Transthyretin AmyloidosisNCT04513600
ATTR-Cardiomyopathy Stabilization Following Tafamidis Therapy
An observational study of Transthyretin Amyloidosis, run by The Christ Hospital at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 90 years
- Conditions the study is for
- Transthyretin Amyloidosis
- What is being tested?
- Cardiac MRI
- How many people
- 131 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Aug 2020to 1 Aug 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 90 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: 131 patients diagnosed with ATTR-CM being treated with Tafamidis
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
The Christ Hospital
Cincinnati, Ohio, 45219
Wojciech Mazur, MD, 513-206-1120, wojciech.mazur@thechristhospital.com
Cassady Palmer, BS, 513-585-1400, cassady.palmer@thechristhospital.com
About this study
Official title: 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:
- Utilize cardiac magnetic resonance to assess stabilization of ATTR after Tafamidis therapy based on extracellular volume mapping.
- Investigate left ventricular myocardial mass, native T1, T2, and extracellular volume mapping after 12 month follow-up.
- 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:
- Utilize cardiac magnetic resonance to assess stabilization of ATTR after Tafamidis therapy based on extracellular volume mapping.
- Investigate left ventricular myocardial mass, native T1, T2, and extracellular volume mapping after 12 month follow-up.
- 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.
- Conditions
- Transthyretin Amyloidosis
- Being tested
- Cardiac MRI
- Medical subject headings
- Amyloidosis, Hereditary, Transthyretin-Related
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cardiac MRIDiagnostic test
Also called Blood sample
Lab work: Hs- Troponin, Serum N-terminal-proBNP CMR: Native T1, Native T2, extracellular volume, strain and strain rate.
What is being measured
The primary outcome is the main question the study is designed to answer.
Stabilization of Transthyretin Amyloid Cardiomyopathy
Stabilization as defined by reduction in extracellular volume on cardiac magnetic resonance after one year of Tafamidis therapy
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Aug 2020
- Study start1 Aug 2020
- Primary completion1 Aug 2022 (estimated)
- Study completion1 Aug 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transthyretin AmyloidosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 131 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The Christ Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- The Cleveland Clinic, Ohio State University
- Responsible party
- Principal investigator, Dr. Wojciech Mazur, The Christ Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04513600
- Sponsor's protocol ID
- 20-02
- Status last verified
- Jan 2021
- First submitted
- 12 Aug 2020
- Last updated
- 27 Jan 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Transthyretin Amyloid Cardiomyopathy: Stabilization Assessed by Cardiac Magnetic Resonance
- ClinicalTrials.gov ID
- NCT04513600
- Acronym / sponsor ID
- 20-02
- Conditions
- Transthyretin Amyloidosis
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 131 participants (estimated)
- Lead sponsor
- The Christ HospitalUniversity, hospital or other
- Collaborators
- The Cleveland Clinic, Ohio State University
- Study start
- 1 Aug 2020 (actual)
- Primary completion
- 1 Aug 2022 (estimated)
- Study completion
- 1 Aug 2023 (estimated)
- First posted
- 14 Aug 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- Utilize cardiac magnetic resonance to assess stabilization of ATTR after Tafamidis therapy based on extracellular volume mapping.
- Investigate left ventricular myocardial mass, native T1, T2, and extracellular volume mapping after 12 month follow-up.
- Utilize cardiac magnetic resonance feature tracking at baseline and at 12 month follow-up.
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:
- Utilize cardiac magnetic resonance to assess stabilization of ATTR after Tafamidis therapy based on extracellular volume mapping.
- Investigate left ventricular myocardial mass, native T1, T2, and extracellular volume mapping after 12 month follow-up.
- 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
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- 131 patients diagnosed with ATTR-CM being treated with Tafamidis
Key inclusion criteria (5 listed)
- Patients will be included if they meet the following criteria:
Key exclusion criteria (6 listed)
- Patients will be excluded if any one of the following criteria are not met:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cardiac MRIAlso: Blood sampleLab work: Hs- Troponin, Serum N-terminal-proBNP CMR: Native T1, Native T2, extracellular volume, strain and strain rate. | Diagnostic test | – |
Primary outcome
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jan 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Transthyretin Amyloidosis, run by The Christ Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 27 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Wojciech Mazur, MD (513-206-1120, wojciech.mazur@thechristhospital.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Stabilization of Transthyretin Amyloid Cardiomyopathy (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jan 2021.