Cardiac AmyloidosisNCT05101304
Registre HEAR, Healthcare European Amyloidosis Registry
An observational study of Cardiac Amyloidosis, run by Saving Lives Matters at 1 site in France.
Trial summary
This is a non-interventional, prospective, retrospective, non-comparative, multi-center study. In order not to interfere with patient management, the study is observational. Thus, no follow-up visit is imposed. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Cardiac Amyloidosis
- How many people
- 5,000 participants
- Where
- 1 siteFrance
- Study dates
- 29 Jun 2021to 31 Dec 2027
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: This study is intended for all patients with a confirmed or suspected diagnosis of cardiac amyloidosis.
Who may be able to join
- Patients must meet all of the following inclusion criteria to be included in the study:
- Major patient
- Protected adult patient (guardianship or curatorship)
- Prospective Cohort:
- Patients referred or who have been referred to the participating centre for suspected amyloidosis.
- Patient who signed the patient information "Prospective Cohort" note
Show all 12Show fewer
- Retro-prospective Cohort:
- Patient already followed in the center with a confirmed diagnosis of amyloidosis Patient who signed the "retro-prospective cohort" patient information note
- Retrospective cohort:
- Deceased patients followed in the center with a confirmed diagnosis of amyloidosis
- Study participants will not be compensated for their participation
- EXCLUSION CRITERIA The patient has expressed his/her refusal to participate Participation in another study, even an interventional one, is not a criterion for non-inclusion.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site, 1 recruiting
- Recruiting
Hôpital Henri Mondor
Créteil, 94000
Mounira Kharoubi, +33650029257, mounira.kharoubi@gmail.com
Thibaud Damy
About this study
This is a non-interventional, prospective, retrospective, non-comparative, multi-center study.
In order not to interfere with patient management, the study is observational. Thus, no follow-up visit is imposed. The data collection will be limited to the data related to the management of the patients included throughout their follow-up.
This study is intended for all patients with a confirmed or suspected diagnosis of cardiac amyloidosis. Three cohorts will be identified: the HEAR (Healthcare European Amyloidosis Registry)-Retrospective Cohort, the HEAR(Healthcare European Amyloidosis Registry)-Retrospective-Prospective Cohort and the HEAR (Healthcare European Amyloidosis Registry)-Prospective Cohort.
Read the detailed description
Amyloidosis is a rare disease characterized by infiltration and continuous accumulation of insoluble fibrillar proteins in the extracellular matrix in various organs including kidney, nerve, liver, heart and skeletal muscle. Its prevalence is estimated at 0.5-1.3/100,000. The main forms are:
- Primary amyloidosis is caused by deposits of monoclonal immunoglobulin light chains produced by a plasma cell clone in the bone marrow.
- Hereditary (familial) amyloidosis, the major form of which is mutated transthyretin amyloidosis of autosomal dominant transmission. More than 100 different mutations of transthyretin are known and several mutations have been described as amyloidogenic.
- Systemic senile amyloidosis which is due to deposits of wild-type (unmutated) transthyretin.
- AA amyloidosis of chronic inflammatory causes.
- Localized amyloidosis. They are in the vast majority of cases primary amyloidosis (or immunoglobulinic) amyloidosis. The deposition of amyloidosis formed by light chains of antibodies occurs here in contact with a proliferation of plasma cells located in a particular organ. There is no passage of the immunoglobulin light chain into the bloodstream and therefore deposits do not form remotely in other organs.
- Rare amyloidoses. The prognosis of the disease is most often related to the cardiac involvement. Unfortunately, its diagnosis is often delayed, which worsens the prognosis. This delay is linked to the absence of simple diagnostic tools (biomarkers, imaging, etc.) allowing early diagnosis of the disease. The absence of early diagnostic tools, the heterogeneity of the expression (multi-systemic) of this disease and the difficulty of its management lead to delays in diagnosis and non-management of certain organ disorders, which have an impact on the quality of life of patients.
There is a strong need to help physicians better characterize the clinical and biological presentations of the disease and to improve diagnostic tools and standardize therapeutic management.
All data collected for the study are key, routine data for the condition, readily available in the patients' medical records. It is also possible to use additional and specific computerized tools to collect these data, within the participating expert centers.
Data will be recorded in an electronic observation book.
- Conditions
- Cardiac Amyloidosis
- Medical subject headings
- Amyloid Neuropathies, Familial
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 |
|---|---|---|
| HEAR(Healthcare European Amyloidosis Registry)-Retrospective Cohort | Retrospective collection of deceased patients data with inclusion criteria | |
| HEAR(Healthcare European Amyloidosis Registry)-Retrospective-Prospective Cohort | Retrospective and prospective collection of patient data and real-life follow-up of patients from the date of inclusion Living patients who met the inclusion criteria | |
| HEAR(Healthcare European Amyloidosis Registry)-Prospective Cohort | Prospective data collection and real-life follow-up of patients from the date of inclusion These patients are either newly followed in the centre with the inclusion criteria |
What is being measured
The primary outcome is the main question the study is designed to answer.
The clinical, biological and imaging characteristics of patients with cardiac amyloidosis
Describe the clinical, biological and imaging characteristics of patients with cardiac amyloidosis.
Measured: 6 years
Secondary outcomes (2)
he prevalence and incidence of the different types of amyloidosis and their evolution.
To estimate the prevalence and incidence of the different types of amyloidosis and their evolution.
Measured: 6 years
The therapeutic management (cardiological and specific), their beneficial and secondary effects.
Describe the therapeutic management (cardiological and specific), their beneficial and secondary effects.
