Transthyretin Amyloid CardiomyopathyNCT04424914
Global Prevalence of ATTR-CM in Participants With HFpEF
A study testing Scintigraphy in people with Transthyretin Amyloid Cardiomyopathy and Heart Failure With Preserved Ejection Fraction, run by Pfizer at 51 sites in 8 countries.
Trial summary
This study is a global, multi-center study designed to estimate the global prevalence of transthyretin amyloid cardiomyopathy (ATTR-CM) within a clinically at risk population [participants with heart failure with preserved ejection fraction (HFpEF)]. Read more about this study
- Who is it for?
- Ages 60 years and older
- Conditions the study is for
- Transthyretin Amyloid Cardiomyopathy, Heart Failure With Preserved Ejection Fraction
- What is being tested?
- Scintigraphy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 347 participants
- Where
- 51 sitesin 8 countries
- Study dates
- 30 Dec 2020to 2 Jun 2023
- Phase
- Interventional
- 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 60 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Medical history of heart failure (HF) with:
- At least 1 episode with clinical evidence of HF (without hospitalization) by signs or symptoms of volume overload or elevated intracardiac pressures that required/requires treatment with a diuretic for improvement; OR
- 1 prior hospitalization for HF.
- Left ventricular ejection fraction (LVEF) >40%.
- End-diastolic interventricular septal wall thickness (IVST) ≥12 mm.
- Willing and able to undergo scintigraphy.
Who cannot join
- Diagnosis of heart failure with reduced ejection fraction (HFrEF) (EF ≤40%).
- Prior clinical history of myocardial infarction, CABG or multi-vessel obstructive coronary disease (>50% stenosis of ≥2 epicardial coronary arteries).
- Presence or history of any severe valvular heart disease (obstructive or regurgitant).
- A confirmed diagnosis of a non-amyloid infiltrative cardiomyopathy (ie, cardiac sarcoidosis, hemochromatosis), muscular dystrophies, cardiomyopathy with reversible causes, hypertrophic obstructive cardiomyopathy with known genetic etiology, or known pericardial constriction.
- Any type of diagnosed amyloidosis (eg, amyloid A amyloidosis, primary [light chain] amyloidosis) or prior diagnosis of ATTR-CM.
- -
Trial locations and contacts
51 sites in 8 countries.
51 sites in 8 countries
United States22 sites
Eastern shore Research Institute LLC
Fairhope, Alabama, 36532
Corewell Health
Grand Rapids, Michigan, 49503
Spectrum Health Medical Group Cardiovascular Medicine
Grand Rapids, Michigan, 49525
Chicago Medical Research, LLC
Hazel Crest, Illinois, 60429
Heart Center Research, LLC
Huntsville, Alabama, 35801
The Jackson Clinic
Jackson, Tennessee, 38301
Advance Medical Research Center
Miami, Florida, 33135
Bioclinical Research Alliance Inc.
Miami, Florida, 33155
Biogenix Molecular
Miami, Florida, 33165
CIRA (Nuclear Imaging Facility)
Miami, Florida, 33165
Nucleotron/ Doral Imaging Institute, CIRA DBA
Miami, Florida, 33165
Innova Pharma Research
Miami, Florida, 33175
Ochsner Medical Center
New Orleans, Louisiana, 70121
NYU Langone Health
New York, New York, 10016
Advocate Christ Medical Center
Oak Lawn, Illinois, 60453
Ocala Cardiovascular Research
Ocala, Florida, 34471
WakeMed Health and Hospital
Raleigh, North Carolina, 27610
Providence Sacred Heart Medical Center and Children's Hospital
Spokane, Washington, 99204
Stormont Vail Health
Topeka, Kansas, 66604
Cotton O'Neil Heart Center
Topeka, Kansas, 66606
Cardiology Associates of North Mississippi, LLC
Tupelo, Mississippi, 38801
Cardiology Associates Research, LLC
Tupelo, Mississippi, 38801
Spain7 sites
Hospital Universitari Vall d´Hebron
Barcelona, 08035
Hospital Clinic de Barcelona
Barcelona, 08036
Hospital Universitari De Bellvitge
Barcelona, 08907
Hospital Universitario Ramón y Cajal
Madrid, 28034
Hospital Universitario Puerta de Hierro Majadahonda
Majadahonda, Madrid, 28222
Hospital Universitario de Salamanca - Complejo Asistencial Universitario de Salamanca
Salamanca, 37007
Hospital Universitari i Politècnic La Fe
Valencia, 46026
France5 sites
Hôpital Louis Pradel
Bron, 69677
Centre Hospitalier Saint-Joseph Saint-Luc
Lyon, 69365
