Recruiting

TTR Stabilizing Therapy

Sponsor:

University of Texas Southwestern Medical Center

Code:

NCT07196839

Conditions

Transthyretin (TTR) Amyloid Cardiomyopathy

Eligibility Criteria

Sex: All

Age: 30 - 70+

Healthy Volunteers: Not accepted

Study Details

Brief 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 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.

Conditions

Transthyretin (TTR) Amyloid Cardiomyopathy

Study ID

NCT07196839

Start date

Dec 31, 2025

Status verified date

Jan, 2026

Completion date

Mar, 2027

Anticipated

Primary completion date

Dec, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 30 - 70+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

50 participants

Anticipated

Interventions and Outcome Measures

Arms

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

Primary outcome measure

  • Serial blood levels of circulating TTR amyloid aggregates (TAAs) [ Time Frame: Baseline, 1 month (+/- 5 days), and 3 months (+/- 5 days). ]

Central Contacts and Locations

Central contacts

Jerah Sanchez

214-645-7303jerahmarie.sanchez@utsouthwestern.edu

Amy Browning

Amy.Browning@utsouthwestern.edu

Locations

UT Southwestern Medical Center

Recruiting

Dallas, Texas, United States, 75248

Contacts

Jerah Sanchez

214-645-7303jerahmarie.sanchez@utsouthwestern.edu

Principal Investigator:

Justin Grodin, MD MPH

More Information

Sponsor

University of Texas Southwestern Medical Center

Last update posted

Jan 7, 2026

Last verified

Jan, 2026

Keywords

  • transthyretin
  • tafamidis
  • acoramidis
  • amyloidosis

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by University of Texas Southwestern Medical Center on 2026-01-07.