Characterizing wild-type transthyretin amyloidosis cardiomyopathy and its impact on quality of life

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Acoramidis for ATTR-CM: Cardiac Imaging Study

  This study tests whether acoramidis can improve heart structure and function in people with transthyretin amyloid cardiomyopathy.

  Recruiting Phase 4 Eidos Therapeutics, a BridgeBio company · St Louis, Missouri · NCT07695701

• Ocular Imaging to help detect ATTR-CM in adults with heart failure

  This multicenter pilot study tests whether eye imaging can help distinguish people with transthyretin amyloid cardiomyopathy from other heart failure patients.

  Recruiting AstraZeneca · Palo Alto, California and 19 other locations · NCT07690436

• Long-term safety of eplontersen in adults with transthyretin amyloidosis, including those with prior liver transplant or severe liver impairment

  This study will see if eplontersen remains safe over time for adults with transthyretin amyloidosis, including people who have had a liver transplant or have severe liver problems.

  Recruiting at 6 sites AstraZeneca · Los Angeles, California and 5 other locations · NCT07654855

• Annular ligament biopsy during endoscopic carpal tunnel surgery to screen for transthyretin amyloidosis

  This study tests whether a biopsy of the annular ligament during carpal tunnel surgery can reliably indicate transthyretin amyloidosis and match the results of the standard synovial biopsy.

  Recruiting Clinique Saint Jean, France · Montpellier · NCT07644325

• Coramitug PET/CT imaging to track and affect heart amyloid in ATTR-CM

  This study tests whether a radioactive version of the antibody coramitug can be detected in and displaced from heart amyloid in people aged 60 and older with transthyretin amyloid cardiomyopathy, and whether the drug is safe and well tolerated.

  Recruiting Phase 1 Novo Nordisk A/S · Groningen · NCT07448623

• Switching from tafamidis to acoramidis to see how transthyretin blood levels change

  This test will see if switching people with transthyretin amyloid cardiomyopathy who are taking tafamidis to acoramidis raises transthyretin levels in the blood.

  Recruiting at 10 sites Phase 4 Bayer · Sankt Pölten, Lower Austria and 25 other locations · NCT07298044

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.