Trial Outcomes & Findings for Global Prevalence of ATTR-CM in Participants With HFpEF (NCT NCT04424914)
NCT ID: NCT04424914
Last Updated: 2024-07-19
Results Overview
Global prevalence of ATTR-CM in HFpEF participants was obtained by dividing the number of participants who were diagnosed with ATTR-CM in the study by the total number of HFpEF participants evaluated. Diagnosis of ATTR-CM was defined as: cardiac scintigraphy Grade 1, with confirmation of ATTR by cardiac biopsy; or cardiac scintigraphy Grade 2 or above. Exact 95% confidence intervals for the prevalence estimates were calculated using the method of Clopper and Pearson.
Recruitment status
TERMINATED
Study phase
NA
Target enrollment
347 participants
Primary outcome timeframe
Day 1
Results posted on
2024-07-19
Participant Flow
A total of 347 heart failure with preserved ejection fraction (HFpEF) participants within a clinically at-risk population were enrolled.
Participant milestones
| Measure |
Participants With ATTR-CM
Participants enrolled in the study who were positive for transthyretin amyloid cardiomyopathy (ATTR-CM) by scintigraphy and were evaluable.
|
Participants Without ATTR-CM
Participants enrolled in the study who were negative for ATTR-CM by scintigraphy and were evaluable.
|
Non-evaluable Participants
Participants enrolled in the study who were non-evaluable.
|
|---|---|---|---|
|
Overall Study
STARTED
|
56
|
259
|
32
|
|
Overall Study
COMPLETED
|
27
|
251
|
3
|
|
Overall Study
NOT COMPLETED
|
29
|
8
|
29
|
Reasons for withdrawal
| Measure |
Participants With ATTR-CM
Participants enrolled in the study who were positive for transthyretin amyloid cardiomyopathy (ATTR-CM) by scintigraphy and were evaluable.
|
Participants Without ATTR-CM
Participants enrolled in the study who were negative for ATTR-CM by scintigraphy and were evaluable.
|
Non-evaluable Participants
Participants enrolled in the study who were non-evaluable.
|
|---|---|---|---|
|
Overall Study
Other
|
0
|
0
|
28
|
|
Overall Study
Withdrawal by Subject
|
3
|
0
|
0
|
|
Overall Study
Study terminated by sponsor
|
23
|
7
|
0
|
|
Overall Study
Lost to Follow-up
|
0
|
0
|
1
|
|
Overall Study
Death
|
3
|
1
|
0
|
Baseline Characteristics
Global Prevalence of ATTR-CM in Participants With HFpEF
Baseline characteristics by cohort
| Measure |
Participants With ATTR-CM
n=56 Participants
Participants enrolled in the study who were positive for transthyretin amyloid cardiomyopathy (ATTR-CM) by scintigraphy and were evaluable.
|
Participants Without ATTR-CM
n=259 Participants
Participants enrolled in the study who were negative for ATTR-CM by scintigraphy and were evaluable.
|
Non-evaluable Participants
n=32 Participants
Participants enrolled in the study who were non-evaluable.
|
Total
n=347 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
84.13 Years
STANDARD_DEVIATION 6.64 • n=5 Participants
|
76.52 Years
STANDARD_DEVIATION 7.87 • n=7 Participants
|
75.56 Years
STANDARD_DEVIATION 7.51 • n=5 Participants
|
77.66 Years
STANDARD_DEVIATION 8.15 • n=4 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=5 Participants
|
129 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
164 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
42 Participants
n=5 Participants
|
130 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
183 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
22 Participants
n=5 Participants
|
99 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
130 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
33 Participants
n=5 Participants
|
158 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
214 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
20 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
23 Participants
n=4 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
|
|
Race (NIH/OMB)
Black or African American
|
6 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
23 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
48 Participants
n=5 Participants
|
226 Participants
n=7 Participants
|
25 Participants
n=5 Participants
|
299 Participants
n=4 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
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Day 1Population: Evaluable analysis set included all participants who met the inclusion/exclusion criteria and completed Visit 1, excluding participants with cardiac scintigraphy assessed as non-evaluable.
