Cardiac AmyloidosisICARNCT06894290
International Cardiac Amyloidosis Registry
An observational study of Cardiac Amyloidosis, run by Leiden University Medical Center at 6 sites in 5 countries.
Trial summary
Cardiac amyloidosis is a relatively rare disease. However, with the newer imaging techniques that have become available to us in recent years, determining the diagnosis is becoming more common. There are several variants. Each variant involves protein accumulation between heart muscle cells. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cardiac Amyloidosis
- How many people
- 300 participants
- Where
- 6 sitesin 5 countries
- Study dates
- 30 Jun 2023to Jul 2026
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: All consecutive patients with diagnosis of AL or ATTR diagnosis will be prospectively included from 1st of January 2023 in the registry (if consent is provided). Currently, about 10 patients per year are diagnosed in the LUMC, therefore multicenter cooperation is needed to include sufficient patients. We have a consortium of at least 15 centers that are willing to participate. Therefore we estimate an inclusion of ~150 patients per year. The rate of diagnosis will probably increase with increasing awareness for amyloidosis, with potentially increasing inclusion rates. An estimated 1/3 of patient will have AL and 2/3 will have ATTR amyloidosis.
Who may be able to join
- Diagnosed with AL or ATTR cardiac amyloidosis following the recommendations of the European Society of Cardiology [1] which include:
- Signs and symptoms, ECG, echo or CMR suggestive of cardiac amyloidosis and
- negative hematological test and bone scintigraphy Perugini stage 2 or 3 (is diagnostic for ATTR amyloidosis)
- negative hematological test and bone scintigraphy Perugini stage 1 and histological confirmation (either extracardiac or cardiac biopsy) with subtyping of ATTR amyloidosis
- positive hematological test and bone scintigraphy Perugini stage 1-3 and histological confirmation (usually cardiac biopsy) with subtyping of ATTR amyloidosis
- positive hematological rest and bone scintigraphy Perugini stage 0, MRI consistent with amyloidosis and histological confirmation (either extracardiac or cardiac biopsy) with subtyping AL amyloidosis
Who cannot join
- Unable to provide informed consent due to insufficient language proficiency or intellectual capabilities
Trial locations and contacts
6 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
6 sites in 5 countries
Belgium2 sites, 2 recruiting
- Recruiting
AZ St Jan
Bruges, 8000
Philippe Debonnaire, MD, PhD, +32 50 45 21 11, Philippe.Debonnaire@azsintjan.be
- Recruiting
UZ Antwerpen
Edegem, 2650
Victor Sarli Issa, MD, +32 3 821 30 00, VictorSarli.Issa@uza.be
Italy1 site, 1 recruiting
- Recruiting
Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Roma
Roma, 00168
Francesca Graziani, MD, PhD, +39 06 30151, francesca.graziani@policlinicogemelli.it
Japan1 site, 1 recruiting
- Recruiting
Kitasato University Hospital
Sagamihara, 252-0375
Takeru Nabeta, MD, +81 42-778-8111, takerunabeta@gmail.com
Netherlands1 site, 1 recruiting
- Recruiting
Leiden University Medical Center
Leiden, 2333ZA
Nina Ajmone Marsan, MD, PhD, +31715262020, n.ajmone@lumc.nl
Madelien Regeer, MD, PhD
Portugal1 site, 1 recruiting
- Recruiting
Santa Marta Hospital, CHULC, Lisbon
Lisbon, 1169-024
Ana Timoteo, MD, PhD, +351 21 359 4000, ana_timoteo@yahoo.com
About this study
Cardiac amyloidosis is a relatively rare disease. However, with the newer imaging techniques that have become available to us in recent years, determining the diagnosis is becoming more common. There are several variants. Each variant involves protein accumulation between heart muscle cells. This leads to the heart stiffening and, as a result, the heart has a hard time filling. This can lead to heart failure with complaints such as fluid retention, cardiac arrhythmias such as atrial fibrillation, conduction abnormalities that sometimes require a pacemaker, and clot formation in the heart that can cause a stroke and narrowing of the aortic valve. Getting the correct diagnosis is important because specific treatment is available in some cases for the different variants of cardiac amyloidosis. This research is needed to better understand the course of this disease profile and which patients respond well to the specific treatment.
