Cardiac AmyloidosisPhase 4NCT01683825
Imaging Cardiac Amyloidosis: A Pilot Study Using F-18 Florbetapir Positron Emission Tomography
A Phase 4 study testing F-18 florbetapir PET in people with Cardiac Amyloidosis, run by Brigham and Women's Hospital at 1 site in United States.
Trial summary
The primary aim of this pilot study is to determine whether amyloid deposits in the heart can be measured non-invasively by F-18 florbetapir (Trade Name: Amyvid) positron emission tomography (PET) in 30 individuals with documented cardiac amyloidosis. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cardiac Amyloidosis
- What is being tested?
- F-18 florbetapir PET
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 1 siteUnited States
- Study dates
- 23 Mar 2013to 10 Oct 2018
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Age > 18 years
- Biopsy proven amyloidosis outside the heart with typical echocardiographic appearance of cardiac involvement, or a positive cardiac biopsy.
- Diagnosis of AL amyloidosis by standard criteria (evidence of plasma cell dyscrasia with appropriate tissue staining for AL) OR
- Diagnosis of TTR amyloidosis (no evidence of plasma call dyscrasia and positive TTR staining of amyloid in tissue biopsy)
- Able and willing to provide informed consent to participate in the study procedures
Who cannot join
- Pregnancy
- Serious non-cardiac medical illness which will preclude research study participation
Trial locations and contacts
1 site in 1 country.
United States1 site
Brigham and Women's Hospital
Boston, Massachusetts, 02115
About this study
The primary aim of this pilot study is to determine whether amyloid deposits in the heart can be measured non-invasively by F-18 florbetapir (Trade Name: Amyvid) positron emission tomography (PET) in 30 individuals with documented cardiac amyloidosis. We will also enroll 15 individuals without cardiac amyloidosis to undergo the F-18 florbetapir imaging as a control group.
The primary hypothesis of this study is that a specific amyloid binding radiotracer will bind to the myocardial amyloid deposits and help quantify cardiac amyloid burden.
A secondary aim of this study is to determine reproducibility of F-18 florbetapir imaging of the myocardium.
Read the detailed description
F-18 florbetapir has been studied in multiple clinical trials to image beta-amyloid deposition in the brain of subjects with Alzheimers' disease. Florbetapir F-18 has been well tolerated in studies of more than 2000 human subjects. Biodistribution studies in humans revealed predominantly hepatobiliary excretion. The tracer clears rapidly from the blood pool in about 20 minutes. This radiotracer has been recently approved for clinical imaging of brain amyloid in subjects with suspected Alzheimers disease. The investigators propose to test this FDA approved radiotracer for an off label indication in a pilot study to evaluate its potential utility, if any, to image cardiac amyloidosis.
Amyloid related heart disease is associated with LV wall thickening due to infiltration; however, this myocardial wall thickening is not definitively distinguishable from left ventricular myocyte hypertrophy from increased afterload to the heart from hypertension or aortic stenosis. Typically myocardial or other tissue biopsy with typical echo features of amyloidosis is required for confirmation of amyloidosis. This pilot study is designed to understand whether cardiac amyloid burden can be measured using a specific radiotracer targeted against amyloid protein (F-18 Florbetapir). At this point it is unknown of F-18 Florbetapir will bind to either AL or TTR amyloid protein or to both of them or to neither of them. The investigators would like to study 15 patients with AL and 15 patients with TTR amyloidosis to understand these differences if any. The investigators also seek to understand if the signal to noise ratio of the circulating amyloid protein in the blood pool (AL amyloid disease) allows for good differentiation of myocardial amyloid uptake. 15 individuals without cardiac amyloidosis will also be enrolled as controls for this study.
The purpose of the proposed research study is to examine in detail, using quantitative PET, myocardial F-18 Florbetapir uptake in cardiac amyloidosis in order to better understand mechanisms of heart damage in this disease. A secondary aim of this study is to determine reproducibility of F-18 florbetapir imaging of the myocardium in a subset of patients with amyloidosis who return for a second F-18 florbetapir scan within 30 days.
