TTR stabilizers and blood transthyretin amyloid aggregates in ATTR-CM patients

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Liver and Coagulation Markers in ATTR Cardiac Amyloidosis

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• Acoramidis in adults with transthyretin amyloid cardiomyopathy in France

  This is an observational study to see how acoramidis is used in everyday care for adults with ATTR‑CM in France and how it affects their quality of life and daily function.

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• Acoramidis for transthyretin amyloid cardiomyopathy in routine care

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  Recruiting at 17 sites Bayer · Multiple Locations and 16 other locations · NCT07235462

• Coramitug (NNC6019-0001) for heart failure from transthyretin amyloid cardiomyopathy

  This study tests whether coramitug (NNC6019-0001) can lower the risk of heart-related death and illness in adults with transthyretin amyloid cardiomyopathy (ATTR-CM) while they continue their usual heart medicines.

  Recruiting at 237 sites Phase 3 Novo Nordisk A/S · Phoenix, Arizona and 298 other locations · NCT07207811

• Registry for studying Transthyretin Amyloid Cardiomyopathy in Thailand

  This study is collecting information from people with Transthyretin Amyloid Cardiomyopathy in Thailand to better understand the disease and improve early detection and treatment options.

  Recruiting Mahidol University · Bangkok and 7 other locations · NCT06338696

• Ocular Imaging to help detect ATTR-CM in adults with heart failure

  This multicenter pilot study tests whether eye imaging can help distinguish people with transthyretin amyloid cardiomyopathy from other heart failure patients.

  Recruiting AstraZeneca · Palo Alto, California and 19 other locations · NCT07690436

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.