Massachusetts General Hospital in Boston is conducting a study to assess the presence of transthyretin amyloidosis (ATTR) in patients with heart failure with preserved ejection fraction (HFpEF).
The aim of the study is to predict which patients with heart failure might have ATTR so that appropriate treatment can be started as early as possible.
The single-center, interventional study will enroll up to 515 participants aged 65 or older with HFpEF. All participants will have a single study visit at which they will undergo a positive nuclear amyloid scan, also known as a 99mTc-pyrophosphate SPECT scan, as well as other tests, to determine the presence of ATTR. These tests include blood and an optional DNA sample collection, questionnaires related to symptoms and quality of life, a 6-minute walk test to determine exercise capacity, and an electrocardiogram and echocardiogram.
The main endpoints of interest are days alive outside the hospital following heart failure after 1 and 5 years, and the presence of autonomic neuropathy, carpal tunnel syndrome, and polyneuropathy. Additional outcomes of interest include individual endpoints such as all-cause mortality, cardiovascular mortality, all-cause hospitalizations, and heart failure hospitalizations.
Read more about ATTR diagnosis
To determine hospitalization and survival, the patients’ electronic health records will be reviewed for up to 5 years.
The study began on October 2, 2020, the estimated primary completion date is June 30, 2023, and the estimated completion date is December 31, 2023.
ATTR is a slowly progressive heart disease involving the aggregation and deposit of misfolded proteins known as amyloid into the interstitial space, which leads to diastolic disorders and heart failure. There are three main types of ATTR: (1) neuropathic, which affects the peripheral and autonomic nervous systems, (2) leptomeningeal, which affects the central nervous system, and (3) cardiac, which primarily affects the heart and leads to arrhythmia, cardiomegaly, or orthostatic hypertension.
Prevalence and prediction of ATTR in ambulatory patients with HFpEF (TTRinHFpEF). ClinicalTrials.gov. October 13, 2022. Updated October 13, 2022. Accessed October 18, 2022.