CHU Nimes - Hospital Caremeau
Nîmes, 30029
CHU de Toulouse - Hôpital de Rangueil
Toulouse, 50032 - 31059
Médecine Nucléaire de la Doua
Villeurbanne, 69100
Italy5 sites
IRCCS Azienda Ospedaliero-Universitaria di Bologna, Policlinico di Sant'Orsola
Bologna, BO, 40138
U.O. Clinica delle Malattie dell'Apparato Cardiovascolare - Ospedale Policlinico San Martino IRCCS
Genova, Genoa, 16132
U.O. Clinica delle Malattie dell'Apparato Cardiovascolare - Ospedale Policlinico San Martino IRCCS
Genova, 16132
Fondazione IRCCS Policlinico San Matteo - Centro per lo Studio e la Cura delle Amiloidosi Sistemiche
Pavia, 27100
Fondazione Toscana Gabriele Monasterio-UOC Cardiologia e Medicina Cardiovascolare
Pisa, 56124
Japan4 sites
Fukuoka Tokushukai Hospital
Kasuga, Fukuoka, 816-0864
Nagoya Tokushukai General Hospital
Kasugai, Aichi-ken, 487-0016
Okayama University Hospital
Okayama, 700-8558
Keio University Hospital
Shinjuku-ku, Tokyo, 160-8582
Poland4 sites
Uniwersytecki Szpital Kliniczny w Białymstoku
Bialystok, Podlaskie Voivodeship, 15-276
Krakowski Szpital Specjalistyczny im. Jana Pawła II
Krakow, Lesser Poland Voivodeship, 31-202
SPZOZ Uniwersytecki Szpital Kliniczny Im. Wojskowej Akademii Medycznej UM W Łodzi Centralny Szpital
Lodz, Łódź Voivodeship, 90-549
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu
Wroclaw, Lower Silesian Voivodeship, 50-556
Canada3 sites
University of Ottawa Heart Institute
Ottawa, Ontario, K1Y 4W7
CardioVasc HR Inc
Saint-Jean-sur-Richelieu, Quebec, J3A 1C3
Diex Recherche Trois-Rivieres
Trois-Rivières, Quebec, G9A 4P3
United Kingdom1 site
St George's Hospital, St George's University Hospitals NHS Foundation Trust
London, SW17 0QT
About this study
Official title: GLOBAL PREVALENCE OF TRANSTHYRETIN AMYLOID CARDIOMYOPATHY (ATTR-CM) IN PARTICIPANTS WITH HEART FAILURE WITH PRESERVED EJECTION FRACTION (HFpEF)
This study is a global, multi-center study designed to estimate the global prevalence of transthyretin amyloid cardiomyopathy (ATTR-CM) within a clinically at risk population [participants with heart failure with preserved ejection fraction (HFpEF)].
- Being tested
- Scintigraphy
- Keywords
- prevalence, transthyretin amyloid, ATTR-CM, HFpEF, scintigraphy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ScintigraphyDiagnostic test
scintigraphy
| Study group | Role | What they receive |
|---|---|---|
| ATTR-CM positive | Other | Participants diagnosed with ATTR-CM by scintigraphy Receives: Diagnostic Test: Scintigraphy |
| ATTR-CM negative | Other | Participants who are scintigraphy negative for ATTR-CM Receives: Diagnostic Test: Scintigraphy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Global Prevalence of ATTR-CM in HFpEF Participants Clinically At-Risk of Disease Among Total Evaluable Participants
Global prevalence of ATTR-CM in HFpEF participants was obtained by dividing the number of participants who were diagnosed with ATTR-CM in the study by the total number of HFpEF participants evaluated. Diagnosis of ATTR-CM was defined as: cardiac scintigraphy Grade 1, with confirmation of ATTR by cardiac biopsy; or cardiac scintigraphy Grade 2 or above. Exact 95% confidence intervals for the prevalence estimates were calculated using the method of Clopper and Pearson.
Measured: Day 1
Secondary outcomes (4)
Global Prevalence of ATTR-CM in Participants With HFpEF Clinically At-Risk of Disease by Subgroups (Regions, Age, Gender) Among Total Evaluable Participants
The prevalence of ATTR-CM in HFpEF participants was evaluated for subgroups including regions (North America, Europe, and Asia), age categories (60-64 years, 65-69 years, 70-74 years, 75-79 years, 80-85 years, 85-89 years, >=90 years), and gender (male, female). The estimate of prevalence was defined as the number of participants meeting the diagnosis criteria of ATTR-CM divided by the number of evaluable participants in the study in the given subgroup category. Diagnosis of ATTR-CM was defined as: cardiac scintigraphy Grade 1, with confirmation of ATTR by cardiac biopsy; or cardiac scintigraphy Grade 2 or above. Exact 95% confidence intervals for the prevalence estimates were calculated using the method of Clopper and Pearson.