The aim of this research is to find out more about the course of the cardiac amyloidosis disease. The investigators see to what extent patients deteriorate in their condition due to the disease and how often they need to be hospitalized. Furthermore, the investigators want to learn to what extent certain abnormalities (on e.g. cardiac ultrasound) can predict how quickly clinical deterioration occurs. Finally, for certain forms of cardiac amyloidosis there is a specific drug treatment. Through this research, the investigators want to try to determine who really benefits from the specific medication in order to prescribe it to the right patients.
Read the detailed description
Rationale: Patients with cardiac amyloidosis are in general underdiagnosed and undertreated. AL amyloidosis is a disease with a very poor prognosis if left untreated. For ATTR amyloidosis, recently, specific treatment has become available for patients fulfilling certain criteria with good results in clinical trials. The rationale behind this prospective registry is to collect real-world data on diagnostic process, management, progression of disease and outcome of patients with cardiac AL and ATTR amyloidosis who are treated following local standards and to obtain (echocardiographic) predictors of prognosis in these patients.
Objective: The objective of the study is to evaluate the diagnostic process from onset symptoms to diagnosis, the outcome in a real-world cohort of amyloidosis patients and to find predictors (both clinical and echocardiographic) of outcome Study design: Prospective long-term observational study Study population: All patients >18 years old, prospectively diagnosed with cardiac AL or ATTR amyloidosis Intervention (if applicable): n/a Main study parameters/endpoints: The main study endpoints are death, heart failure hospitalization and cardiovascular events Nature and extent of the burden and risks associated with participation, benefit and group relatedness: Patients who participate in the registry will undergo structured follow up according to the clinical care pathway for amyloidosis patients based on the recommendations in the most recent Position Statement from the European Society of Cardiology [1]. There will be no additional visits planned, therefore no additional burden is associated with participation in the registry. Patients agree that their anonymized data is shared with other centers within the amyloidosis consortium.
- Conditions
- Cardiac Amyloidosis
- Keywords
- Cardiac amyloidosis, ATTR amyloidosis, AL amyloidosis, Echocardiography
- Medical subject headings
- Amyloid Neuropathies, Familial, Immunoglobulin Light-chain Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| CA | Patients diagnosed with cardiac amyloidosis. Either ATTR-CA or AL-CA. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mortality
The main study endpoint is all-cause mortality
Measured: 1 year
Secondary outcomes (3)
Progression
Progression of disease (defined by disease specific progression stages such as Mayo score for AL amyloidosis and NAC score for ATTR amyloidosis)
Measured: 1 year
HFH
Heart failure hospitalization
Measured: 1 year
Events
Cardiovascular events (including cerebrovascular accident and other thromboembolic events, cardiac arrhythmias and pacemaker implantations)
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 posted25 Mar 2025
- Study start30 Jun 2023
- Primary completionJul 2026 (estimated)
- Study completionJul 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)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 Leiden University 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 300 participants (estimated)
- Target duration
- 2 Years
Sponsor and investigators
- Lead sponsor
- Leiden University Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Pfizer
- Responsible party
- Principal investigator, Nina Ajmone Marsan, Leiden University Medical Center
- Investigators
- Nina Ajmone Marsan, MD, PhD, Principal investigator, Leiden University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06894290
- Sponsor's protocol ID
- ICAR
- Acronym
- ICAR
- Status last verified
- Mar 2025
- First submitted
- 12 Mar 2025
- Last updated
- 25 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: We will share individual participant data between centers when asked for, but we will not make it public available
Publications
Bukhari S, Khan SZ, Bashir Z. Atrial Fibrillation, Thromboembolic Risk, and Anticoagulation in Cardiac Amyloidosis: A Review. J Card Fail. 2023 Jan;29(1):76-86. doi: 10.1016/j.cardfail.2022.08.008. Epub 2022 Sep 17.
PubMed 36122817Maurer MS, Schwartz JH, Gundapaneni B, Elliott PM, Merlini G, Waddington-Cruz M, Kristen AV, Grogan M, Witteles R, Damy T, Drachman BM, Shah SJ, Hanna M, Judge DP, Barsdorf AI, Huber P, Patterson TA, Riley S, Schumacher J, Stewart M, Sultan MB, Rapezzi C; ATTR-ACT Study Investigators. Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy. N Engl J Med. 2018 Sep 13;379(11):1007-1016. doi: 10.1056/NEJMoa1805689. Epub 2018 Aug 27.
