Cardiac AmyloidosisNCT02798705
Physiologic Assessment of Microvascular Function in Patients With Cardiac Amyloidosis
An observational study of Cardiac Amyloidosis, run by Samsung Medical Center at 1 site in South Korea.
Trial summary
The aim of the study is to evaluate coronary flow reserve (CFR), index of microcirculatory resistance (IMR), and proportion of overt microvascular disease, defined as depressed CFR as well as elevated IMR in patients with cardiac amyloidosis. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Cardiac Amyloidosis
- How many people
- 30 participants
- Where
- 1 siteSouth Korea
- Study dates
- 11 Apr 2016to Dec 2030
- How long will I take part?
- 2 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Pathologically confirmed amyloidosis patients with suspected cardiac involvement undergoing coronary angiography for coronary disease evaluation
Who may be able to join
- Subject age 18-85 years old
- Patients with confirmed cardiac amyloidosis by heart biopsy
- Patients with confirmed amyloidosis by biopsy other than heart and evidence of cardiac involvement in echocardiography
- Patients who underwent invasive physiologic assessment within 3 months from diagnosis of primary disease
Who cannot join
- Patients with cardiogenic shock
- Patients with unstable vital sign that precludes coronary angiography
- Patients with major bleeding in last 3 months
- Patients with active bleeding
- Patients with coagulopathy
- Patients with severe valvular heart disease
- Patients who refused to provide informed consent
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
South Korea1 site, 1 recruiting
- Recruiting
Samsung Medical Center
Seoul
Joo Myung Lee, MD, MPH, 82-2-3410-1246, drone80@hanmail.net
About this study
Official title: Physiologic Assessment of Microvascular Function in Patients With Cardiac Amyloidosis: Prospective Registry and Pilot Study
The aim of the study is to evaluate coronary flow reserve (CFR), index of microcirculatory resistance (IMR), and proportion of overt microvascular disease, defined as depressed CFR as well as elevated IMR in patients with cardiac amyloidosis. The second objective of this study is to compare results of non-invasive test including serum light chain amount, Doppler echocardiography with 2D strain, and cardiac perfusion MRI. The third object of this study is to evaluate the association between physiologic indices and pathologically measured percent area involvement of interstitium.
Read the detailed description
Amyloidosis is rare systemic disorder characterized by the extracellular deposition of misfolded protein in various organ system, including heart. Among the several types of amyloid fibrils, the light chain and transthyretin amyloid proteins most commonly affect the heart.
Cardiac amyloid deposits result in increased ventricular wall thickness and produce a restrictive cardiomyopathy presenting primarily as biventricular congestive heart failure. Anginal symptoms and signs of ischemia have been reported in some patients with cardiac amyloidosis without obstructive epicardial coronary artery disease (CAD). Autopsy studies have shown amyloid deposits around and between cardiac myocytes in the interstitium, the perivascular regions, and the media of intramyocardial coronary vessels. Amyloidosis is thus a prime example of a disorder with the potential to cause coronary microvascular dysfunction via 3 major mechanisms: (1) structural (amyloid deposition in the vessel wall causing wall thickening and luminal stenosis), (2) extravascular (extrinsic compression of the microvasculature from perivascular and interstitial amyloid deposits and decreased diastolic perfusion), and (3) functional (autonomic and endothelial dysfunction).
Previous basic research presented that adipose arteriole or atrial coronary arterioles showed endothelial dysfunction even after brief exposure to physiologic amounts of light chain, and also showed increased oxidative stress, reduced NO bioavailability, and peroxynitrite production. All these previous evidences imply that coronary microvascular dysfunction and subsequent global ischemic insult can be precursor of overt diastolic or systolic dysfunction in patients with cardiac amyloidosis.
However, there have only 1 study which evaluated microvascular function in vivo using N13-ammonium positron emission tomography (PET). In that study, Dorbala et al. demonstrated that amyloidosis patients showed depressed global resting myocardial blood flow (MBF), stress MBF, and CFR and higher minimal coronary vascular resistance, compared with patients with left ventricular hypertrophy. However, low availability, high cost, and limited resolution of N13 ammonium PET precludes the generalizability of the results.
