Trial Outcomes & Findings for Repeat PET/CT Imaging of Patients With Amyloid 124I-AT-01 to Measure Changes in Organ-specific Amyloid Load (NCT NCT05968846)
NCT ID: NCT05968846
Last Updated: 2026-09-02
Results Overview
The goal of this research study is to determine whether changes in organ-specific uptake of 124I- AT-01 can be measured by PET/CT imaging and further, whether these values correlate with changes in a subject's disease status and thereby enable monitoring of disease response over time in terms of organ-specific amyloid load. The specific aim of this study is to measure visual and quantitative changes in organ-specific uptake of 124I-AT-01 from baseline, in patients with systemic amyloidosis, using PET/CT imaging
COMPLETED
PHASE2
19 participants
Baseline PET/CT imaging from AMY1001 (NCT03678259) and repeat PET/CT imaging on Day 2 of the current study
2026-09-02
Participant Flow
Data of participants diagnosed with systemic AL or ATTR amyloidosis who were previously imaged with 124I-AT-01 as part of the AMY1001 study (NCT03678259) and in whom there was visually positive uptake of radiotracer in at least one abdominothoracic organ.
Prior to assignment, potential participants were screened to verify their medical records, including confirming that participants must have been imaged with 124I-AT-01 as part of the AMY1001 study more than 12 months prior to the repeat imaging.
Participant milestones
| Measure |
All Participants
Experimental: Patients will receive a single IV injection of 124I-AT-01 radiotracer and PET/CT imaging
124I-AT-01 (124I-p5+14, iodine-124I evuzamitide) is an iodine-124 (124I) labeled 45 L-amino acid peptide suitable for PET/CT imaging. The peptide binds many forms of amyloid through multivalent electrostatic interactions with the amyloid fibril and ubiquitous heparan sulfate proteoglycans.
124I-AT-01 has been evaluated previously in an open-label Phase 1/2 clinical trial, AMY1001, performed at the University of Tennessee Medical Center (IND# 132282; NCT T03678259).
In this repeat imaging study, patients previously enrolled in the AMY1001 study, in whom positive PET/CT imaging findings were observed will undergo repeat imaging to assess changes in radiotracer uptake in the liver, spleen, heart and kidneys.
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Overall Study
STARTED
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19
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Overall Study
COMPLETED
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19
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
% of AL patients; n=9
Baseline characteristics by cohort
| Measure |
All Participants
n=19 Participants
Experimental: Patients will receive a single IV injection of 124I-AT-01 radiotracer and PET/CT imaging
In this repeat imaging study, patients previously enrolled in the AMY1001 study, in whom positive PET/CT imaging findings were observed will undergo repeat imaging to assess changes in radiotracer uptake in the liver, spleen, heart and kidneys.
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Age, Continuous
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72 Years
n=19 Participants
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Sex: Female, Male
Female
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8 Participants
n=19 Participants
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Sex: Female, Male
Male
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11 Participants
n=19 Participants
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Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=19 Participants
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Race (NIH/OMB)
Asian
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0 Participants
n=19 Participants
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Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=19 Participants
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Race (NIH/OMB)
Black or African American
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1 Participants
n=19 Participants
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Race (NIH/OMB)
White
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18 Participants
n=19 Participants
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Race (NIH/OMB)
More than one race
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0 Participants
n=19 Participants
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Race (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=19 Participants
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Ethnicity (NIH/OMB)
Hispanic or Latino
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0 Participants
n=19 Participants
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Ethnicity (NIH/OMB)
Not Hispanic or Latino
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19 Participants
n=19 Participants
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Ethnicity (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=19 Participants
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Time Since SA Diagnosis
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7 Years
n=19 Participants
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Time Since baseline imaging in AMY1001 study (years)
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2.9 Years
STANDARD_DEVIATION 0.9 • n=19 Participants
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Amyloid Type
AL Kappa
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6 Participants
n=19 Participants
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Amyloid Type
AL Lambda
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3 Participants
n=19 Participants
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Amyloid Type
ATTR; Mutation type L58H
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3 Participants
n=19 Participants
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Amyloid Type
ATTR; Mutation type T60A
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5 Participants
n=19 Participants
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Amyloid Type