PubMed 30145929Garcia-Pavia P, Rapezzi C, Adler Y, Arad M, Basso C, Brucato A, Burazor I, Caforio ALP, Damy T, Eriksson U, Fontana M, Gillmore JD, Gonzalez-Lopez E, Grogan M, Heymans S, Imazio M, Kindermann I, Kristen AV, Maurer MS, Merlini G, Pantazis A, Pankuweit S, Rigopoulos AG, Linhart A. Diagnosis and treatment of cardiac amyloidosis. A position statement of the European Society of Cardiology Working Group on Myocardial and Pericardial Diseases. Eur J Heart Fail. 2021 Apr;23(4):512-526. doi: 10.1002/ejhf.2140. Epub 2021 Apr 7.
PubMed 33826207
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- International Cardiac Amyloidosis Registry
- ClinicalTrials.gov ID
- NCT06894290
- Acronym / sponsor ID
- ICAR / ICAR
- Conditions
- Cardiac Amyloidosis
- Keywords
- Cardiac amyloidosis, ATTR amyloidosis, AL amyloidosis, Echocardiography
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Leiden University Medical CenterUniversity, hospital or other
- Collaborators
- Pfizer
- Study start
- 30 Jun 2023 (actual)
- Primary completion
- Jul 2026 (estimated)
- Study completion
- Jul 2026 (estimated)
- First posted
- 25 Mar 2025 (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.
Cardiac amyloidosis is a relatively rare disease. However, with the newer imaging techniques that have become available to us in recent years, determining the diagnosis is becoming more common. There are several variants. Each variant involves protein accumulation between heart muscle cells. This leads to the heart stiffening and, as a result, the heart has a hard time filling. This can lead to heart failure with complaints such as fluid retention, cardiac arrhythmias such as atrial fibrillation, conduction abnormalities that sometimes require a pacemaker, and clot formation in the heart that can cause a stroke and narrowing of the aortic valve. Getting the correct diagnosis is important because specific treatment is available in some cases for the different variants of cardiac amyloidosis. This research is needed to better understand the course of this disease profile and which patients respond well to the specific treatment.
The aim of this research is to find out more about the course of the cardiac amyloidosis disease. The investigators see to what extent patients deteriorate in their condition due to the disease and how often they need to be hospitalized. Furthermore, the investigators want to learn to what extent certain abnormalities (on e.g. cardiac ultrasound) can predict how quickly clinical deterioration occurs. Finally, for certain forms of cardiac amyloidosis there is a specific drug treatment. Through this research, the investigators want to try to determine who really benefits from the specific medication in order to prescribe it to the right patients.
Detailed description
Rationale: Patients with cardiac amyloidosis are in general underdiagnosed and undertreated. AL amyloidosis is a disease with a very poor prognosis if left untreated. For ATTR amyloidosis, recently, specific treatment has become available for patients fulfilling certain criteria with good results in clinical trials. The rationale behind this prospective registry is to collect real-world data on diagnostic process, management, progression of disease and outcome of patients with cardiac AL and ATTR amyloidosis who are treated following local standards and to obtain (echocardiographic) predictors of prognosis in these patients.
Objective: The objective of the study is to evaluate the diagnostic process from onset symptoms to diagnosis, the outcome in a real-world cohort of amyloidosis patients and to find predictors (both clinical and echocardiographic) of outcome Study design: Prospective long-term observational study Study population: All patients >18 years old, prospectively diagnosed with cardiac AL or ATTR amyloidosis Intervention (if applicable): n/a Main study parameters/endpoints: The main study endpoints are death, heart failure hospitalization and cardiovascular events Nature and extent of the burden and risks associated with participation, benefit and group relatedness: Patients who participate in the registry will undergo structured follow up according to the clinical care pathway for amyloidosis patients based on the recommendations in the most recent Position Statement from the European Society of Cardiology [1]. There will be no additional visits planned, therefore no additional burden is associated with participation in the registry. Patients agree that their anonymized data is shared with other centers within the amyloidosis consortium.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Target follow-up
- 2 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CA | – | –Patients diagnosed with cardiac amyloidosis. Either ATTR-CA or AL-CA. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- All consecutive patients with diagnosis of AL or ATTR diagnosis will be prospectively included from 1st of January 2023 in the registry (if consent is provided). Currently, about 10 patients per year are diagnosed in the LUMC, therefore multicenter cooperation is needed to include sufficient patients. We have a consortium of at least 15 centers that are willing to participate. Therefore we estimate an inclusion of ~150 patients per year. The rate of diagnosis will probably increase with increasing awareness for amyloidosis, with potentially increasing inclusion rates. An estimated 1/3 of patient will have AL and 2/3 will have ATTR amyloidosis.
