ATTR-Cardiomyopathy Stabilization Following Tafamidis Therapy

January 25, 2021 updated by: Dr. Wojciech Mazur, The Christ Hospital

Transthyretin Amyloid Cardiomyopathy: Stabilization Assessed by Cardiac Magnetic Resonance

The study will investigate the stabilization effects of Tafamidis utilizing cardiac imaging cardiac magnetic resonance imaging (CMR). The investigators propose to pursue the following specific aims:

  1. Utilize cardiac magnetic resonance to assess stabilization of ATTR after Tafamidis therapy based on extracellular volume mapping.
  2. Investigate left ventricular myocardial mass, native T1, T2, and extracellular volume mapping after 12 month follow-up.
  3. Utilize cardiac magnetic resonance feature tracking at baseline and at 12 month follow-up.

Study Overview

Status

Recruiting

Intervention / Treatment

Detailed Description

The investigators hypothesize that participants with earlier stages (NYHA Class I and Class II) of ATTR wild type and ATTR mutant will demonstrate stabilization of ATTR following 1 year of Tafamidis.

The investigators propose to pursue the following specific aims:

  1. Utilize cardiac magnetic resonance to assess stabilization of ATTR after Tafamidis therapy based on extracellular volume mapping.
  2. Investigate left ventricular myocardial mass, native T1, T2, and extracellular volume mapping after 12 month follow-up.
  3. Utilize cardiac magnetic resonance feature tracking at baseline and at 12 month follow-up.

The investigators will enroll 131 participants with confirmed ATTR. Participants will be screened to exclude light chain amyloidosis by either measuring the proportion of kappa: lambda light chains with the serum free light chain assay, and tested for immunofixation electrophoresis of serum and urine. Once participants has confirmed diagnosis of ATTR (pyrophosphate scan positive scoring >1.5 ratio) and undergone baseline testing participants will be ask to enroll in the study. Genetic testing will performed to further distinguish between mutation and wild type. All participants will be required to sign informed consent agreeing to follow up testing at 1 year.

Participants will undergo a baseline cardiac magnetic resonance imaging for the purpose of evaluating native T1, T2, first pass perfusion, and extracellular volumes for patients with glomerular filtration rate >30. Patients with glomerular filtration rate <30 will only have native T1 and T2 values evaluated. If participants undergo implanted cardiac device during Tafamidis therapy, follow up cardiac magnetic resonance imaging will only evaluate featuring tracking and left ventricular mass. Left ventricular mass and cardiac magnetic feature tracking values will be extracted from all cardiac magnetic resonance imaging studies. After one year of Tafamidis therapy, participants will return to initial facility where testing was preformed to undergo a follow up cardiac magnetic resonance imaging study.

There is emerging evidence that there may be biomarkers yet identified for earlier detection of this disease. Henceforth, investigators propose to collect and store blood samples for all participants for future analyses.

Study Type

Observational

Enrollment (Anticipated)

131

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 90 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

131 patients diagnosed with ATTR-CM being treated with Tafamidis

Description

Inclusion Criteria:

  • Patients will be included if they meet the following criteria:

    • 18 and 90 years of age
    • Transthyretin amyloid cardiomyopathy (ATTRwt or ATTRm) confirmed by genetic testing and/or presence of transthyretin precursor protein confirmed on immunohistochemical analysis, and/or scintigraphy
    • History of heart failure (NYHA I, II, or III)
    • Agreeable to treatment with Tafamidis

Exclusion Criteria:

  • Patients will be excluded if any one of the following criteria are not met:

    • Heart failure not due to transthyretin amyloid cardiomyopathy
    • New York Heart Association (NYHA) class IV heart failure
    • Presence of light-chain amyloidosis (serum or urine)
    • Implanted cardiac device at baseline
    • Treatment with ATTR stabilizer or gene silencer within the past 6 months

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Other
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Stabilization of Transthyretin Amyloid Cardiomyopathy
Time Frame: 1 year
Stabilization as defined by reduction in extracellular volume on cardiac magnetic resonance after one year of Tafamidis therapy
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 1, 2020

Primary Completion (Anticipated)

August 1, 2022

Study Completion (Anticipated)

August 1, 2023

Study Registration Dates

First Submitted

August 12, 2020

First Submitted That Met QC Criteria

August 12, 2020

First Posted (Actual)

August 14, 2020

Study Record Updates

Last Update Posted (Actual)

January 27, 2021

Last Update Submitted That Met QC Criteria

January 25, 2021

Last Verified

January 1, 2021

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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