ATTR; Mutation type V30M and V122I
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1 Participants
n=19 Participants
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Amyloid Type
ATTR; Mutation type Wild type
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1 Participants
n=19 Participants
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Amyloid therapy at time of imaging of AL
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2 Participants
n=9 Participants • % of AL patients; n=9
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Amyloid therapy at time of imaging of ATTR
Stabilizer only
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1 Participants
n=10 Participants • % of ATTR patients; n=10, Abbreviations: AL = light chain-associated amyloidosis; ATTR = transthyretin-associated amyloidosis
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Amyloid therapy at time of imaging of ATTR
Silencer only
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5 Participants
n=10 Participants • % of ATTR patients; n=10, Abbreviations: AL = light chain-associated amyloidosis; ATTR = transthyretin-associated amyloidosis
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Amyloid therapy at time of imaging of ATTR
Stabilizer and silencer
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4 Participants
n=10 Participants • % of ATTR patients; n=10, Abbreviations: AL = light chain-associated amyloidosis; ATTR = transthyretin-associated amyloidosis
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PRIMARY outcome
Timeframe: Baseline PET/CT imaging from AMY1001 (NCT03678259) and repeat PET/CT imaging on Day 2 of the current studyPopulation: Summary of change in visual radiotracer uptake from baseline PET/CT imaging
The goal of this research study is to determine whether changes in organ-specific uptake of 124I- AT-01 can be measured by PET/CT imaging and further, whether these values correlate with changes in a subject's disease status and thereby enable monitoring of disease response over time in terms of organ-specific amyloid load. The specific aim of this study is to measure visual and quantitative changes in organ-specific uptake of 124I-AT-01 from baseline, in patients with systemic amyloidosis, using PET/CT imaging
Outcome measures
| Measure |
All Participants
n=19 Participants
Experimental: Patients will receive a single IV injection of 124I-AT-01 radiotracer and PET/CT imaging In this repeat imaging study, patients previously enrolled in the AMY1001 study, in whom positive PET/CT imaging findings were observed will undergo repeat imaging to assess changes in radiotracer uptake in the liver, spleen, heart and kidneys.
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To Measure Visual Changes and Quantitative Changes in Organ-specific Uptake of 124I-AT-01
Heart · No change in visual uptake
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16 Participants
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To Measure Visual Changes and Quantitative Changes in Organ-specific Uptake of 124I-AT-01
Heart · With visual increase
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1 Participants
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To Measure Visual Changes and Quantitative Changes in Organ-specific Uptake of 124I-AT-01
Heart · Decrease in visual uptake
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2 Participants
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To Measure Visual Changes and Quantitative Changes in Organ-specific Uptake of 124I-AT-01
Liver · No change in visual uptake
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12 Participants
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To Measure Visual Changes and Quantitative Changes in Organ-specific Uptake of 124I-AT-01
Liver · With visual increase
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1 Participants
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To Measure Visual Changes and Quantitative Changes in Organ-specific Uptake of 124I-AT-01
Liver · Decrease in visual uptake
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6 Participants
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To Measure Visual Changes and Quantitative Changes in Organ-specific Uptake of 124I-AT-01
Spleen · No change in visual uptake
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14 Participants
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To Measure Visual Changes and Quantitative Changes in Organ-specific Uptake of 124I-AT-01
Spleen · With visual increase
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0 Participants
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To Measure Visual Changes and Quantitative Changes in Organ-specific Uptake of 124I-AT-01
Spleen · Decrease in visual uptake
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5 Participants
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To Measure Visual Changes and Quantitative Changes in Organ-specific Uptake of 124I-AT-01
Kidney · No change in visual uptake
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14 Participants
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To Measure Visual Changes and Quantitative Changes in Organ-specific Uptake of 124I-AT-01
Kidney · With visual increase
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4 Participants
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To Measure Visual Changes and Quantitative Changes in Organ-specific Uptake of 124I-AT-01
Kidney · Decrease in visual uptake
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1 Participants
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OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline (assessed during previous study NCT03678259) and Repeat Assessment (Day 1 to Day 2 of current study)Population: For serum biomarker (NT-proBNP), correlations are reported for both the absolute repeat values and the change from baseline. Because echocardiographic data was not available from the baseline imaging session, the correlation for echocardiographic parameters was performed using absolute values at repeat imaging.