- Conditions
- Cardiac Amyloidosis
- Being tested
- F-18 florbetapir PET
- Keywords
- Amyloidosis, Cardiac, Heart, Imaging, Diagnosis
- Medical subject headings
- Amyloid Neuropathies, Familial, Amyloidosis, Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
F-18 florbetapir PETDrug
Also called F-18 labeled Florbetapir (Trade Name: Amyvid)
Cardiac PET images will be obtained following injection of F-18 labeled Florbetapir (Trade Name: Amyvid)
| Study group | Role | What they receive |
|---|---|---|
| F-18 florbetapir PET-Amyloid Subjects | Experimental | Individuals with documented cardiac amyloidosis will undergo F-18 florbetapir (Trade Name: Amyvid) positron emission tomography (PET). Receives: Drug: F-18 florbetapir PET |
| F-18 florbetapir PET-Healthy Controls | Other | Individuals without documented cardiac amyloidosis will undergo F-18 florbetapir (Trade Name: Amyvid) positron emission tomography (PET). Receives: Drug: F-18 florbetapir PET |
| F-18 florbetapir PET-Amyloid Reproducibility Subjects | Experimental | Some of the individuals with documented cardiac amyloidosis from arm 1 will undergo a second F-18 florbetapir (Trade Name: Amyvid) positron emission tomography (PET) within 30 days to measure reproducibility Receives: Drug: F-18 florbetapir PET |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Left Ventricular Myocardial F-18 Florbetapir SUVmean
Standardized uptake value (SUV) mean is defined as the mean F-18 florbetapir activity concentration [kBq/ml] measured within the left ventricular myocardial region of interest multiplied by the decay-corrected amount of injected F-18 florbetapir [kBq] normalized to patient weight [g]. In this study we will measure mean left ventricular global myocardial F-18 Florbetapir SUVmean on static images between 4 minutes to 30 minutes post injection of F-18 florbetapir.
Measured: 1 day
Secondary outcomes (1)
Percent Change in Global Left Ventricular F-18 Florbetapir SUVmean From Scan 1 to Scan 2
We evaluated change in F-18 florbetapir SUVmean from scan 1 to scan 2 as a percentage value [(SUVmean1 minus SUVmean 2)/(SUVmean 1)*100]. In the 7 participants with cardiac amyloidosis who underwent two studies (median of 7 days apart), the percentage change in F-18 florbetapir SUVmean from scan 1 to scan 2 ranged from -38.5% to +20.8% with a median value of +2.24%.
Measured: Repeat scan was performed a median of 7 days after baseline study (range 1-46 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Sep 2012 (estimated)
- Study start23 Mar 2013
- Primary completion4 Oct 2018
- Study completion10 Oct 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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 Brigham and Women's HospitalThe 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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Brigham and Women's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Sharmila Dorbala, Brigham and Women's Hospital
- Investigators
- Sharmila Dorbala, MBBS, MPH, Principal investigator, Brigham and Women's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01683825
- Sponsor's protocol ID
- 2012P001322
- Status last verified
- Nov 2025
- First submitted
- 4 Sep 2012
- Last updated
- 20 Nov 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Dorbala S, Vangala D, Semer J, Strader C, Bruyere JR Jr, Di Carli MF, Moore SC, Falk RH. Imaging cardiac amyloidosis: a pilot study using (1)(8)F-florbetapir positron emission tomography. Eur J Nucl Med Mol Imaging. 2014 Sep;41(9):1652-62. doi: 10.1007/s00259-014-2787-6. Epub 2014 May 20.
PubMed 24841414
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Imaging Cardiac Amyloidosis: A Pilot Study Using F-18 Florbetapir Positron Emission Tomography
- ClinicalTrials.gov ID
- NCT01683825
- Acronym / sponsor ID
- 2012P001322
- Conditions
- Cardiac Amyloidosis
- Keywords
- Amyloidosis, Cardiac, Heart, Imaging, Diagnosis
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Brigham and Women's HospitalUniversity, hospital or other
- Study start
- 23 Mar 2013 (actual)
- Primary completion
- 4 Oct 2018 (actual)
- Study completion
- 10 Oct 2018 (actual)
- First posted
- 12 Sep 2012 (estimated)
- Results
- Posted 13 Apr 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary aim of this pilot study is to determine whether amyloid deposits in the heart can be measured non-invasively by F-18 florbetapir (Trade Name: Amyvid) positron emission tomography (PET) in 30 individuals with documented cardiac amyloidosis. We will also enroll 15 individuals without cardiac amyloidosis to undergo the F-18 florbetapir imaging as a control group.
The primary hypothesis of this study is that a specific amyloid binding radiotracer will bind to the myocardial amyloid deposits and help quantify cardiac amyloid burden.
A secondary aim of this study is to determine reproducibility of F-18 florbetapir imaging of the myocardium.
Detailed description
F-18 florbetapir has been studied in multiple clinical trials to image beta-amyloid deposition in the brain of subjects with Alzheimers' disease. Florbetapir F-18 has been well tolerated in studies of more than 2000 human subjects. Biodistribution studies in humans revealed predominantly hepatobiliary excretion. The tracer clears rapidly from the blood pool in about 20 minutes. This radiotracer has been recently approved for clinical imaging of brain amyloid in subjects with suspected Alzheimers disease. The investigators propose to test this FDA approved radiotracer for an off label indication in a pilot study to evaluate its potential utility, if any, to image cardiac amyloidosis.
