Trial Outcomes & Findings for Evuzamitide in PET/CT to Measure Potential Therapeutic Response in ATTR (NCT NCT05635045)
NCT ID: NCT05635045
Last Updated: 2025-07-18
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
10 participants
Primary outcome timeframe
Up to one month after imaging
Results posted on
2025-07-18
Participant Flow
Participant milestones
| Measure |
Stabilizer Only
Subjects treated with tafamidis selected to re-scan to determine the change over time in Evuzamitide uptake
|
Silencer Only
Subjects treated with vutrisiran selected to re-scan to determine the change over time in Evuzamitide uptake
|
Stabilizer and Silencer (Tafamidis + Patisiran)
Subjects treated with tafamidis and patisiran selected to re-scan to determine the change over time in Evuzamitide uptake
|
Stabilizer and Silencer (Vutrisiran + Diflunisal)
Subjects treated with vutrisiran and diflunisal selected to re-scan to determine the change over time in Evuzamitide uptake
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
4
|
1
|
3
|
2
|
|
Overall Study
COMPLETED
|
4
|
1
|
2
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
1
|
0
|
Reasons for withdrawal
| Measure |
Stabilizer Only
Subjects treated with tafamidis selected to re-scan to determine the change over time in Evuzamitide uptake
|
Silencer Only
Subjects treated with vutrisiran selected to re-scan to determine the change over time in Evuzamitide uptake
|
Stabilizer and Silencer (Tafamidis + Patisiran)
Subjects treated with tafamidis and patisiran selected to re-scan to determine the change over time in Evuzamitide uptake
|
Stabilizer and Silencer (Vutrisiran + Diflunisal)
Subjects treated with vutrisiran and diflunisal selected to re-scan to determine the change over time in Evuzamitide uptake
|
|---|---|---|---|---|
|
Overall Study
measurements were not usable due material error in imaging agent supplied
|
0
|
0
|
1
|
0
|
Baseline Characteristics
Evuzamitide in PET/CT to Measure Potential Therapeutic Response in ATTR
Baseline characteristics by cohort
| Measure |
Stabilizer Only
n=4 Participants
Subjects treated with tafamidis selected to re-scan to determine the change over time in Evuzamitide uptake
|
Silencer Only
n=1 Participants
Subjects treated with vutrisiran selected to re-scan to determine the change over time in Evuzamitide uptake
|
Stabilizer and Silencer (Tafamidis + Patisiran)
n=3 Participants
Subjects treated with tafamidis and patisiran selected to re-scan to determine the change over time in Evuzamitide uptake
|
Stabilizer and Silencer (Vutrisiran + Diflunisal)
n=2 Participants
Subjects treated with vutrisiran and diflunisal selected to re-scan to determine the change over time in Evuzamitide uptake
|
Total
n=10 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Continuous
|
68.75 years
n=5 Participants
|
65 years
n=7 Participants
|
75 years
n=5 Participants
|
60 years
n=4 Participants
|
67.77 years
n=21 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
10 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
9 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
7 Participants
n=21 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
TTR Genotype
Wild-Type
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
5 Participants
n=21 Participants
|
|
TTR Genotype
V122i
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
|
TTR Genotype
Val30Met
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
TTR Genotype
Thr60Ile
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
TTR Genotype
Asp38Glu
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
PYP Scan result
PYP Score 0 - no uptake
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
PYP Scan result
PYP Score 1 - less uptake than bone
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
|
PYP Scan result
PYP Score 2 - as much uptake as bone
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
PYP Scan result
PYP Score 3 - more uptake than bone
|
3 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
6 Participants
n=21 Participants
|
|
PYP Scan result
PYP Score 2 or 3 - indeterminate whether uptake is equal to or greater than bone
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
PRIMARY outcome
Timeframe: Up to one month after imagingPopulation: 1 subject was scanned but their measurements were not usable due material error in imaging agent supplied.
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.