For AL, ATTR and all patients, we will correlate visual and quantitative changes in 124I- AT-01 uptake, from baseline, in the heart, liver and kidney with echocardiographic parameters (including left ventricular wall thickness (cm), intraventricular wall thickness (cm), and global logitudinal strain (%)) and serum biomarker levels (including NTproBNP, eGFR, creatinine, AST and ALT) assessed prior to imaging and from data recorded in the patients' medical record.
Outcome measures
| Measure |
All Participants
n=19 Participants
Experimental: Patients will receive a single IV injection of 124I-AT-01 radiotracer and PET/CT imaging In this repeat imaging study, patients previously enrolled in the AMY1001 study, in whom positive PET/CT imaging findings were observed will undergo repeat imaging to assess changes in radiotracer uptake in the liver, spleen, heart and kidneys.
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Measures of Interest
Cardiac SUVR change (%) vs NTproBNP change (%)
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0.57 spearman correlation coefficient
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Measures of Interest
Scaled cardiac SUVR change (%) vs NTproBNP change (%)
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0.62 spearman correlation coefficient
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Measures of Interest
Cardiac SUVR change vs NTproBNP change (pg/mL)
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0.43 spearman correlation coefficient
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Measures of Interest
correlation with cardiac SUVR - Left Ventricular (LV) wall thickness (cm) (Repat Imaging)
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0.76 spearman correlation coefficient
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Measures of Interest
Correlation with cardiac SUVR - interventricular septum thickness (cm) (Repeat Imaging)
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0.68 spearman correlation coefficient
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Measures of Interest
Correlation with cardiac SUVR - global longitudinal strain (GLS)(%) (Repeat Imaging)
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0.54 spearman correlation coefficient
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Measures of Interest
Correlation with cardiac SUVR - NTproBNP (pg/mL) (Repeat Imaging)
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0.65 spearman correlation coefficient
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Measures of Interest
Correlation with renal SUVR - estimated glomerular filtration rate (eGFR) (Repeat Imaging)
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-0.43 spearman correlation coefficient
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Measures of Interest
Correlation with renal SUVR - Creatinine (Repat Imaging)
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0.47 spearman correlation coefficient
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Measures of Interest
Correlation with renal SUVR - blood urea nitrogen (BUN) (Repeat Imaging)
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0.25 spearman correlation coefficient
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Measures of Interest
Correlation with renal SUVR - Urinary albumin/cre ratio (mg/g cre) (Repeat Imaging)
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0.00 spearman correlation coefficient
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Measures of Interest
Correlation with hepatic SUVR - Aspartate Aminotransferase (AST) (Repeat Imaging)
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0.06 spearman correlation coefficient
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Measures of Interest
Correlation with hepatic SUVR - Alanine transaminase (ALT) (Repeat Imaging)
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0.18 spearman correlation coefficient
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Measures of Interest
Correlation with hepatic SUVR - Alkaline phosphatase (Repeat Imaging)
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0.29 spearman correlation coefficient
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OTHER_PRE_SPECIFIED outcome
Timeframe: From enrollment to the end of the study is 8 daysPopulation: No echocardiographic data was available from the baseline imaging session with which to compare changes in the quantitative uptake and there was no contemporaneous echocardiography data in the medical record contemporaneous with baseline imaging
For AL, ATTR and all patients, we will correlate visual and quantitative changes in organ-specific 124I-AT-01 uptake (percent change in radiotracer uptake from baseline) with change in disease status (progression, response, or stable state) when this can be established from data (serum biomarkers and cardiac imaging parameters) in the patients' medical record or collected during the AMY1001 trial. Disease status is categorized based on NTproBNP, creatinine, blood urea nitrogen or alkaline phosphatase compared to baseline values taken at the original scan time or noted in the medical record based on a sum of clinical findings. Progression is defined as a ≥30% increase; Response is defined as a ≥30% decrease; and Stable disease is defined as a \<30% increase or decrease.
