ATTR-Cardiomyopathy Stabilization Following Tafamidis Therapy

NCT04513600 | Unknown

• 1 other identifier: 20-02
• Study Type: observational
• Target: 131
• Locations: 1 country
• Sites: 1

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

Conditions

Transthyretin Amyloidosis

Outcome Measures

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

  1 year

Interventions

Cardiac MRI DIAGNOSTIC_TEST

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

Also known as: Blood sample

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

131 patients diagnosed with ATTR-CM being treated with Tafamidis

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• The Christ Hospital: lead
• The Cleveland Clinic: collaborator
• Ohio State University: collaborator

Study Sites (1)

The Christ Hospital

Cincinnati, Ohio, 45219, United States

RECRUITING

MeSH Terms

Conditions

Amyloidosis, Hereditary, Transthyretin-Related

Interventions

Blood Specimen Collection

Intervention Hierarchy (Ancestors)

Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques

Central Study Contacts

Wojciech Mazur, MD

CONTACT

513-206-1120; wojciech.mazur@thechristhospital.com

Cassady Palmer, BS

CONTACT

513-585-1400; cassady.palmer@thechristhospital.com

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of Advanced Cardiac Imaging

Study Record Dates

• First Submitted: August 12, 2020
• First Posted: August 14, 2020
• Study Start: August 1, 2020
• Primary Completion: August 1, 2022
• Study Completion: August 1, 2023
• Last Updated: January 27, 2021

Record last verified: 2021-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)