Key inclusion criteria (6 listed)
- Diagnosed with AL or ATTR cardiac amyloidosis following the recommendations of the European Society of Cardiology [1] which include:
- Signs and symptoms, ECG, echo or CMR suggestive of cardiac amyloidosis and
- negative hematological test and bone scintigraphy Perugini stage 2 or 3 (is diagnostic for ATTR amyloidosis)
- negative hematological test and bone scintigraphy Perugini stage 1 and histological confirmation (either extracardiac or cardiac biopsy) with subtyping of ATTR amyloidosis
- positive hematological test and bone scintigraphy Perugini stage 1-3 and histological confirmation (usually cardiac biopsy) with subtyping of ATTR amyloidosis
Key exclusion criteria (1 listed)
- Unable to provide informed consent due to insufficient language proficiency or intellectual capabilities
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
Mortality
Time frame: 1 year
The main study endpoint is all-cause mortality
3 secondary outcomes
Progression
Time frame: 1 year
HFH
Time frame: 1 year
Events
Time frame: 1 year
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 25 Mar 2025.
- Background: PubMed 36122817Bukhari S, Khan SZ, Bashir Z. Atrial Fibrillation, Thromboembolic Risk, and Anticoagulation in Cardiac Amyloidosis: A Review. J Card Fail. 2023 Jan;29(1):76-86. doi: 10.1016/j.cardfail.2022.08.008. Epub 2022 Sep 17.
- Background: PubMed 30145929Maurer MS, Schwartz JH, Gundapaneni B, Elliott PM, Merlini G, Waddington-Cruz M, Kristen AV, Grogan M, Witteles R, Damy T, Drachman BM, Shah SJ, Hanna M, Judge DP, Barsdorf AI, Huber P, Patterson TA, Riley S, Schumacher J, Stewart M, Sultan MB, Rapezzi C; ATTR-ACT Study Investigators. Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy. N Engl J Med. 2018 Sep 13;379(11):1007-1016. doi: 10.1056/NEJMoa1805689. Epub 2018 Aug 27.
- Background: PubMed 33826207Garcia-Pavia P, Rapezzi C, Adler Y, Arad M, Basso C, Brucato A, Burazor I, Caforio ALP, Damy T, Eriksson U, Fontana M, Gillmore JD, Gonzalez-Lopez E, Grogan M, Heymans S, Imazio M, Kindermann I, Kristen AV, Maurer MS, Merlini G, Pantazis A, Pankuweit S, Rigopoulos AG, Linhart A. Diagnosis and treatment of cardiac amyloidosis. A position statement of the European Society of Cardiology Working Group on Myocardial and Pericardial Diseases. Eur J Heart Fail. 2021 Apr;23(4):512-526. doi: 10.1002/ejhf.2140. Epub 2021 Apr 7.
- Individual participant dataSharing planned: No. We will share individual participant data between centers when asked for, but we will not make it public available
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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.
Nina Ajmone Marsan, MD, PhD principal investigator
Leiden University Medical Center
6 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cardiac Amyloidosis, run by Leiden University Medical Center at 6 sites in 5 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 1 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 25 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 6 sites in Belgium, Italy, Japan, Netherlands, Portugal. 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 Nina Ajmone Marsan, MD, PhD (+31715262020, N.ajmone@lumc.nl), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mortality (time frame: 1 year). 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 NCT06894290, which also lists 3 linked publications.
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 25 Mar 2025.