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.
Primary amyloidosis (AL) or secondary amyloidosis (AA).
Active malignancy or non-amyloid disease with expected survival of less than 1 year.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
I 124-EvuzamitideAlso: Attralus AT-01Amyloid reactive protein used as imaging agent in whole body SPECT-CT imaging	Drug	Silencer Only, Stabilizer Only, Stabilizer and Silencer (tafamidis + patisiran), Stabilizer and Silencer (vutrisiran + diflunisal)

Primary outcome

Median of Uptake of Evuzamitide as Measured by Volume of Interest in Milliliters
Time frame: Up to one month after imaging
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.
Median of Change in Evuzamitide Uptake as Measured by Percentage Change in Injected Dose (%ID)
Time frame: Up to one month after imaging
Median percentage change in injected dose (%ID), calculated as VOI mean activity concentration x VOI volume/injected activity.
Median of Uptake of Evuzamitide as Measured by Cardiac Amyloid Activity (CAA) in Grams
Time frame: Up to one month after imaging
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.

Results and safety

Results posted to the registry on 18 Jul 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Stabilizer Only	4	4
Silencer Only	1	1
Stabilizer and Silencer (Tafamidis + Patisiran)	3	2
Stabilizer and Silencer (Vutrisiran + Diflunisal)	2	2

Primary outcome: Median of Uptake of Evuzamitide as Measured by Volume of Interest in Milliliters

Time frame: Up to one month after imaging. Median (Inter-Quartile Range). Unit: mL

Measure	Stabilizer Only n = 4	Silencer Only n = 1	Stabilizer and Silencer (Tafamidis + Patisiran) n = 2	Stabilizer and Silencer (Vutrisiran + Diflunisal) n = 2
Value	78.7 (13.4 to 137.8)	253.6 (253.6 to 253.6)	177.2 (-31.2 to 385.5)	39.6 (1.4 to 77.8)

Population analysed: 1 subject was scanned but their measurements were not usable due material error in imaging agent supplied.

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

Time frame: Up to one month after imaging. Median (Inter-Quartile Range). Unit: percent change

Measure	Stabilizer Only n = 4	Silencer Only n = 1	Stabilizer and Silencer (Tafamidis + Patisiran) n = 2	Stabilizer and Silencer (Vutrisiran + Diflunisal) n = 2
Value	0.2 (-0.1 to 0.5)	0.8 (0.8 to 0.8)	1.3 (1.2 to 1.5)	0.3 (0.2 to 0.4)

Population analysed: 1 subject was scanned but their measurements were not usable due material error in imaging agent supplied.

Primary outcome: Median of Uptake of Evuzamitide as Measured by Cardiac Amyloid Activity (CAA) in Grams

Time frame: Up to one month after imaging. Median (Inter-Quartile Range). Unit: grams

Measure	Stabilizer Only n = 4	Silencer Only n = 1	Stabilizer and Silencer (Tafamidis + Patisiran) n = 2	Stabilizer and Silencer (Vutrisiran + Diflunisal) n = 2
Value	180.2 (-75.9 to 437.8)	852.8 (852.8 to 852.8)	952.5 (812.5 to 1092.5)	155.4 (-18.7 to 329.4)

Population analysed: 1 subject was scanned but their measurements were not usable due material error in imaging agent supplied.

Adverse events

Time frame: 28 days. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Stabilizer Only	0 of 4 (0%)	0 of 4 (0%)	0 of 4
Silencer Only	0 of 1 (0%)	0 of 1 (0%)	0 of 1
Stabilizer and Silencer (Tafamidis + Patisiran)	0 of 3 (0%)	1 of 3 (33.3%)	0 of 3
Stabilizer and Silencer (Vutrisiran + Diflunisal)	0 of 2 (0%)	0 of 2 (0%)	0 of 2

Limitations and caveats (from the sponsor): Material problems with one dose of agent prevented one subject's measures from being analyzed.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 18 Jul 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 40439628Smiley 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.
Individual participant dataSharing planned: No

Related trials for Transthyretin Amyloidosis

Active, not recruitingObservational study
A Retrospective Observational Study of Epidemiology and Outcomes in Transthyretin Amyloid Cardiomyopathy in Germany
ATTR-CM (Transthyretin Amyloid Cardiomyopathy)
Active, not recruitingPhase 3
EPIC-ATTR: A Study to Evaluate the Effect of Eplontersen on the Transthyretin Reduction and Long-term Safety in Chinese Subjects With Transthyretin Amyloid Cardiomyopathy
Transthyretin Amyloid Cardiomyopathy
RecruitingObservational study
Essen Amyloidosis Registry
Amyloidosis; Systemic, Amyloidosis Cardiac
Active, not recruitingPhase 3
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Neuromuscular Disease, Neuromuscular Diseases (NMD)
RecruitingPhase 3
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Mathew Maurer, MD principal investigator

Columbia University

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing I 124-Evuzamitide in people with Transthyretin Amyloidosis, run by Columbia University at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 18 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 18 Jul 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Median of Uptake of Evuzamitide as Measured by Volume of Interest in Milliliters (time frame: Up to one month after imaging). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05635045, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jul 2025.