TTR stabilizers and blood transthyretin amyloid aggregates in ATTR-CM patients
Determining the Association of TTR Stabilizing Therapy With Circulating TTR Amyloid Aggregates Over Time in Patients With ATTR-CM: Longitudinal Biomarker Study
This research will test whether TTR-stabilizing drugs like tafamidis or acoramidis lower blood levels of transthyretin amyloid aggregates over time in people with ATTR-CM.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 50 (estimated) |
| Ages | 30 Years to 80 Years |
| Sex | All |
| Sponsor | University of Texas Southwestern Medical Center Academic / other |
| Locations | 1 site (Dallas, Texas) |
| Trial ID | NCT07196839 on ClinicalTrials.gov |
What this trial studies
This is a single-center, observational longitudinal biomarker study that will measure circulating transthyretin amyloid aggregates (TAAs) serially in patients with symptomatic transthyretin cardiac amyloidosis (ATTR-CM) who are on clinically prescribed, on‑label TTR-stabilizing therapy. Enrollment is stratified with about 30 participants starting TTR-stabilizing therapy within five days of enrollment and about 20 participants already taking such therapy, targeting roughly 50 total participants. Eligible participants are adults aged 30–80 with ATTR-CM confirmed by biopsy with mass spectrometry/immunohistochemistry or by bone scintigraphy without an abnormal M-protein, and with TTR genotyping available or pending. The study will correlate changes in blood TAA levels over time with treatment status and clinical markers to see if blood testing can monitor treatment response.
Who should consider this trial
Good fit: Adults aged 30–80 with symptomatic transthyretin cardiac amyloidosis confirmed by biopsy or bone scintigraphy (without abnormal M‑protein) who are either about to start or already taking on‑label TTR‑stabilizing therapy are ideal candidates.
Not a fit: Patients with non‑TTR amyloidosis (for example light‑chain amyloidosis), other known causes of cardiomyopathy, those outside the 30–80 age range, or those unable to attend serial visits are unlikely to benefit from this study.
Why it matters
Potential benefit: If successful, this could provide a simple blood test to track whether TTR-stabilizing drugs are reducing amyloid activity and help guide treatment decisions.
How similar studies have performed: Large randomized trials have shown clinical benefit of tafamidis in ATTR-CM, but using serial circulating TTR amyloid aggregate measurements as a blood biomarker for treatment response is relatively new and has limited prior validation.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Exclusion Criteria: * 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
Where this trial is running
Dallas, Texas
- UT Southwestern Medical Center — Dallas, Texas, United States (Recruiting)
Study contacts
- Principal investigator: Justin Grodin, MD MPH — University of Texas Southwestern Medical Center
- Study coordinator: Jerah Sanchez
- Email: jerahmarie.sanchez@utsouthwestern.edu
- Phone: 214-645-7303
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.