Global prevalence of ATTR-CM in HFpEF participants was obtained by dividing the number of participants who were diagnosed with ATTR-CM in the study by the total number of HFpEF participants evaluated. Diagnosis of ATTR-CM was defined as: cardiac scintigraphy Grade 1, with confirmation of ATTR by cardiac biopsy; or cardiac scintigraphy Grade 2 or above. Exact 95% confidence intervals for the prevalence estimates were calculated using the method of Clopper and Pearson.
Outcome measures
| Measure |
Participants With HFpEF
n=315 Participants
All participants diagnosed with HFpEF who were noted with and without ATTR-CM.
|
Participants Without ATTR-CM
Participants enrolled in the study who were negative for ATTR-CM by scintigraphy.
|
|---|---|---|
|
Global Prevalence of ATTR-CM in HFpEF Participants Clinically At-Risk of Disease Among Total Evaluable Participants
|
17.8 Percentage of participants
Interval 13.72 to 22.46
|
—
|
SECONDARY outcome
Timeframe: Day 1Population: Evaluable analysis set included all participants who met the inclusion/exclusion criteria and completed Visit 1, excluding participants with cardiac scintigraphy assessed as non-evaluable. Here, "Number Analyzed" signifies number of participants evaluable for specified rows of the arm.
The prevalence of ATTR-CM in HFpEF participants was evaluated for subgroups including regions (North America, Europe, and Asia), age categories (60-64 years, 65-69 years, 70-74 years, 75-79 years, 80-85 years, 85-89 years, \>=90 years), and gender (male, female). The estimate of prevalence was defined as the number of participants meeting the diagnosis criteria of ATTR-CM divided by the number of evaluable participants in the study in the given subgroup category. Diagnosis of ATTR-CM was defined as: cardiac scintigraphy Grade 1, with confirmation of ATTR by cardiac biopsy; or cardiac scintigraphy Grade 2 or above. Exact 95% confidence intervals for the prevalence estimates were calculated using the method of Clopper and Pearson.
Outcome measures
| Measure |
Participants With HFpEF
n=315 Participants
All participants diagnosed with HFpEF who were noted with and without ATTR-CM.
|
Participants Without ATTR-CM
Participants enrolled in the study who were negative for ATTR-CM by scintigraphy.
|
|---|---|---|
|
Global Prevalence of ATTR-CM in Participants With HFpEF Clinically At-Risk of Disease by Subgroups (Regions, Age, Gender) Among Total Evaluable Participants
Region: Europe
|
23.81 Percentage of participants
Interval 18.22 to 30.16
|
—
|
|
Global Prevalence of ATTR-CM in Participants With HFpEF Clinically At-Risk of Disease by Subgroups (Regions, Age, Gender) Among Total Evaluable Participants
Region: North America
|
4.82 Percentage of participants
Interval 1.33 to 11.88
|
—
|
|
Global Prevalence of ATTR-CM in Participants With HFpEF Clinically At-Risk of Disease by Subgroups (Regions, Age, Gender) Among Total Evaluable Participants
Region: Asia
|
9.09 Percentage of participants
Interval 1.12 to 29.16
|
—
|
|
Global Prevalence of ATTR-CM in Participants With HFpEF Clinically At-Risk of Disease by Subgroups (Regions, Age, Gender) Among Total Evaluable Participants
Age: 60-64 years
|
0 Percentage of participants
Interval 0.0 to 0.0
|
—
|
|
Global Prevalence of ATTR-CM in Participants With HFpEF Clinically At-Risk of Disease by Subgroups (Regions, Age, Gender) Among Total Evaluable Participants
Age: 65-69 years
|
5.88 Percentage of participants
Interval 0.72 to 19.68
|
—
|
|
Global Prevalence of ATTR-CM in Participants With HFpEF Clinically At-Risk of Disease by Subgroups (Regions, Age, Gender) Among Total Evaluable Participants