Since thermodilution-derived coronary flow reserve and index or microcirculatory reserve using pressure-temperature wire has been well validated prognostic index in assessment of patients with coronary artery disease or functionally insignificant epicardial coronary stenosis, invasive physiologic assessment might more specifically assess macro- and microvascular function in patients with cardiac amyloidosis. Moreover, adding physiologic measurement in the current frame in diagnosis of cardiac amyloidosis might enhance risk stratification of patients.
Therefore, the current study will perform physiologic assessment including fractional flow reserve, coronary flow reserve, and index of microcirculatory resistance in patients with cardiac amyloidosis, and explore correlation among the physiologic indices and conventional measurements of echocardiography, perfusion MRI, serum light chain amount, or NT-proBNP. In addition, the differences of physiologic indices according to disease severity of cardiac amyloidosis, which measured by endomyocardial biopsy findings will be also explored. Since there was no previous study which performed invasive physiologic assessment in amyloidosis patients, this study will be performed as pilot study. Target sample size will be at least 30 patients.
- Conditions
- Cardiac Amyloidosis
- Keywords
- Cardiac amyloidosis, Fractional flow reserve, Microvascular dysfunction, Coronary flow reserve, Index of microcirculatory resistance
- Medical subject headings
- Amyloid Neuropathies, Familial
What is being measured
The primary outcome is the main question the study is designed to answer.
all-cause mortality
Comparison of all-cause mortality in amyloidosis patients with or without microvascular disease
Measured: 2 Years
Secondary outcomes (8)
Correlation between IMR and echocardiographic parameters
Correlation between IMR and echocardiographic parameters (E/E', LV ejection fraction, etc.)
Measured: within 3 months of diagnosis
Correlation between IMR and serum biochemicalmarkers
Correlation between IMR and serum biochemicalmarkers (troponin T, NT-proBNP, serum free light chain)
Measured: within 3 months of diagnosis
Distribution of CFR or IMR according to pathologic severity of myocardial involvement
Distribution of CFR or IMR according to pathologic severity of myocardial involvement (Percent amyloid load)
Measured: within 3 months of diagnosis
Comparison of CFR or IMR value in amyloidosis patient with or without relative perfusion defect in myocardial perfusion imaging (ex> adenosine-SPECT or perfusion MRI)
Measured: within 3 months of diagnosis
Comparison of CFR or IMR value in different type of amyloidosis (AL type, hereditary type, AA type, senile type)
Measured: within 3 months of diagnosis
Comparison of CFR or IMR value in amyloidosis patient according to current staging by Troponin T, NT-proBNP and free light chain difference
Measured: within 3 months of diagnosis
Optimal cut-off values of physiologic indices (CFR, IMR) for prediction of all-cause mortality
Measured: 2 Years
Association between physiologic indices and pathologically measured percent area involvement in myocardial interstitium
Measured: within 3 months of diagnosis
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2016 (estimated)
- Study start11 Apr 2016
- Primary completionApr 2029 (estimated)
- Study completionDec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cardiac AmyloidosisStudies of the same condition, open and under way.
- More trials run by Samsung Medical CenterThe sponsor's other registered studies.
- 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 30 participants (estimated)
- Target duration
- 2 Years
Sponsor and investigators
- Lead sponsor
- Samsung Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Joo Myung Lee, Samsung Medical Center
- Investigators
- Joo Myung Lee, MD, MPH, PhD, Principal investigator, Samsung Medical CenterEun-Seok Jeon, MD, PhD, Principal investigator, Samsung Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02798705
- Sponsor's protocol ID
- AMYL2016-04-122
- Status last verified
- Mar 2025
- First submitted
- 9 Jun 2016
- Last updated
- 14 Mar 2025
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: After publication of first manuscript and trial results, the de-identified data will be shared by permission of principle investigator, when asked
Publications
Choi KH, Lee JM, Kim SR, Kim D, Choi JO, Kim SJ, Kim K, Kim JS, Koo BK, Jeon ES. Prognostic Value of the Index of Microcirculatory Resistance Over Serum Biomarkers in Cardiac Amyloidosis. J Am Coll Cardiol. 2020 Feb 11;75(5):560-561. doi: 10.1016/j.jacc.2019.11.045. No abstract available.
