Clinical trial NCT04776824: Swiss Cardiac Amyloidosis REgistry (Swiss-CARE)
Official title: Swiss Cardiac Amyloidosis REgistry (Swiss-CARE)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Amyloid Cardiomyopathy
Keywords: Cardiac Amyloidosis
Tafamidis
CMR feature tracking
Strain
T1 and T2 Mapping
Outcomes
Lead sponsor: Insel Gruppe AG, University Hospital Bern
Sponsor class: OTHER
First posted: 2021-03-02
Enrollment: 300
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Confirmed diagnosis of amyloidosis w/wo cardiac involvement

Brief summary: Cardiac transthyretin amyloidosis (ATTR), caused by ventricular depositions of misfolded transthyretin, results in an infiltrative cardiomyopathy, progressing from pronounced myocardial wall thickening, diastolic and systolic dysfunction to the development of terminal heart failure. Recently, treatment options for TTR amyloidosis have become available. However costs for therapy are enormous and previous trials were not able to differentiate between patients that might benefit from treatment and those without a need for treatment.

the investigators study aims to determine markers, as assessed by cardiac magnet resonance imaging (CMR) feature tracking (FT) and T1- and T2- mapping, that might reliably indicate disease severity and could help to identify patients that might benefit from (ongoing) TTR stabilization treatment.

Detailed description: Cardiac transthyretin amyloidosis (ATTR), the most common amyloidosis form with cardiac involvement, is caused by tissue deposition of misfolded TTR, a transport Protein for thyroxine and retinol. Ventricular depositions of amyloid fibrils results in an infiltrative cardiomyopathy, progressing from pronounced myocardial wall thickening, to diastolic and systolic dysfunction and finally chronic heart failure.

While treatment options are now available, it remains unclear how to monitor therapy response and disease progression. No makers have been identified that predict outcome prior to initiation of therapy, thus patient selection for therapy remains challenging.

The investigators study will address these issues and will provide systematically assessed CMR data before and over the course of 18 months after therapy initiation. Clinical and laboratory follow-up will be performed every 3-6 months. The investigators study is based on an open, uncontrolled, structured collection of retrospective and prospective data from all patients diagnosed with amyloidosis at the Inselspital Bern with the aim to follow patients undergoing therapy.

The investigators hypothesize that CMR feature tracking (FT) and measures of T1- and T2- mapping, such as extracellular volume (ECV) may better correlate with disease severity and help to identify patients likely to benefit from (ongoing) TTR stabilizing therapy. Beside standard CMR assessments, the investigators will use CMR feature tracking to quantify global and regional myocardial function. FT has proven to be an excellent predictor in various cardiomyopathies.

The proposed study will evaluate the potential of CMR to identify patients likely to benefit from therapy, monitor treatment response and balance individual patient benefit and health care cost.

Eligibility criteria: Inclusion Criteria:

* Confirmed diagnosis of amyloidosis w/wo cardiac involvement
* General Consent

Exclusion Criteria:

* Inability to give consent or existence of a written or documented oral refusal of the data subject.\<18 years of age

Primary outcomes:
measure: LV (left ventricle) and RV (right ventricle) function as assessed by CMR feature tracking as predictor for MACE (major adverse cardiac event)
timeFrame: 5 years
measure: LV and RV tissue characterization as assessed by T1 and T2 mapping as predictor for MACE
timeFrame: 5 years
measure: Late gadolinium enhancement as predictor for MACE
timeFrame: 5 years
measure: Extracellular volume (ECV) as predictor for MACE
timeFrame: 5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Basel
country: Switzerland
facility: USB
city: Bern
country: Switzerland
facility: Department of Cardiology, University Hospital Bern, Inselspital, Bern
city: Genf
country: Switzerland
facility: HUG
city: Lausanne
country: Switzerland
facility: CHUV
city: Luzern
country: Switzerland
facility: LUKS
city: St. Gallen
country: Switzerland
facility: KSSG
city: Zürich
country: Switzerland
facility: Stadtspital Triemli
Study officials:
affiliation: Department of Cardiology, University Hospital Bern, Inselspital, Bern
name: Christoph Gräni, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: