Characterizing wild-type transthyretin amyloidosis cardiomyopathy and its impact on quality of life
Phenotyping and Characterization of Danish Wild-type Transthyretin Amyloidosis Cardiomyopathy Patients: A Cross-sectional Study
This study is trying to understand how wild-type transthyretin amyloidosis cardiomyopathy affects the quality of life for patients at different stages of the disease compared to healthy individuals.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 120 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Aarhus University Hospital Skejby Academic / other |
| Locations | 1 site (Aarhus, Arrhus N) |
| Trial ID | NCT06291805 on ClinicalTrials.gov |
What this trial studies
This observational study aims to assess the quality of life (QoL) in 100 patients with wild-type transthyretin amyloidosis cardiomyopathy (ATTRwt-CM) across various stages of the disease. It will explore the correlation between disease severity and QoL measures, as well as investigate the presence of misfolded and fragmented transthyretin proteins in plasma, urine, and endomyocardial biopsies. A control group of 20 heart-healthy individuals will be included for comparison. The findings may provide insights into disease progression and response to treatment.
Who should consider this trial
Good fit: Ideal candidates include adults over 18 diagnosed with ATTRwt-CM through specific diagnostic methods.
Not a fit: Patients with AL amyloidosis or other significant cardiovascular diseases may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved monitoring and management strategies for patients with ATTRwt-CM, enhancing their quality of life.
How similar studies have performed: While the approach of correlating QoL with disease severity in amyloidosis is not widely tested, similar studies in related conditions have shown promising results.
Eligibility criteria
Show full inclusion / exclusion criteria
Group 1: wtATTR-CM patients Inclusion Criteria: * Patients \> 18 years diagnosed with ATTRwt-CM by: * endomyocardial biopsy * DPD scintigraphy with Perugini grade 2-3 where variant amyloidosis is ruled out due to genetic testing. * Informed oral and written consent Exclusion Criteria: * AL amyloidosis (light-chain amyloidosis). * Myelomatosis * Waldenström macroglobulinemia Group 2: Control group Inclusion Criteria: * Patients \> 18 years * Informed oral and written consent Exclusion Criteria: * Known cardiovascular disease including ischemic heart disease, heart failure, atrial fibrillation, presence of a pacemaker, or malignant hypertension. Well-controlled hypertension is acceptable. * Suspicion of cardiac amyloidosis assessed through clinical history, physical examination, ECG, and echocardiography focusing on "red flags": * Echocardiography with: * Myocardial hypertrophy (septum \>11 mm) * Apical sparing in LV-GLS * Infiltrative changes in the right ventricle free wall, thickened atrioventricular valves, or thickened atrial septum * Symptoms of polyneuropathy * Low voltage on ECG or discrepancy between left ventricular thickness and ECG amplitude indicative of low voltage * Atrioventricular block (AV block) * Bilateral carpal tunnel syndrome * Surgery for spinal stenosis * Elevated troponin I or NT-pro-BNP
Where this trial is running
Aarhus, Arrhus N
- Aarhus University Hospital — Aarhus, Arrhus N, Denmark (Recruiting)
Study contacts
- Study coordinator: Sie Kronborg Fensman, MD
- Email: siefensman@gmail.com
- Phone: +45 30 48 88 85
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.