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Determining the Association of TTR Stabilizing Therapy With Circulating TTR Amyloid Aggregates Over Time in Patients With ATTR-CM: Longitudinal Biomarker Study

NCT ID: NCT07196839

Last Updated: 2025-12-26

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

50 participants

Study Classification

OBSERVATIONAL

Study Start Date

2025-12-31

Study Completion Date

2027-03-31

Brief Summary

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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.

Detailed Description

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Conditions

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Transthyretin (TTR) Amyloid Cardiomyopathy

Keywords

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transthyretin tafamidis acoramidis amyloidosis

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Taking on-label TTR stabilizing treatment

Taking on-label TTR stabilizing treatment for \>14 days prior to enrollment

No interventions assigned to this group

Initiating on-label TTR stabilizing treatment

Initiating on-label TTR stabilizing treatment within 5 days after enrollment

No interventions assigned to this group

Eligibility Criteria

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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
Minimum Eligible Age

30 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role collaborator

University of Texas Southwestern Medical Center

OTHER

Sponsor Role lead

Responsible Party

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Justin Grodin

Associate Professor of Medicine

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Justin Grodin, MD MPH

Role: PRINCIPAL_INVESTIGATOR

University of Texas Southwestern Medical Center

Locations

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UT Southwestern Medical Center

Dallas, Texas, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Jerah Sanchez

Role: CONTACT

Phone: 214-645-7303

Email: [email protected]

Amy Browning

Role: CONTACT

Email: [email protected]

Facility Contacts

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Jerah Sanchez

Role: primary

References

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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.

Reference Type BACKGROUND
PMID: 38768274 (View on PubMed)

Other Identifiers

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STU-2024-1119

Identifier Type: -

Identifier Source: org_study_id