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Status unknownNCT04513600Updated Jan 27, 2021

ATTR-Cardiomyopathy Stabilization Following Tafamidis Therapy

An observational study in Transthyretin Amyloidosis, sponsored by The Christ Hospital. Status unknown at 1 site in United States. Open to participants aged 18 Years to 90 Years. Per ClinicalTrials.gov, last updated 2021-01-27.

Sponsored by The Christ Hospital · Observational

The sponsor has not verified this record recently (last verified Jan 2021), so the status shown — last known as Recruiting — may be out of date.
Study type
Observational
Model
Other
Time perspective
Prospective
Enrollment
131
Ages
18 Years to 90 Years
Sex
All
01

Study 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.
Read the 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.

02

Conditions studied

  • Transthyretin Amyloidosis
03

In context

Cardiomyopathies

1,176 studies on the registry are indexed under Cardiomyopathies; 287 are open to participants now.

This study's planned enrollment of 131 is below the median of 153 across 515 observational studies indexed under Cardiomyopathies.

Browse Cardiomyopathies studies →

Lead sponsor

The Christ Hospital is the lead sponsor of 18 studies on the registry; 3 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 90 Years
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

131 patients diagnosed with ATTR-CM being treated with Tafamidis

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

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
05

Study design

Observational model
Other
Time perspective
Prospective
Enrollment
131 participants (estimated)
Patient registry
No

Interventions

  • Diagnostic testCardiac MRI

    Lab work: Hs- Troponin, Serum N-terminal-proBNP CMR: Native T1, Native T2, extracellular volume, strain and strain rate.

    Also known as: Blood sample

06

What researchers measure

Primary outcomes

  1. Stabilization of Transthyretin Amyloid Cardiomyopathy

    Stabilization as defined by reduction in extracellular volume on cardiac magnetic resonance after one year of Tafamidis therapy

    Time frame: 1 year

07

Study locations

1 of 1 sites recruiting
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jan 27, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT04513600
Lead sponsor
The Christ Hospital
Collaborators
The Cleveland Clinic, Ohio State University
Responsible party
Dr. Wojciech Mazur (Director of Advanced Cardiac Imaging, The Christ Hospital) — Principal investigator
First posted
Aug 14, 2020
Start date
Aug 1, 2020
Primary completion
Aug 1, 2022 (estimated)
Completion
Aug 1, 2023 (estimated)
Last update
Jan 27, 2021

Study contacts

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

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is status unknown, as verified in Jan 2021. You cannot join it, but the record below documents what was studied.

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