Registre HEAR, Healthcare European Amyloidosis Registry

NCT ID: NCT05101304

Last Updated: 2021-11-01

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

5000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-06-29

Study Completion Date

2027-12-31

Brief Summary

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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

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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:

1. Primary amyloidosis is caused by deposits of monoclonal immunoglobulin light chains produced by a plasma cell clone in the bone marrow.
2. 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.
3. Systemic senile amyloidosis which is due to deposits of wild-type (unmutated) transthyretin.
4. AA amyloidosis of chronic inflammatory causes.
5. 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.
6. 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

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Cardiac Amyloidosis

Study Design

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Observational Model Type

COHORT

Study Time Perspective

OTHER

Study Groups

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HEAR(Healthcare European Amyloidosis Registry)-Retrospective Cohort

Retrospective collection of deceased patients data with inclusion criteria

No interventions assigned to this group

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

No interventions assigned to this group

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

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

* Major patient
* Protected adult patient (guardianship or curatorship)
2. 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
3. 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
4. Retrospective cohort:

Deceased patients followed in the center with a confirmed diagnosis of amyloidosis

Study participants will not be compensated for their participation
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Saving Lives Matters

NETWORK

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Thibaud Pr Damy

Role: PRINCIPAL_INVESTIGATOR

Henri Mondor University Hospital

Locations

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Hôpital Henri Mondor

Créteil, , France

Site Status RECRUITING

Countries

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France

Central Contacts

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Mounira Kharoubi

Role: CONTACT

Phone: +33650029257

Email: [email protected]

Rébecca Gene

Role: CONTACT

Phone: +33628274249

Email: [email protected]

Facility Contacts

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Mounira Kharoubi

Role: primary

Other Identifiers

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HR01

Identifier Type: -

Identifier Source: org_study_id