Outcome measures
| Measure |
All Participants
Experimental: Patients will receive a single IV injection of 124I-AT-01 radiotracer and PET/CT imaging In this repeat imaging study, patients previously enrolled in the AMY1001 study, in whom positive PET/CT imaging findings were observed will undergo repeat imaging to assess changes in radiotracer uptake in the liver, spleen, heart and kidneys.
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Measure of Interest
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0 Participants
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OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline (assessed during previous study NCT03678259) and Repeat Assessment (Day 1 to Day 2 of current study)Population: Of the 19 total enrolled participants, this analysis is limited to the 9 patients with AL amyloidosis because evaluating the difference between involved and uninvolved serum free light chains (dFLC) is clinically relevant only to that cohort. Correlation (Spearman) analyses were performed to assess the relationship between the change in radiotracer uptake (SUVR) and changes in dFLC and the free κ/λ ratio.
For AL patients, correlate visual and quantitative changes in organ-specific 124I-AT-01 uptake (percent change in radiotracer uptake from baseline) with change in serum free light chains over the period between imaging studies, when available in the medical record. Baseline assessments reflect the values assessed during the previous study (NCT03678259). Repeat assessments reflect the values assessed during this current study. The 'change in' values are calculated as the repeat value from this current study minus the baseline value from the previous study (NCT03678259).
Outcome measures
| Measure |
All Participants
n=9 Participants
Experimental: Patients will receive a single IV injection of 124I-AT-01 radiotracer and PET/CT imaging In this repeat imaging study, patients previously enrolled in the AMY1001 study, in whom positive PET/CT imaging findings were observed will undergo repeat imaging to assess changes in radiotracer uptake in the liver, spleen, heart and kidneys.
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Measure of Interest
Heart SUVRmean change (%) - dFLC change
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-0.262 spearman correlation coefficient
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Measure of Interest
Heart SUVRmean change (%) - κ/λ ratio change
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-0.619 spearman correlation coefficient
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Measure of Interest
Change in SUVRmean absolute - dFLC change
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-0.252 spearman correlation coefficient
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Measure of Interest
Change in SUVRmean absolute - κ/λ ratio change
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-0.515 spearman correlation coefficient
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Measure of Interest
Heart SUVRmean change (%) - Baseline dFLC
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-0.405 spearman correlation coefficient
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Measure of Interest
Heart SUVRmean change (%) - Repeat dFLC
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-0.929 spearman correlation coefficient
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Measure of Interest
Change in SUVRmean absolute - Baseline dFLC
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-0.491 spearman correlation coefficient
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Measure of Interest
Change in SUVRmean absolute - Repeat dFLC
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-0.970 spearman correlation coefficient
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OTHER_PRE_SPECIFIED outcome
Timeframe: From enrollment to the end of the study is 8 daysPopulation: Software not available to perform quantitation
Compare changes, when possible, in radiotracer distribution from baseline based on analysis dynamic PET data.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: From enrollment to the end of the study is 8 daysPopulation: Software not available to perform quantitation
Compare changes, when possible, in ejection fraction from baseline based on analysis dynamic PET data.
Outcome measures
| Measure |
All Participants
Experimental: Patients will receive a single IV injection of 124I-AT-01 radiotracer and PET/CT imaging In this repeat imaging study, patients previously enrolled in the AMY1001 study, in whom positive PET/CT imaging findings were observed will undergo repeat imaging to assess changes in radiotracer uptake in the liver, spleen, heart and kidneys.
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Measure of Interest
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0 Participants
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Adverse Events
All Participants
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
All Participants
n=19 participants at risk
Experimental: Patients will receive a single IV injection of 124I-AT-01 radiotracer and PET/CT imaging
In this repeat imaging study, patients previously enrolled in the AMY1001 study, in whom positive PET/CT imaging findings were observed will undergo repeat imaging to assess changes in radiotracer uptake in the liver, spleen, heart and kidneys.
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Musculoskeletal and connective tissue disorders
Arthralgia
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5.3%
1/19 • From enrollment to the end of the study is 8 days
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Additional Information
Jonathan Wall, PhD
University of Tennessee Graduate School of Medicine
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place