Measured: Day 1
Number of Participants According to TTR Genotypes Among Participants Diagnosed With ATTR-CM
Number of participants diagnosed with ATTR-CM were evaluated for TTR genotypes (wild-type or hereditary form).
Measured: Day 1
Number of HFpEF Participants With and Without ATTR-CM Based on New York Heart Association (NYHA) Classification
Participants were evaluated using the NYHA classification at Day 1. Class I: participants with cardiac disease but without resulting limitations of physical activity. Class II: participants with cardiac disease resulting in slight limitation of physical activity. Class III: participants with cardiac disease resulting in marked limitation of physical activity. Class IV: participants with cardiac disease resulting in inability to carry on any physical activity without discomfort.
Measured: Day 1
N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) in Participants With and Without ATTR-CM
Participants were evaluated using NT-proBNP cardiac biomarker that was assessed at Day 1.
Measured: Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2020
- Study start30 Dec 2020
- Primary completion2 Jun 2023
- Study completion2 Jun 2023
Why it stopped: Sponsor terminated study early to not prolong timelines and to enable sharing of data collected which has potential to better support understanding of the prevalence of ATTR-CM in patients with HFpEF. Decision was not based on any safety findings.
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 Transthyretin Amyloid CardiomyopathyStudies of the same condition, open and under way.
- More trials run by PfizerThe 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 N/A
- Primary purpose
- Screening
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- To determine global prevalence of transthyretin amyloid cardiomyopathy in participants diagnosed with heart failure with preserved ejection fraction. No study treatment will be dispensed however, participants will undergo scintigraphy.
- Masking
- None (open label)
- Enrolment
- 347 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04424914
- Sponsor's protocol ID
- B3461087
- Other IDs
- HFpEF ATTR-CM prevalence study, 2020-002378-29, ATTR-POP
- Status last verified
- Feb 2024
- First submitted
- 27 May 2020
- Last updated
- 19 Jul 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
Publications
Yun S, Palladini G, Anderson LJ, Cariou E, Wang R, Angeli FS, Ebede B, Garcia-Pavia P. International prevalence of transthyretin amyloid cardiomyopathy in high-risk patients with heart failure and preserved or mildly reduced ejection fraction. Amyloid. 2024 Dec;31(4):291-301. doi: 10.1080/13506129.2024.2398446. Epub 2024 Sep 8.
PubMed 39245873
Study at a glance
From the registry record, last updated by the sponsor on 19 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- GLOBAL PREVALENCE OF TRANSTHYRETIN AMYLOID CARDIOMYOPATHY (ATTR-CM) IN PARTICIPANTS WITH HEART FAILURE WITH PRESERVED EJECTION FRACTION (HFpEF)
- ClinicalTrials.gov ID
- NCT04424914
- Acronym / sponsor ID
- B3461087
- Keywords
- prevalence, transthyretin amyloid, ATTR-CM, HFpEF, scintigraphy
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor terminated study early to not prolong timelines and to enable sharing of data collected which has potential to better support understanding of the prevalence of ATTR-CM in patients with HFpEF. Decision was not based on any safety findings.
- Enrolment
- 347 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- 30 Dec 2020 (actual)
- Primary completion
- 2 Jun 2023 (actual)
- Study completion
- 2 Jun 2023 (actual)
- First posted
- 11 Jun 2020 (actual)
- Results
- Posted 19 Jul 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is a global, multi-center study designed to estimate the global prevalence of transthyretin amyloid cardiomyopathy (ATTR-CM) within a clinically at risk population [participants with heart failure with preserved ejection fraction (HFpEF)].
Study design
- Primary purpose
- Screening
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- To determine global prevalence of transthyretin amyloid cardiomyopathy in participants diagnosed with heart failure with preserved ejection fraction. No study treatment will be dispensed however, participants will undergo scintigraphy.
| Arm | Type | Receives |
|---|---|---|
| ATTR-CM positive | Other | Diagnostic Test: ScintigraphyParticipants diagnosed with ATTR-CM by scintigraphy |
| ATTR-CM negative | Other | Diagnostic Test: ScintigraphyParticipants who are scintigraphy negative for ATTR-CM |
Who may qualify
- Ages
- 60 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Medical history of heart failure (HF) with:
- Left ventricular ejection fraction (LVEF) >40%.
