Global Prevalence of ATTR-CM in Participants With HFpEF

Not Applicable

Terminated

• Conditions: Heart Failure With Preserved Ejection Fraction; Transthyretin Amyloid Cardiomyopathy

• Registration Number: NCT04424914
• Lead Sponsor: Pfizer

Brief Summary

This study is a global, multi-center study designed to estimate the global prevalence of transthyretin amyloid cardiomyopathy (ATTR-CM) within a clinically at risk population \[participants with heart failure with preserved ejection fraction (HFpEF)\].

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-05-27
• Study First Submitted QC: 2020-06-05
• Study First Posted: 2020-06-11
• Study Start: 2020-12-30
• Primary Completion: 2023-06-02
• Study Completion: 2023-06-02
• Status Verified: 2024-02
• Results First Submitted: 2024-02-01
• Results First Submitted QC: 2024-02-01
• Last Update Submitted: 2024-02-01
• Results First Posted: 2024-07-19
• Last Update Posted: 2024-07-19

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 347

Inclusion Criteria

1. Medical history of heart failure (HF) with:

   1. At least 1 episode with clinical evidence of HF (without hospitalization) by signs or symptoms of volume overload or elevated intracardiac pressures that required/requires treatment with a diuretic for improvement; OR
   2. 1 prior hospitalization for HF.

2. Left ventricular ejection fraction (LVEF) >40%.

3. End-diastolic interventricular septal wall thickness (IVST) ≥12 mm.

4. Willing and able to undergo scintigraphy.

Exclusion Criteria

1. Diagnosis of heart failure with reduced ejection fraction (HFrEF) (EF ≤40%).

2. Prior clinical history of myocardial infarction, CABG or multi-vessel obstructive coronary disease (>50% stenosis of ≥2 epicardial coronary arteries).

3. Presence or history of any severe valvular heart disease (obstructive or regurgitant).

4. A confirmed diagnosis of a non-amyloid infiltrative cardiomyopathy (ie, cardiac sarcoidosis, hemochromatosis), muscular dystrophies, cardiomyopathy with reversible causes, hypertrophic obstructive cardiomyopathy with known genetic etiology, or known pericardial constriction.

5. Any type of diagnosed amyloidosis (eg, amyloid A amyloidosis, primary [light chain] amyloidosis) or prior diagnosis of ATTR-CM.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Global Prevalence of ATTR-CM in HFpEF Participants Clinically At-Risk of Disease Among Total Evaluable Participants	Day 1	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.

Secondary Outcome Measures

Name	Time	Method
Number of Participants According to TTR Genotypes Among Participants Diagnosed With ATTR-CM	Day 1	Number of participants diagnosed with ATTR-CM were evaluated for TTR genotypes (wild-type or hereditary form).
Number of HFpEF Participants With and Without ATTR-CM Based on New York Heart Association (NYHA) Classification	Day 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.
Global Prevalence of ATTR-CM in Participants With HFpEF Clinically At-Risk of Disease by Subgroups (Regions, Age, Gender) Among Total Evaluable Participants	Day 1	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.
N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) in Participants With and Without ATTR-CM	Day 1	Participants were evaluated using NT-proBNP cardiac biomarker that was assessed at Day 1.

Trial Locations

Locations (50)

Eastern shore Research Institute LLC; 🇺🇸 Fairhope, Alabama, United States

Heart Center Research, LLC; 🇺🇸 Huntsville, Alabama, United States

Advance Medical Research Center; 🇺🇸 Miami, Florida, United States

Bioclinical Research Alliance Inc.; 🇺🇸 Miami, Florida, United States

Biogenix Molecular; 🇺🇸 Miami, Florida, United States

CIRA (Nuclear Imaging Facility); 🇺🇸 Miami, Florida, United States

Nucleotron/ Doral Imaging Institute, CIRA DBA; 🇺🇸 Miami, Florida, United States

Innova Pharma Research; 🇺🇸 Miami, Florida, United States

Ocala Cardiovascular Research; 🇺🇸 Ocala, Florida, United States

Chicago Medical Research, LLC; 🇺🇸 Hazel Crest, Illinois, United States

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