Clinical trial NCT05577819: Prevalence and Prediction of ATTR in Ambulatory Patients With HFpEF
Official title: Prevalence and Prediction of Transthyretin Amyloidosis in Ambulatory Patients With Heart Failure With Preserved Ejection Fraction
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Transthyretin Amyloidosis
Heart Failure
Heart Failure, Diastolic
Amyloidosis
Keywords: Transthyretin Amyloidosis
Heart Failure
Heart Failure, Diastolic
Amyloidosis
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: Akcea Therapeutics
name: Pfizer
name: Alnylam Pharmaceuticals
name: Eidos Therapeutics, a BridgeBio company
First posted: 2022-10-13
Enrollment: 515
Sex: ALL
Minimum age (years): 65
Standard ages: OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: DIAGNOSTIC

Brief 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.

Detailed 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.

Eligibility criteria: 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)

Primary outcomes:
measure: Diagnosis of 99mTc-pyrophosphate scan positive ATTR
timeFrame: Day 1 (day of study visit)
Secondary outcomes:
measure: New York Heart Association function classification
timeFrame: Day 1 (day of study visit)
measure: Impact of HF as assessed by Kansas City Cardiomyopathy Questionnaire
timeFrame: Day 1 (day of study visit)
measure: Exercise capacity as determined by a 6-minute walk test
timeFrame: Day 1 (day of study visit)
Other outcomes:
measure: Presence of autonomic neuropathy
timeFrame: Time from study Visit until the date of documented event up to 5 years after the study closure
measure: Presence of carpal tunnel syndrome
timeFrame: Time from study Visit until the date of documented event up to 5 years after the study closure
measure: Presence of polyneuropathy
timeFrame: Time from study Visit until the date of documented event up to 5 years after the study closure
measure: Combined endpoint of days alive outside of hospital from HF hospitalizations at one and five years
timeFrame: Time from study Visit until the date of documented event up to 5 years after the study closure
measure: Individual endpoints of all-cause mortality, cardiovascular mortality, all-cause hospitalizations, cardiovascular hospitalizations and HF hospitalizations.
timeFrame: Time from study Visit until the date of documented event up to 5 years after the study closure
Locations:
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
Study officials:
affiliation: Massachusetts General Hospital
name: Hanna K Gaggin, MD
role: PRINCIPAL_INVESTIGATOR
References: