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| Name | Class |
|---|---|
| Akcea Therapeutics | INDUSTRY |
| Pfizer | INDUSTRY |
| Alnylam Pharmaceuticals | INDUSTRY |
| Eidos Therapeutics, a BridgeBio company |
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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.
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:
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 99mTc-pyrophosphate Scintigraphy | Diagnostic Test | Cardiac Imaging Technique used to diagnose Transthyretin Cardiac Amyloidosis by use of 15 mCi of 99mTC-Pyrophosphate tracer |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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) |
| Measure | Description | Time Frame |
|---|---|---|
| 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) |
| Impact of HF as assessed by Kansas City Cardiomyopathy Questionnaire |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Presence of carpal tunnel syndrome |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Abbie Macher, BS | Contact | 617-643-6328 | ajmacher@mgh.harvard.edu | |
| Laura Stockhausen, BS | Contact | 617-724-1339 | lstockhausen@mgh.harvard.edu |
| Name | Affiliation | Role |
|---|---|---|
| Hanna K Gaggin, MD | Massachusetts General Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Massachusetts General Hospital | Recruiting | Boston | Massachusetts | 02114 | United States |
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| ID | Term |
|---|---|
| C567782 | Amyloidosis, Hereditary, Transthyretin-Related |
| D006333 | Heart Failure |
| D054144 | Heart Failure, Diastolic |
| D000686 | Amyloidosis |
| ID | Term |
|---|---|
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
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| INDUSTRY |
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|
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) |
| 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) |
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 |
| 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 |
| 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 |
| 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 |
| D009750 |
| Nutritional and Metabolic Diseases |