Amyloid related heart disease is associated with LV wall thickening due to infiltration; however, this myocardial wall thickening is not definitively distinguishable from left ventricular myocyte hypertrophy from increased afterload to the heart from hypertension or aortic stenosis. Typically myocardial or other tissue biopsy with typical echo features of amyloidosis is required for confirmation of amyloidosis. This pilot study is designed to understand whether cardiac amyloid burden can be measured using a specific radiotracer targeted against amyloid protein (F-18 Florbetapir). At this point it is unknown of F-18 Florbetapir will bind to either AL or TTR amyloid protein or to both of them or to neither of them. The investigators would like to study 15 patients with AL and 15 patients with TTR amyloidosis to understand these differences if any. The investigators also seek to understand if the signal to noise ratio of the circulating amyloid protein in the blood pool (AL amyloid disease) allows for good differentiation of myocardial amyloid uptake. 15 individuals without cardiac amyloidosis will also be enrolled as controls for this study.
The purpose of the proposed research study is to examine in detail, using quantitative PET, myocardial F-18 Florbetapir uptake in cardiac amyloidosis in order to better understand mechanisms of heart damage in this disease. A secondary aim of this study is to determine reproducibility of F-18 florbetapir imaging of the myocardium in a subset of patients with amyloidosis who return for a second F-18 florbetapir scan within 30 days.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| F-18 florbetapir PET-Amyloid Subjects | Experimental | Drug: F-18 florbetapir PETIndividuals with documented cardiac amyloidosis will undergo F-18 florbetapir (Trade Name: Amyvid) positron emission tomography (PET). |
| F-18 florbetapir PET-Healthy Controls | Other | Drug: F-18 florbetapir PETIndividuals without documented cardiac amyloidosis will undergo F-18 florbetapir (Trade Name: Amyvid) positron emission tomography (PET). |
| F-18 florbetapir PET-Amyloid Reproducibility Subjects | Experimental | Drug: F-18 florbetapir PETSome of the individuals with documented cardiac amyloidosis from arm 1 will undergo a second F-18 florbetapir (Trade Name: Amyvid) positron emission tomography (PET) within 30 days to measure reproducibility |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age > 18 years
- Biopsy proven amyloidosis outside the heart with typical echocardiographic appearance of cardiac involvement, or a positive cardiac biopsy.
- Diagnosis of AL amyloidosis by standard criteria (evidence of plasma cell dyscrasia with appropriate tissue staining for AL) OR
- Diagnosis of TTR amyloidosis (no evidence of plasma call dyscrasia and positive TTR staining of amyloid in tissue biopsy)
- Able and willing to provide informed consent to participate in the study procedures
Key exclusion criteria (2 listed)
- Pregnancy
- Serious non-cardiac medical illness which will preclude research study participation
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 |
|---|---|---|
| F-18 florbetapir PETAlso: F-18 labeled Florbetapir (Trade Name: Amyvid)Cardiac PET images will be obtained following injection of F-18 labeled Florbetapir (Trade Name: Amyvid) | Drug | F-18 florbetapir PET-Amyloid Reproducibility Subjects, F-18 florbetapir PET-Amyloid Subjects, F-18 florbetapir PET-Healthy Controls |
Primary outcome
Mean Left Ventricular Myocardial F-18 Florbetapir SUVmean
Time frame: 1 day
Standardized uptake value (SUV) mean is defined as the mean F-18 florbetapir activity concentration [kBq/ml] measured within the left ventricular myocardial region of interest multiplied by the decay-corrected amount of injected F-18 florbetapir [kBq] normalized to patient weight [g]. In this study we will measure mean left ventricular global myocardial F-18 Florbetapir SUVmean on static images between 4 minutes to 30 minutes post injection of F-18 florbetapir.
1 secondary outcome
Percent Change in Global Left Ventricular F-18 Florbetapir SUVmean From Scan 1 to Scan 2
Time frame: Repeat scan was performed a median of 7 days after baseline study (range 1-46 days)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Nov 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 24841414Dorbala S, Vangala D, Semer J, Strader C, Bruyere JR Jr, Di Carli MF, Moore SC, Falk RH. Imaging cardiac amyloidosis: a pilot study using (1)(8)F-florbetapir positron emission tomography. Eur J Nucl Med Mol Imaging. 2014 Sep;41(9):1652-62. doi: 10.1007/s00259-014-2787-6. Epub 2014 May 20.
- 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.
Sharmila Dorbala, MBBS, MPH principal investigator
Brigham and Women's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing F-18 florbetapir PET in people with Cardiac Amyloidosis, run by Brigham and Women's 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 2 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: finished, as of the sponsor's last update on 20 Nov 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 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 13 Apr 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean Left Ventricular Myocardial F-18 Florbetapir SUVmean (time frame: 1 day). 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 NCT01683825, 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 20 Nov 2025.