Measured: 6 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Nov 2021
- Study start29 Jun 2021
- Primary completion30 Jun 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cardiac 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
- Cohort
- Time perspective
- Other
- Enrolment
- 5,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Saving Lives Matters
- Sponsor type
- Research network
- Responsible party
- Sponsor
- Investigators
- Thibaud Pr Damy, Principal investigator, Henri Mondor University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05101304
- Sponsor's protocol ID
- HR01
- Status last verified
- Oct 2021
- First submitted
- 5 Oct 2021
- Last updated
- 1 Nov 2021
- Results posted
- 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 1 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Registre HEAR, Healthcare European Amyloidosis Registry
- ClinicalTrials.gov ID
- NCT05101304
- Acronym / sponsor ID
- HR01
- Conditions
- Cardiac Amyloidosis
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 5,000 participants (estimated)
- Lead sponsor
- Saving Lives MattersResearch network
- Study start
- 29 Jun 2021 (actual)
- Primary completion
- 30 Jun 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 1 Nov 2021 (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.
This is a non-interventional, prospective, retrospective, non-comparative, multi-center study.
In order not to interfere with patient management, the study is observational. Thus, no follow-up visit is imposed. The data collection will be limited to the data related to the management of the patients included throughout their follow-up.
This study is intended for all patients with a confirmed or suspected diagnosis of cardiac amyloidosis. Three cohorts will be identified: the HEAR (Healthcare European Amyloidosis Registry)-Retrospective Cohort, the HEAR(Healthcare European Amyloidosis Registry)-Retrospective-Prospective Cohort and the HEAR (Healthcare European Amyloidosis Registry)-Prospective Cohort.
Detailed description
Amyloidosis is a rare disease characterized by infiltration and continuous accumulation of insoluble fibrillar proteins in the extracellular matrix in various organs including kidney, nerve, liver, heart and skeletal muscle. Its prevalence is estimated at 0.5-1.3/100,000. The main forms are:
- Primary amyloidosis is caused by deposits of monoclonal immunoglobulin light chains produced by a plasma cell clone in the bone marrow.
- Hereditary (familial) amyloidosis, the major form of which is mutated transthyretin amyloidosis of autosomal dominant transmission. More than 100 different mutations of transthyretin are known and several mutations have been described as amyloidogenic.
- Systemic senile amyloidosis which is due to deposits of wild-type (unmutated) transthyretin.
- AA amyloidosis of chronic inflammatory causes.
- Localized amyloidosis. They are in the vast majority of cases primary amyloidosis (or immunoglobulinic) amyloidosis. The deposition of amyloidosis formed by light chains of antibodies occurs here in contact with a proliferation of plasma cells located in a particular organ. There is no passage of the immunoglobulin light chain into the bloodstream and therefore deposits do not form remotely in other organs.
- Rare amyloidoses. The prognosis of the disease is most often related to the cardiac involvement. Unfortunately, its diagnosis is often delayed, which worsens the prognosis. This delay is linked to the absence of simple diagnostic tools (biomarkers, imaging, etc.) allowing early diagnosis of the disease. The absence of early diagnostic tools, the heterogeneity of the expression (multi-systemic) of this disease and the difficulty of its management lead to delays in diagnosis and non-management of certain organ disorders, which have an impact on the quality of life of patients.
There is a strong need to help physicians better characterize the clinical and biological presentations of the disease and to improve diagnostic tools and standardize therapeutic management.
All data collected for the study are key, routine data for the condition, readily available in the patients' medical records. It is also possible to use additional and specific computerized tools to collect these data, within the participating expert centers.
Data will be recorded in an electronic observation book.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| HEAR(Healthcare European Amyloidosis Registry)-Retrospective Cohort | – | –Retrospective collection of deceased patients data with inclusion criteria |
| HEAR(Healthcare European Amyloidosis Registry)-Retrospective-Prospective Cohort | – | –Retrospective and prospective collection of patient data and real-life follow-up of patients from the date of inclusion Living patients who met the inclusion criteria |
| HEAR(Healthcare European Amyloidosis Registry)-Prospective Cohort | – | –Prospective data collection and real-life follow-up of patients from the date of inclusion These patients are either newly followed in the centre with the inclusion criteria |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- This study is intended for all patients with a confirmed or suspected diagnosis of cardiac amyloidosis.
Key inclusion criteria (12 listed)
- Patients must meet all of the following inclusion criteria to be included in the study:
- Prospective Cohort:
- Patients referred or who have been referred to the participating centre for suspected amyloidosis.
- Patient who signed the patient information "Prospective Cohort" note
- Retro-prospective Cohort:
Key exclusion criteria (0 listed)
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
The clinical, biological and imaging characteristics of patients with cardiac amyloidosis
Time frame: 6 years
Describe the clinical, biological and imaging characteristics of patients with cardiac amyloidosis.
2 secondary outcomes
he prevalence and incidence of the different types of amyloidosis and their evolution.
Time frame: 6 years
The therapeutic management (cardiological and specific), their beneficial and secondary effects.
Time frame: 6 years
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 1 Nov 2021.
- Individual participant dataSharing planned: No
Related trials for Cardiac Amyloidosis
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
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A Phase 3 Study of NTLA-2001 in ATTRv-PN
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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.
Thibaud Pr Damy principal investigator
Henri Mondor University Hospital
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 Saving Lives Matters at 1 site in France.
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 12 inclusion and 0 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 1 Nov 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 France. 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 Mounira Kharoubi (+33650029257, mounira.kharoubi@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The clinical, biological and imaging characteristics of patients with cardiac amyloidosis (time frame: 6 years). 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 1 Nov 2021.