Age: 70-74 years
|
5.77 Percentage of participants
Interval 1.21 to 15.95
|
—
|
|
Global Prevalence of ATTR-CM in Participants With HFpEF Clinically At-Risk of Disease by Subgroups (Regions, Age, Gender) Among Total Evaluable Participants
Age: 75-79 years
|
14.71 Percentage of participants
Interval 7.28 to 25.39
|
—
|
|
Global Prevalence of ATTR-CM in Participants With HFpEF Clinically At-Risk of Disease by Subgroups (Regions, Age, Gender) Among Total Evaluable Participants
Age: 80-84 years
|
13.43 Percentage of participants
Interval 6.33 to 23.97
|
—
|
|
Global Prevalence of ATTR-CM in Participants With HFpEF Clinically At-Risk of Disease by Subgroups (Regions, Age, Gender) Among Total Evaluable Participants
Age: 85-89 years
|
44.44 Percentage of participants
Interval 30.92 to 58.6
|
—
|
|
Global Prevalence of ATTR-CM in Participants With HFpEF Clinically At-Risk of Disease by Subgroups (Regions, Age, Gender) Among Total Evaluable Participants
Age: >=90 years
|
42.11 Percentage of participants
Interval 20.25 to 66.5
|
—
|
|
Global Prevalence of ATTR-CM in Participants With HFpEF Clinically At-Risk of Disease by Subgroups (Regions, Age, Gender) Among Total Evaluable Participants
Gender: Female
|
9.79 Percentage of participants
Interval 5.46 to 15.88
|
—
|
|
Global Prevalence of ATTR-CM in Participants With HFpEF Clinically At-Risk of Disease by Subgroups (Regions, Age, Gender) Among Total Evaluable Participants
Gender: Male
|
24.42 Percentage of participants
Interval 18.2 to 31.54
|
—
|
SECONDARY outcome
Timeframe: Day 1Population: Evaluable analysis set included all participants who met the inclusion/exclusion criteria and completed Visit 1, excluding participants with cardiac scintigraphy assessed as non-evaluable.
Number of participants diagnosed with ATTR-CM were evaluated for TTR genotypes (wild-type or hereditary form).
Outcome measures
| Measure |
Participants With HFpEF
n=56 Participants
All participants diagnosed with HFpEF who were noted with and without ATTR-CM.
|
Participants Without ATTR-CM
Participants enrolled in the study who were negative for ATTR-CM by scintigraphy.
|
|---|---|---|
|
Number of Participants According to TTR Genotypes Among Participants Diagnosed With ATTR-CM
TTR Genotype: Wild-Type
|
47 Participants
|
—
|
|
Number of Participants According to TTR Genotypes Among Participants Diagnosed With ATTR-CM
TTR Genotype: Variant
|
7 Participants
|
—
|
|
Number of Participants According to TTR Genotypes Among Participants Diagnosed With ATTR-CM
TTR Genotype: Not reported
|
2 Participants
|
—
|
SECONDARY outcome
Timeframe: Day 1Population: Enrolled analysis set included all participants who met the inclusion/exclusion criteria and completed Visit 1.
Participants were evaluated using the NYHA classification at Day 1. Class I: participants with cardiac disease but without resulting limitations of physical activity. Class II: participants with cardiac disease resulting in slight limitation of physical activity. Class III: participants with cardiac disease resulting in marked limitation of physical activity. Class IV: participants with cardiac disease resulting in inability to carry on any physical activity without discomfort.
Outcome measures
| Measure |
Participants With HFpEF
n=56 Participants
All participants diagnosed with HFpEF who were noted with and without ATTR-CM.
|
Participants Without ATTR-CM
n=259 Participants
Participants enrolled in the study who were negative for ATTR-CM by scintigraphy.
