Ultrasound Therapy In Cardiac Amyloidosis
This is a prospective pilot clinical study of subjects with cardiac amyloidosis and control subjects without amyloidosis where we plan to evaluate changes in myocardial blood flow, systolic and diastolic function before and after sonotherapy.
Conditions:
🦠 Amyloidosis Cardiac
🗓️ Study Start (Actual) 31 August 2023
🗓️ Primary Completion (Estimated) 30 April 2026
✅ Study Completion (Estimated) 30 April 2027
👥 Enrollment (Estimated) 70
🔬 Study Type INTERVENTIONAL
📊 Phase PHASE4
Locations:
📍 Boston, Massachusetts, United States

📋 Eligibility Criteria

Description

    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
Ages Eligible for Study: 18 Years to 90 Years (ADULT, OLDER_ADULT)
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: Yes

🗓️ Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

  • First Submitted 3 December 2020
  • First Submitted that Met QC Criteria 8 December 2020
  • First Posted 14 December 2020

Study Record Updates

  • Last Update Submitted that Met QC Criteria 5 March 2024
  • Last Update Posted 6 March 2024
  • Last Verified March 2024