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ATTR-Cardiomyopathy Stabilization Following Tafamidis Therapy

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ClinicalTrials.gov Identifier: NCT04513600
Recruitment Status : Recruiting
First Posted : August 14, 2020
Last Update Posted : January 27, 2021
Sponsor:
Collaborators:
The Cleveland Clinic
Ohio State University
Information provided by (Responsible Party):
Dr. Wojciech Mazur, The Christ Hospital

Study Description
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Brief Summary:

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.

Condition or disease Intervention/treatment
Transthyretin Amyloidosis Diagnostic Test: Cardiac MRI

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 Design
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Study Type : Observational
Estimated Enrollment : 131 participants
Observational Model: Other
Time Perspective: Prospective
Official Title: Transthyretin Amyloid Cardiomyopathy: Stabilization Assessed by Cardiac Magnetic Resonance
Actual Study Start Date : August 1, 2020
Estimated Primary Completion Date : August 1, 2022
Estimated Study Completion Date : August 1, 2023

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Intervention Details:
  • Diagnostic Test: Cardiac MRI
    Lab work: Hs- Troponin, Serum N-terminal-proBNP CMR: Native T1, Native T2, extracellular volume, strain and strain rate.
    Other Name: Blood sample

Outcome Measures
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Primary Outcome Measures :
  1. 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


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 90 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
131 patients diagnosed with ATTR-CM being treated with Tafamidis
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04513600


Contacts
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Contact: Wojciech Mazur, MD 513-206-1120 wojciech.mazur@thechristhospital.com
Contact: Cassady Palmer, BS 513-585-1400 cassady.palmer@thechristhospital.com

Locations
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United States, Ohio
The Christ Hospital Recruiting
Cincinnati, Ohio, United States, 45219
Contact: Wojciech Mazur, MD    513-206-1120    wojciech.mazur@thechristhospital.com   
Contact: Cassady Palmer, BS    513-585-1400    cassady.palmer@thechristhospital.com   
Sponsors and Collaborators
The Christ Hospital
The Cleveland Clinic
Ohio State University
More Information
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Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
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Responsible Party: Dr. Wojciech Mazur, Director of Advanced Cardiac Imaging, The Christ Hospital
ClinicalTrials.gov Identifier: NCT04513600    
Other Study ID Numbers: 20-02
First Posted: August 14, 2020    Key Record Dates
Last Update Posted: January 27, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cardiomyopathies
Amyloidosis
Heart Diseases
 Cardiovascular Diseases
Proteostasis Deficiencies
Metabolic Diseases