|
|---|---|---|
|
Number of HFpEF Participants With and Without ATTR-CM Based on New York Heart Association (NYHA) Classification
NYHA Classification: Class I+II
|
45 Participants
|
191 Participants
|
|
Number of HFpEF Participants With and Without ATTR-CM Based on New York Heart Association (NYHA) Classification
NYHA Classification: Class I
|
2 Participants
|
26 Participants
|
|
Number of HFpEF Participants With and Without ATTR-CM Based on New York Heart Association (NYHA) Classification
NYHA Classification: Class II
|
43 Participants
|
165 Participants
|
|
Number of HFpEF Participants With and Without ATTR-CM Based on New York Heart Association (NYHA) Classification
NYHA Classification: Class III
|
8 Participants
|
66 Participants
|
|
Number of HFpEF Participants With and Without ATTR-CM Based on New York Heart Association (NYHA) Classification
NYHA Classification: Class IV
|
3 Participants
|
2 Participants
|
|
Number of HFpEF Participants With and Without ATTR-CM Based on New York Heart Association (NYHA) Classification
NYHA Classification: Class III+IV
|
11 Participants
|
68 Participants
|
SECONDARY outcome
Timeframe: Day 1Population: Enrolled analysis set included all participants who met the inclusion/exclusion criteria and completed Visit 1. Here, "Number of Participants Analyzed" signifies number of participants evaluable for this outcome measure.
Participants were evaluated using NT-proBNP cardiac biomarker that was assessed at Day 1.
Outcome measures
| Measure |
Participants With HFpEF
n=52 Participants
All participants diagnosed with HFpEF who were noted with and without ATTR-CM.
|
Participants Without ATTR-CM
n=240 Participants
Participants enrolled in the study who were negative for ATTR-CM by scintigraphy.
|
|---|---|---|
|
N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) in Participants With and Without ATTR-CM
|
2470.0 Nanogram per liter (ng/L)
Interval 184.0 to 18449.0
|
817.5 Nanogram per liter (ng/L)
Interval 36.0 to 29370.0
|
Adverse Events
Participants With ATTR-CM
Serious events: 0 serious events
Other events: 11 other events
Deaths: 3 deaths
Participants Without ATTR-CM
Serious events: 0 serious events
Other events: 10 other events
Deaths: 1 deaths
Non-evaluable Participants
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Participants With ATTR-CM
n=56 participants at risk
Participants enrolled in the study who were positive for ATTR-CM by scintigraphy and were evaluable.
|
Participants Without ATTR-CM
n=259 participants at risk
Participants enrolled in the study who were negative for ATTR-CM by scintigraphy and were evaluable.
|
Non-evaluable Participants
n=32 participants at risk
Participants enrolled in the study who were non-evaluable.
|
|---|---|---|---|
|
Cardiac disorders
Bradycardia
|
3.6%
2/56 • Day 1 up to 6 months of follow-up
|
0.39%
1/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Cardiac disorders
Cardiac failure
|
3.6%
2/56 • Day 1 up to 6 months of follow-up
|
0.77%
2/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Cardiac disorders
Cardiac failure chronic
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Gastrointestinal disorders
Abdominal pain upper
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Gastrointestinal disorders
Constipation
|
3.6%
2/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
General disorders
Gravitational oedema
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
General disorders
Oedema peripheral
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Infections and infestations
Pneumonia
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Investigations
Blood pressure decreased
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Metabolism and nutrition disorders
Gout
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.39%
1/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Nervous system disorders
Carpal tunnel syndrome
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Nervous system disorders
Cognitive disorder
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Nervous system disorders
Dizziness
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Renal and urinary disorders
Acute kidney injury
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.39%
1/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Renal and urinary disorders
Chronic kidney disease
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
0.00%
0/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
3.1%
1/32 • Day 1 up to 6 months of follow-up
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/56 • Day 1 up to 6 months of follow-up
|
1.5%
4/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
3.1%
1/32 • Day 1 up to 6 months of follow-up
|
|
Surgical and medical procedures
Cardiac pacemaker insertion
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Surgical and medical procedures
Carpal tunnel decompression
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Surgical and medical procedures
Posterior lens capsulotomy
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.00%
0/259 • Day 1 up to 6 months of follow-up
|
0.00%
0/32 • Day 1 up to 6 months of follow-up
|
|
Vascular disorders
Hypotension
|
1.8%
1/56 • Day 1 up to 6 months of follow-up
|
0.39%
1/259 • Day 1 up to 6 months of follow-up
|
3.1%
1/32 • Day 1 up to 6 months of follow-up
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
- Publication restrictions are in place
Restriction type: OTHER