PubMed 32029139
Study at a glance
From the registry record, last updated by the sponsor on 14 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Physiologic Assessment of Microvascular Function in Patients With Cardiac Amyloidosis: Prospective Registry and Pilot Study
- ClinicalTrials.gov ID
- NCT02798705
- Acronym / sponsor ID
- AMYL2016-04-122
- Conditions
- Cardiac Amyloidosis
- Keywords
- Cardiac amyloidosis, Fractional flow reserve, Microvascular dysfunction, Coronary flow reserve, Index of microcirculatory resistance
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Samsung Medical CenterUniversity, hospital or other
- Study start
- 11 Apr 2016 (actual)
- Primary completion
- Apr 2029 (estimated)
- Study completion
- Dec 2030 (estimated)
- First posted
- 14 Jun 2016 (estimated)
- 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 aim of the study is to evaluate coronary flow reserve (CFR), index of microcirculatory resistance (IMR), and proportion of overt microvascular disease, defined as depressed CFR as well as elevated IMR in patients with cardiac amyloidosis. The second objective of this study is to compare results of non-invasive test including serum light chain amount, Doppler echocardiography with 2D strain, and cardiac perfusion MRI. The third object of this study is to evaluate the association between physiologic indices and pathologically measured percent area involvement of interstitium.
Detailed description
Amyloidosis is rare systemic disorder characterized by the extracellular deposition of misfolded protein in various organ system, including heart. Among the several types of amyloid fibrils, the light chain and transthyretin amyloid proteins most commonly affect the heart.
Cardiac amyloid deposits result in increased ventricular wall thickness and produce a restrictive cardiomyopathy presenting primarily as biventricular congestive heart failure. Anginal symptoms and signs of ischemia have been reported in some patients with cardiac amyloidosis without obstructive epicardial coronary artery disease (CAD). Autopsy studies have shown amyloid deposits around and between cardiac myocytes in the interstitium, the perivascular regions, and the media of intramyocardial coronary vessels. Amyloidosis is thus a prime example of a disorder with the potential to cause coronary microvascular dysfunction via 3 major mechanisms: (1) structural (amyloid deposition in the vessel wall causing wall thickening and luminal stenosis), (2) extravascular (extrinsic compression of the microvasculature from perivascular and interstitial amyloid deposits and decreased diastolic perfusion), and (3) functional (autonomic and endothelial dysfunction).
Previous basic research presented that adipose arteriole or atrial coronary arterioles showed endothelial dysfunction even after brief exposure to physiologic amounts of light chain, and also showed increased oxidative stress, reduced NO bioavailability, and peroxynitrite production. All these previous evidences imply that coronary microvascular dysfunction and subsequent global ischemic insult can be precursor of overt diastolic or systolic dysfunction in patients with cardiac amyloidosis.
However, there have only 1 study which evaluated microvascular function in vivo using N13-ammonium positron emission tomography (PET). In that study, Dorbala et al. demonstrated that amyloidosis patients showed depressed global resting myocardial blood flow (MBF), stress MBF, and CFR and higher minimal coronary vascular resistance, compared with patients with left ventricular hypertrophy. However, low availability, high cost, and limited resolution of N13 ammonium PET precludes the generalizability of the results.
Since thermodilution-derived coronary flow reserve and index or microcirculatory reserve using pressure-temperature wire has been well validated prognostic index in assessment of patients with coronary artery disease or functionally insignificant epicardial coronary stenosis, invasive physiologic assessment might more specifically assess macro- and microvascular function in patients with cardiac amyloidosis. Moreover, adding physiologic measurement in the current frame in diagnosis of cardiac amyloidosis might enhance risk stratification of patients.
