Transthyretin AmyloidosisTTRinHFpEFNCT05577819
Prevalence and Prediction of ATTR in Ambulatory Patients With HFpEF
A study testing 99mTc-pyrophosphate Scintigraphy in people with Transthyretin Amyloidosis and Heart Failure, run by Massachusetts General Hospital at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 65 years and older
- Conditions the study is for
- Transthyretin Amyloidosis, Heart Failure, Heart Failure, Diastolic
- What is being tested?
- 99mTc-pyrophosphate Scintigraphy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 515 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Oct 2020to 31 Dec 2026
- Phase
- Interventional
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 65 years and older
- All sexes
- No healthy volunteers
- Older adult
Who may be able to join
- Patients with a confirmed diagnosis of HFpEF.
- Age ≥65 years old
Who cannot join
- 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)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Massachusetts General Hospital
Boston, Massachusetts, 02114
Heather Jameson, PhD
Hanna Gaggin, MD
About this study
Official title: Prevalence and Prediction of Transthyretin Amyloidosis in Ambulatory Patients With Heart Failure With Preserved Ejection Fraction
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.
Read the 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.
- Being tested
- 99mTc-pyrophosphate Scintigraphy
- Keywords
- Transthyretin Amyloidosis, Heart Failure, Heart Failure, Diastolic, Amyloidosis
- Medical subject headings
- Amyloidosis, Hereditary, Transthyretin-Related, Heart Failure, Heart Failure, Diastolic, Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
99mTc-pyrophosphate ScintigraphyDiagnostic test
Also called PYP Scan, Pyrophosphate Scan
Cardiac Imaging Technique used to diagnose Transthyretin Cardiac Amyloidosis by use of 15 mCi of 99mTC-Pyrophosphate tracer
| Study group | Role | What they receive |
|---|---|---|
| Patients 65 and older with Heart Failure with Preserved Ejection Fraction | Experimental | Patients 65 years and older presenting to Massachusetts General Hospital with a known diagnosis of HFpEF and without a diagnosis of amyloidosis in the ambulatory (outpatient) setting will undergo a 99Tc-Pyrophosphate Scan to identify Cardiac Amyloidosis Receives: Diagnostic Test: 99mTc-pyrophosphate Scintigraphy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Day 1 (day of study visit)
Secondary outcomes (3)
New York Heart Association function classification
Classifies the severity of a patients Heart Failure in terms of their limitations during physical activity
Measured: Day 1 (day of study visit)
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.)
Measured: Day 1 (day of study visit)
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
Measured: Day 1 (day of study visit)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Oct 2022
- Study start2 Oct 2020
- Primary completion17 Jul 2026
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transthyretin AmyloidosisStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 515 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Akcea Therapeutics, Pfizer, Alnylam Pharmaceuticals, Eidos Therapeutics, a BridgeBio company
- Responsible party
- Principal investigator, Hanna Kim Gaggin, Massachusetts General Hospital
- Investigators
- Hanna K Gaggin, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05577819
- Sponsor's protocol ID
- 2020P002061
- Acronym
- TTRinHFpEF
- Status last verified
- Jul 2026
- First submitted
- 16 Jul 2021
- Last updated
- 1 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided