Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT05101304 |
| Other study ID # |
HR01 |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
June 29, 2021 |
| Est. completion date |
December 31, 2027 |
Study information
| Verified date |
October 2021 |
| Source |
Saving Lives Matters |
| Contact |
Mounira Kharoubi |
| Phone |
+33650029257 |
| Email |
mounira.kharoubi[@]gmail.com |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical 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. 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.
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:
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.
