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| Name | Class |
|---|---|
| National Heart, Lung, and Blood Institute (NHLBI) | NIH |
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The objective of this study is to determine the association of clinically prescribed, on-label, TTR stabilizing therapy (e.g. tafamidis or acoramidis) with levels of circulating transthyretin amyloid aggregates (TAAs, a surrogate for amyloid disease activity) measured serially over time in patients with transthyretin cardiac amyloidosis (ATTR-CA). To accomplish this objective, the hypothesis that TTR stabilizing therapy will be associated lower circulating TAAs over time will be tested. Completion of this study will advance the understanding of the influence of ATTR-CA treatments on circulating evidence of amyloidosis and justify the role of blood testing to monitor treatment response in patients with ATTR-CA.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Taking on-label TTR stabilizing treatment | Taking on-label TTR stabilizing treatment for >14 days prior to enrollment | ||
| Initiating on-label TTR stabilizing treatment | Initiating on-label TTR stabilizing treatment within 5 days after enrollment |
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| Measure | Description | Time Frame |
|---|---|---|
| Serial blood levels of circulating TTR amyloid aggregates (TAAs) | The primary hypothesis is that TTR stabilizing therapy will lower circulating evidence of amyloidosis in patients with ATTR-CA from baseline to 3 months. In addition, the study team expects that there will be a time*treatment interaction identifying that treatment initiation will have a differential effect on serial levels of circulating s over time than those currently on treatment. These observations will support the role for measuring circulating TAAs to monitor treatment response in ATTR-CA. | Baseline, 1 month (+/- 5 days), and 3 months (+/- 5 days). |
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Inclusion Criteria:
Exclusion Criteria:
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ATTR-CA patients with a diagnosis of heart failure.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Jerah Sanchez | Contact | 214-645-7303 | jerahmarie.sanchez@utsouthwestern.edu | |
| Amy Browning | Contact | Amy.Browning@utsouthwestern.edu |
| Name | Affiliation | Role |
|---|---|---|
| Justin Grodin, MD MPH | University of Texas Southwestern Medical Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UT Southwestern Medical Center | Recruiting | Dallas | Texas | 75248 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38768274 | Background | Pedretti R, Wang L, Hanna M, Benson MD, Grodin JL, Tang WHW, Masri A, Saelices L. Detection of Circulating Transthyretin Amyloid Aggregates in Plasma: A Novel Biomarker for Transthyretin Amyloidosis. Circulation. 2024 May 21;149(21):1696-1699. doi: 10.1161/CIRCULATIONAHA.123.067225. Epub 2024 May 20. No abstract available. |
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Blood (plasma and serum)
| ID | Term |
|---|---|
| D028227 | Amyloid Neuropathies, Familial |
| D000686 | Amyloidosis |
| ID | Term |
|---|---|
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D009422 | Nervous System Diseases |
| D017772 | Amyloid Neuropathies |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D028226 | Amyloidosis, Familial |
| D008661 | Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D057165 | Proteostasis Deficiencies |
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