Prevalence and Prediction of ATTR in Ambulatory Patients With HFpEF

Heart Failure Clinical Trial

— TTRinHFpEF  

Official title:

Prevalence and Prediction of Transthyretin Amyloidosis in Ambulatory Patients With Heart Failure With Preserved Ejection Fraction

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05577819
• Other study ID #: 2020P002061
• Status: Recruiting
• Phase: N/A
• Start date: October 2, 2020
• Est. completion date: December 31, 2024

Study information

• Verified date: May 2024
• Source: Massachusetts General Hospital
• Contact: Abbie Macher, BS
• Phone: 617-643-6328
• Email: ajmacher[@]mgh.harvard.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Recent studies have shown that transthyretin amyloidosis (ATTR) can sometimes cause a type of heart failure where the pumping function of the heart is normal, also known as Heart Failure with Preserved Ejection Fraction (HFpEF) or diastolic heart failure. In this single center diagnostic study, we will evaluate for ATTR in patients with HFpEF in order to to determine how frequently this occurs and how we can predict which heart failure patients may have TTR amyloidosis. Our goal is to identify amyloidosis in heart failure patients earlier so that they can start treatment.

Description:

Patients 65-years and older with HFpEF will be enrolled to participate in this single center, event driven (positive nuclear amyloid scan also known as 99mTc-pyrophosphate SPECT scan) study.

During the single study visit the following will be obtained:

• 99mTc-pyrophosphate SPECT scan

• Blood and DNA (optional) sample collection

• Questionnaires in regards to neuropathy, carpal tunnel, frailty, and Heart failure symptoms and how they may affect ones quality of life

• 6-Minute Walk Test

• ECG (electrocardiogram)

• Echocardiogram

Electronic health records will be reviewed for up to 5 years in order to determine hospitalization and survival of the study participants. Clinical outcomes of interest include a combined endpoint of days alive outside of the hospital from heart failure hospitalizations at one and five years, presence of autonomic neuropathy, presence of carpal tunnel syndrome, presence of polyneuropathy. Additionally, Individual clinical endpoints are also endpoints of interest.

The results from this study will be used to determine how frequently heart failure patients have transthyretin amyloidosis in their heart and better understand their symptoms. We hope that better understanding transthyretin amyloidosis in heart failure patients will help us identify affected patients so that they can receive treatment.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 515
• Est. completion date: December 31, 2024
• Est. primary completion date: June 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 65 Years and older

Eligibility

Inclusion Criteria:

• Patients with a confirmed diagnosis of HFpEF.

• Age =65 years old

Exclusion Criteria:

• End stage chronic kidney disease on dialysis (CKD stage 5 as defined as eGFR <15mL/min)

• no history of HFrEF (LVEF<40%) with the exception of low LVEF in the setting of acute decompensation, AF RVR, ACS/MI, etc

• Negative 99mTc-pyrophosphate scan within a year

• Unable to lie down for 15 minutes for the 99mTc-pyrophosphate scan

• Known diagnosis of amyloidosis

• Severe valvular heart disease that is uncorrected (moderate to severe is considered exclusionary)

Related Conditions & MeSH terms

• Amyloid Neuropathies, Familial
• Amyloidosis
• Heart Failure
• Heart Failure, Diastolic
• Transthyretin Amyloidosis

Intervention

Diagnostic Test:
• 99mTc-pyrophosphate Scintigraphy
Cardiac Imaging Technique used to diagnose Transthyretin Cardiac Amyloidosis by use of 15 mCi of 99mTC-Pyrophosphate tracer

Locations

Country	Name	City	State
United States	Massachusetts General Hospital	Boston	Massachusetts

Sponsors (5)

Lead Sponsor	Collaborator
Massachusetts General Hospital	Akcea Therapeutics, Alnylam Pharmaceuticals, Eidos Therapeutics, a BridgeBio company, Pfizer

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Presence of autonomic neuropathy	Medical records and phone follow-up with patients and/or their physicians will allow the investigators to ascertain vital status and any significant clinical events	Time from study Visit until the date of documented event up to 5 years after the study closure
Other	Presence of carpal tunnel syndrome	Medical records and phone follow-up with patients and/or their physicians will allow the investigators to ascertain vital status and any significant clinical events	Time from study Visit until the date of documented event up to 5 years after the study closure
Other	Presence of polyneuropathy	Medical records and phone follow-up with patients and/or their physicians will allow the investigators to ascertain vital status and any significant clinical events	Time from study Visit until the date of documented event up to 5 years after the study closure
Other	Combined endpoint of days alive outside of hospital from HF hospitalizations at one and five years	Medical records and phone follow-up with patients and/or their physicians will allow the investigators to ascertain vital status and any significant clinical events	Time from study Visit until the date of documented event up to 5 years after the study closure
Other	Individual endpoints of all-cause mortality, cardiovascular mortality, all-cause hospitalizations, cardiovascular hospitalizations and HF hospitalizations.	Medical records and phone follow-up with patients and/or their physicians will allow the investigators to ascertain vital status and any significant clinical events	Time from study Visit until the date of documented event up to 5 years after the study closure
Primary	Diagnosis of 99mTc-pyrophosphate scan positive ATTR	A cardiac disease in which misfolded proteins aggregate into amyloid fibril and deposit interstitially, leading to diastolic dysfunction and heart failure	Day 1 (day of study visit)
Secondary	New York Heart Association function classification	Classifies the severity of a patients Heart Failure in terms of their limitations during physical activity	Day 1 (day of study visit)
Secondary	Impact of HF as assessed by Kansas City Cardiomyopathy Questionnaire	Kansas City Cardiomyopathy Questionnaire is a questionnaire that determines in the impact of Heart Failure on their health status, such as quality of life, symptoms, function, etc.)	Day 1 (day of study visit)
Secondary	Exercise capacity as determined by a 6-minute walk test	The 6 minute walk test is used to determine an objective measurement of a patients aerobic capacity and endurance	Day 1 (day of study visit)