Therefore, the current study will perform physiologic assessment including fractional flow reserve, coronary flow reserve, and index of microcirculatory resistance in patients with cardiac amyloidosis, and explore correlation among the physiologic indices and conventional measurements of echocardiography, perfusion MRI, serum light chain amount, or NT-proBNP. In addition, the differences of physiologic indices according to disease severity of cardiac amyloidosis, which measured by endomyocardial biopsy findings will be also explored. Since there was no previous study which performed invasive physiologic assessment in amyloidosis patients, this study will be performed as pilot study. Target sample size will be at least 30 patients.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 2 Years
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Pathologically confirmed amyloidosis patients with suspected cardiac involvement undergoing coronary angiography for coronary disease evaluation
Key inclusion criteria (4 listed)
- Subject age 18-85 years old
- Patients with confirmed cardiac amyloidosis by heart biopsy
- Patients with confirmed amyloidosis by biopsy other than heart and evidence of cardiac involvement in echocardiography
- Patients who underwent invasive physiologic assessment within 3 months from diagnosis of primary disease
Key exclusion criteria (7 listed)
- Patients with cardiogenic shock
- Patients with unstable vital sign that precludes coronary angiography
- Patients with major bleeding in last 3 months
- Patients with active bleeding
- Patients with coagulopathy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
all-cause mortality
Time frame: 2 Years
Comparison of all-cause mortality in amyloidosis patients with or without microvascular disease
8 secondary outcomes
Correlation between IMR and echocardiographic parameters
Time frame: within 3 months of diagnosis
Correlation between IMR and serum biochemicalmarkers
Time frame: within 3 months of diagnosis
Distribution of CFR or IMR according to pathologic severity of myocardial involvement
Time frame: within 3 months of diagnosis
Comparison of CFR or IMR value in amyloidosis patient with or without relative perfusion defect in myocardial perfusion imaging (ex> adenosine-SPECT or perfusion MRI)
Time frame: within 3 months of diagnosis
Comparison of CFR or IMR value in different type of amyloidosis (AL type, hereditary type, AA type, senile type)
Time frame: within 3 months of diagnosis
Comparison of CFR or IMR value in amyloidosis patient according to current staging by Troponin T, NT-proBNP and free light chain difference
Time frame: within 3 months of diagnosis
Optimal cut-off values of physiologic indices (CFR, IMR) for prediction of all-cause mortality
Time frame: 2 Years
Association between physiologic indices and pathologically measured percent area involvement in myocardial interstitium
Time frame: within 3 months of diagnosis
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Mar 2025.
- Derived from this study: PubMed 32029139Choi KH, Lee JM, Kim SR, Kim D, Choi JO, Kim SJ, Kim K, Kim JS, Koo BK, Jeon ES. Prognostic Value of the Index of Microcirculatory Resistance Over Serum Biomarkers in Cardiac Amyloidosis. J Am Coll Cardiol. 2020 Feb 11;75(5):560-561. doi: 10.1016/j.jacc.2019.11.045. No abstract available.
- Individual participant dataSharing planned: Yes. After publication of first manuscript and trial results, the de-identified data will be shared by permission of principle investigator, when asked
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MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
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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.
Joo Myung Lee, MD, MPH, PhD principal investigator
Samsung Medical Center
Eun-Seok Jeon, MD, PhD principal investigator
Samsung Medical Center
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 Cardiac Amyloidosis, run by Samsung Medical Center at 1 site in South Korea.
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 4 inclusion and 7 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: looking for participants now, as of the sponsor's last update on 14 Mar 2025. 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 South Korea. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Joo Myung Lee, MD, MPH, PhD (82-2-3410-1246, drone80@hanmail.net), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
all-cause mortality (time frame: 2 Years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02798705, which also lists 1 linked publication.
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 14 Mar 2025.