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Purpose

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.

Condition

Eligibility

Eligible Ages
Between 30 Years and 80 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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

Phase
Study Type
Observational
Observational Model
Cohort
Time Perspective
Prospective

Arm Groups

ArmDescriptionAssigned Intervention
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

Recruiting Locations

UT Southwestern Medical Center
Dallas 4684888, Texas 4736286 75248
Contact:
Amy Browning
214-645-8040
Amy.Browning@utsouthwestern.edu

More Details

Status
Recruiting
Sponsor
University of Texas Southwestern Medical Center

Study Contact

Amy Browning
214-645-8040
Amy.Browning@utsouthwestern.edu

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.