- End-diastolic interventricular septal wall thickness (IVST) ≥12 mm.
- Willing and able to undergo scintigraphy.
Key exclusion criteria (6 listed)
- Diagnosis of heart failure with reduced ejection fraction (HFrEF) (EF ≤40%).
- Prior clinical history of myocardial infarction, CABG or multi-vessel obstructive coronary disease (>50% stenosis of ≥2 epicardial coronary arteries).
- Presence or history of any severe valvular heart disease (obstructive or regurgitant).
- A confirmed diagnosis of a non-amyloid infiltrative cardiomyopathy (ie, cardiac sarcoidosis, hemochromatosis), muscular dystrophies, cardiomyopathy with reversible causes, hypertrophic obstructive cardiomyopathy with known genetic etiology, or known pericardial constriction.
- Any type of diagnosed amyloidosis (eg, amyloid A amyloidosis, primary [light chain] amyloidosis) or prior diagnosis of ATTR-CM.
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 |
|---|---|---|
| Scintigraphyscintigraphy | Diagnostic test | ATTR-CM negative, ATTR-CM positive |
Primary outcome
Global Prevalence of ATTR-CM in HFpEF Participants Clinically At-Risk of Disease Among Total Evaluable Participants
Time frame: Day 1
Global prevalence of ATTR-CM in HFpEF participants was obtained by dividing the number of participants who were diagnosed with ATTR-CM in the study by the total number of HFpEF participants evaluated. Diagnosis of ATTR-CM was defined as: cardiac scintigraphy Grade 1, with confirmation of ATTR by cardiac biopsy; or cardiac scintigraphy Grade 2 or above. Exact 95% confidence intervals for the prevalence estimates were calculated using the method of Clopper and Pearson.
4 secondary outcomes
Global Prevalence of ATTR-CM in Participants With HFpEF Clinically At-Risk of Disease by Subgroups (Regions, Age, Gender) Among Total Evaluable Participants
Time frame: Day 1
Number of Participants According to TTR Genotypes Among Participants Diagnosed With ATTR-CM
Time frame: Day 1
Number of HFpEF Participants With and Without ATTR-CM Based on New York Heart Association (NYHA) Classification
Time frame: Day 1
N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) in Participants With and Without ATTR-CM
Time frame: Day 1
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jul 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39245873Yun S, Palladini G, Anderson LJ, Cariou E, Wang R, Angeli FS, Ebede B, Garcia-Pavia P. International prevalence of transthyretin amyloid cardiomyopathy in high-risk patients with heart failure and preserved or mildly reduced ejection fraction. Amyloid. 2024 Dec;31(4):291-301. doi: 10.1080/13506129.2024.2398446. Epub 2024 Sep 8.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
- Individual participant dataSharing planned: No. Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
Related trials for Transthyretin Amyloid Cardiomyopathy
Active, not recruitingObservational study
A Retrospective Observational Study of Epidemiology and Outcomes in Transthyretin Amyloid Cardiomyopathy in Germany
ATTR-CM (Transthyretin Amyloid Cardiomyopathy)
Active, not recruitingPhase 3
EPIC-ATTR: A Study to Evaluate the Effect of Eplontersen on the Transthyretin Reduction and Long-term Safety in Chinese Subjects With Transthyretin Amyloid Cardiomyopathy
Transthyretin Amyloid Cardiomyopathy
RecruitingObservational study
Essen Amyloidosis Registry
Amyloidosis; Systemic, Amyloidosis Cardiac
Active, not recruitingPhase 3
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Neuromuscular Disease, Neuromuscular Diseases (NMD)
RecruitingPhase 3
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
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.
Pfizer CT.gov Call Center study director
Pfizer
51 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Scintigraphy in people with Transthyretin Amyloid Cardiomyopathy and Heart Failure With Preserved Ejection Fraction, run by Pfizer at 51 sites in 8 countries.
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 6 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: stopped early, as of the sponsor's last update on 19 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 51 sites in United States, Spain, France, Italy, Japan and 3 more countries. 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?
Yes. Results are posted in the registry (first posted 19 Jul 2024). 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?
Global Prevalence of ATTR-CM in HFpEF Participants Clinically At-Risk of Disease Among Total Evaluable Participants (time frame: Day 1). 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 NCT04424914, 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 19 Jul 2024.