Ultrasound therapy for cardiac amyloidosis

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 pilot clinical study aims to evaluate the effects of sonotherapy on myocardial blood flow and cardiac function in patients with cardiac amyloidosis. A total of 70 subjects will be enrolled, including 30 with light chain cardiac amyloidosis, 30 with transthyretin cardiac amyloidosis, and 10 control subjects without amyloidosis. Participants will undergo various imaging techniques, including echocardiography and perfusion PET, to assess changes before and after treatment.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Age \> 18 years for AL-CA, \> 65 years for ATTR-CA, \> 65 years for controls
* Willing and able to provide consent
* Diagnosis of systemic light chain amyloidosis by standard criteria (see below) and in hematological remission (normal serum free light chain levels)

  * (immunofixation of serum and urine, IgG free light chain (FLC) assay, a biopsy of fat pad/ bone marrow, or organ biopsy, followed by typing of the light chain using immunohistochemistry or immunogold assay with confirmation by Mass spectroscopy as needed) AND
  * proof of cardiac involvement by AL amyloidosis
  * abnormal cardiac biomarkers (Abnormal high sensitivity TnT 5th generation levels (\>9 ng/L: female, \>14 ng/L: male) or abnormal age appropriate N terminal pro-brain natriuretic peptide, NT-proBNP (abnormal values: \<50 years: \>450 pg/ml; 50-75 years:\>900 pg/ml; \>75 years: \>1800 pg/ml) or
  * abnormal echocardiogram (wall thickness \> 12 mm) or
  * abnormal cardiac MRI (wall thickness \> 12 mm or extracellular volume \> 0.35) OR
* Diagnosis of transthyretin cardiac amyloidosis by standard criteria

  * endomyocardial biopsy followed by typing of the transthyretin amyloidosis using immunohistochemistry or immunogold assay with confirmation by Mass spectroscopy as needed
  * extracardiac biopsy with typical cardiac imaging findings, or
  * grade 2 or grade 3 myocardial uptake of technetium-99m pyrophosphate (PYP) if AL amyloidosis is excluded

Exclusion Criteria:

* Hemodynamic instability
* Severe claustrophobia despite use of sedatives
* Decompensated heart failure (unable to lie flat for 1 hour)
* Concomitant non-ischemic non-amyloid heart disease (valvular heart disease or dilated cardiomyopathy)
* Severe valve stenosis or regurgitation in the aortic, mitral or tricuspid valves, including prior valve replacement
* Severe pulmonary artery hypertension
* Severe lung disease
* Known obstructive epicardial coronary artery disease with stenosis \> 50% in any single territory
* Prior cardiac surgery
* Regional wall motion abnormality on echocardiogram
* Left ventricular ejection fraction \< 40%
* Pregnant state
* Documented allergy to N-13 ammonia or Definity
* Contraindications to DEFINITY® (Perflutren Lipid Microsphere) Injectable Suspension:

  o Patients with known or suspected Right-to-left, bi-directional, or transient right-to-left cardiac shunts, Hypersensitivity to perflutren
* Contraindications or challenges to sonotherapy

  * Severe electrolyte abnormalities
  * QTc prolongation (values are greater than 450 milliseconds in males and greater than 470 milliseconds in females)
  * BMI \> 35 kg/m2
  * Documented intracardiac thrombus
  * Atrial fibrillation not on anticoagulation
  * Prior history of stroke
* Any other reason determined by the investigator that makes a subject a poor candidate for ultrasound therapy

Where it's running

• Brigham and Women's Hospital
  Boston, Massachusetts, United States Recruiting

Study contacts

• Principal investigator: Sharmila Dorbala, MD — Brigham and Women's Hospital
• Study coordinator: Sharmila Dorbala, MD
• Email: sdorbala@bwh.harvard.edu
• Phone: 617-732-6290

Similar studies

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

• Detecting cardiac amyloidosis in people with severe aortic valve narrowing

  This study tests non-invasively whether imaging tests can find heart amyloidosis in patients with severe aortic stenosis who are being evaluated for valve replacement.

  Recruiting University Hospital Tuebingen · Tübingen · NCT07752732

• Multimodal analysis of heart biopsy samples

  This project will take one extra small heart biopsy during a clinically needed procedure to test whether detailed molecular and immune analyses can find disease-specific signatures in adults with cardiomyopathies, myocarditis, cardiac amyloidosis, or after heart transplant.

  Recruiting University Hospital, Essen · Essen, North Rhine-Westphalia · NCT07359690

• Using tiny blood particles to detect and track ATTR heart amyloidosis

  This project will test whether tiny blood particles called extracellular vesicles can help detect and track transthyretin (ATTR) amyloid cardiomyopathy in people with or at risk for ATTR.

  Recruiting University of Sao Paulo General Hospital · São Paulo, São Paulo · NCT07314268

• Belgian Amyloidosis Biobank and Patient Registry at UZ Leuven

  This project collects and stores blood, tissue samples, and medical data from adults with suspected or confirmed amyloidosis to create a biobank and registry for future research.

  Recruiting at 2 sites Universitaire Ziekenhuizen KU Leuven · Leuven and 1 other locations · NCT07172243

• PET, MRI and ultrasound to track heart changes in ATTR cardiac amyloidosis

  This study will try PET, MRI and ultrasound scans in people with ATTR cardiac amyloidosis starting tafamidis to see if the new images explain why some patients keep getting worse.

  Recruiting University of Zurich · Zurich, Canton of Zurich · NCT07112066

• Registry for patients with systemic amyloidosis

  This study is collecting information from patients with systemic amyloidosis to better understand the disease and how different treatments work over time.

  Recruiting University Hospital, Essen · Essen, North Rhine-Westphalia · NCT06887283

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.