Outcome measures
| Measure |
Stabilizer Only
n=4 Participants
Subjects treated with tafamidis selected to re-scan to determine the change over time in Evuzamitide uptake
|
Silencer Only
n=1 Participants
Subjects treated with vutrisiran selected to re-scan to determine the change over time in Evuzamitide uptake
|
Stabilizer and Silencer (Tafamidis + Patisiran)
n=2 Participants
Subjects treated with tafamidis and patisiran selected to re-scan to determine the change over time in Evuzamitide uptake
|
Stabilizer and Silencer (Vutrisiran + Diflunisal)
n=2 Participants
Subjects treated with vutrisiran and diflunisal selected to re-scan to determine the change over time in Evuzamitide uptake
|
|---|---|---|---|---|
|
Median of Uptake of Evuzamitide as Measured by Volume of Interest in Milliliters
|
78.7 mL
Interval 13.4 to 137.8
|
253.6 mL
Interval 253.6 to 253.6
|
177.2 mL
Interval -31.2 to 385.5
|
39.6 mL
Interval 1.4 to 77.8
|
PRIMARY outcome
Timeframe: Up to one month after imagingPopulation: 1 subject was scanned but their measurements were not usable due material error in imaging agent supplied.
Median percentage change in injected dose (%ID), calculated as VOI mean activity concentration x VOI volume/injected activity.
Outcome measures
| Measure |
Stabilizer Only
n=4 Participants
Subjects treated with tafamidis selected to re-scan to determine the change over time in Evuzamitide uptake
|
Silencer Only
n=1 Participants
Subjects treated with vutrisiran selected to re-scan to determine the change over time in Evuzamitide uptake
|
Stabilizer and Silencer (Tafamidis + Patisiran)
n=2 Participants
Subjects treated with tafamidis and patisiran selected to re-scan to determine the change over time in Evuzamitide uptake
|
Stabilizer and Silencer (Vutrisiran + Diflunisal)
n=2 Participants
Subjects treated with vutrisiran and diflunisal selected to re-scan to determine the change over time in Evuzamitide uptake
|
|---|---|---|---|---|
|
Median of Change in Evuzamitide Uptake as Measured by Percentage Change in Injected Dose (%ID)
|
0.2 percent change
Interval -0.1 to 0.5
|
0.8 percent change
Interval 0.8 to 0.8
|
1.3 percent change
Interval 1.2 to 1.5
|
0.3 percent change
Interval 0.2 to 0.4
|
PRIMARY outcome
Timeframe: Up to one month after imagingPopulation: 1 subject was scanned but their measurements were not usable due material error in imaging agent supplied.
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.
Outcome measures
| Measure |
Stabilizer Only
n=4 Participants
Subjects treated with tafamidis selected to re-scan to determine the change over time in Evuzamitide uptake
|
Silencer Only
n=1 Participants
Subjects treated with vutrisiran selected to re-scan to determine the change over time in Evuzamitide uptake
|
Stabilizer and Silencer (Tafamidis + Patisiran)
n=2 Participants
Subjects treated with tafamidis and patisiran selected to re-scan to determine the change over time in Evuzamitide uptake
|
Stabilizer and Silencer (Vutrisiran + Diflunisal)
n=2 Participants
Subjects treated with vutrisiran and diflunisal selected to re-scan to determine the change over time in Evuzamitide uptake
|
|---|---|---|---|---|
|
Median of Uptake of Evuzamitide as Measured by Cardiac Amyloid Activity (CAA) in Grams
|
180.2 grams
Interval -75.9 to 437.8
|
852.8 grams
Interval 852.8 to 852.8
|
952.5 grams
Interval 812.5 to 1092.5
|
155.4 grams
Interval -18.7 to 329.4
|
Adverse Events
Stabilizer Only
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Silencer Only
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Stabilizer and Silencer (Tafamidis + Patisiran)
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Stabilizer and Silencer (Vutrisiran + Diflunisal)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Stabilizer Only
n=4 participants at risk
Subjects treated with tafamidis selected to re-scan to determine the change over time in Evuzamitide uptake
|
Silencer Only
n=1 participants at risk
Subjects treated with vutrisiran selected to re-scan to determine the change over time in Evuzamitide uptake
|
Stabilizer and Silencer (Tafamidis + Patisiran)
n=3 participants at risk
Subjects treated with tafamidis and patisiran selected to re-scan to determine the change over time in Evuzamitide uptake
|
Stabilizer and Silencer (Vutrisiran + Diflunisal)
n=2 participants at risk
Subjects treated with vutrisiran and diflunisal selected to re-scan to determine the change over time in Evuzamitide uptake
|
|---|---|---|---|---|
|
Skin and subcutaneous tissue disorders
Erythema
|
0.00%
0/4 • 28 days
|
0.00%
0/1 • 28 days
|
33.3%
1/3 • Number of events 1 • 28 days
|
0.00%
0/2 • 28 days
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place