Transthyretin (TTR) Amyloid CardiomyopathyNCT07196839
Determining the Association of TTR Stabilizing Therapy With Circulating TTR Amyloid Aggregates Over Time in Patients With ATTR-CM: Longitudinal Biomarker Study
An observational study of Transthyretin (TTR) Amyloid Cardiomyopathy, run by University of Texas Southwestern Medical Center at 1 site in United States.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 30 to 80 years
- Conditions the study is for
- Transthyretin (TTR) Amyloid Cardiomyopathy
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Dec 2025to Mar 2027
- Phase
- Observational
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 30 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: ATTR-CA patients with a diagnosis of heart failure.
Who may be able to join
- Men and women ages 30-80 who have symptomatic ATTR-CA as determined by a history of HF (this will be assessed by study personnel and defined as : 1) history of hospitalization within the previous 12 months for management of HF; 2) an elevated B-type natriuretic peptide level ≥100 pg/mL or NT-proBNP ≥360 pg/mL within the previous 12 months; or 3) a clinical diagnosis of HF from a treating clinician)
- ATTR-CA previously diagnosed histologically by amyloid staining and tissue typing with immunohistochemistry or mass spectrometry or by bone scintigraphy in without abnormal M-protein
- TTR gene sequencing confirming the TTR genotype has resulted or is pending
- Enrollment will be stratified by n/N=30/50 starting on-label TTR-stabilizing therapy (e.g. tafamidis) within 5 days after enrollment or by n/N=20/50 of those currently taking TTR-stabilizing therapy
Who cannot join
- Other known causes of cardiomyopathy
- History of light-chain cardiac amyloidosis
- Cardiac transplantation
- Liver transplantation
- Has taken patisiran in the past 90 days, or inotersen in the past 180 days, has ever taken vutrisiran, or is participating in a clinical trial for ATTR treatments
- Estimated glomerular filtration rate ≤30 mL/min/1.73 m2
- Anticipated gaps in ATTR-CA treatment for 3 months after enrollment
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Amy Browning contact
United States1 site, 1 recruiting
- Recruiting
UT Southwestern Medical Center
Dallas, Texas, 75248
Jerah Sanchez, 214-645-7303, jerahmarie.sanchez@utsouthwestern.edu
Justin Grodin, MD MPH
About this study
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.
- Keywords
- transthyretin, tafamidis, acoramidis, amyloidosis
- Medical subject headings
- Amyloid Neuropathies, Familial, Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline, 1 month (+/- 5 days), and 3 months (+/- 5 days).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2025
- Study start31 Dec 2025
- Primary completionDec 2026 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Transthyretin (TTR) Amyloid CardiomyopathyStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Texas Southwestern Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Principal investigator, Justin Grodin, University of Texas Southwestern Medical Center
- Investigators
- Justin Grodin, MD MPH, Principal investigator, University of Texas Southwestern Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07196839
- Sponsor's protocol ID
- STU-2024-1119
- Status last verified
- Jan 2026
- First submitted
- 19 Sep 2025
- Last updated
- 7 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
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.
PubMed 38768274