RECRUITING

Ultrasound Therapy In Cardiac Amyloidosis

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

Ultrasound Therapy In Cardiac Amyloidosis

Quick Facts

Study Start:2023-08-31

Study Completion:2027-04-30

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT04667494

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 90 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:Yes

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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	* 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: * 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

Inclusion Criteria

Exclusion 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

* 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:
* 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

Contacts and Locations

Study Contact

Sharmila Dorbala, MD

CONTACT

617-732-6290

sdorbala@bwh.harvard.edu

Principal Investigator

Sharmila Dorbala, MD

PRINCIPAL_INVESTIGATOR

Brigham and Women's Hospital

Study Locations (Sites)

Brigham and Women's Hospital

Boston, Massachusetts, 02115

United States

Collaborators and Investigators

Sponsor: Brigham and Women's Hospital

Sharmila Dorbala, MD, PRINCIPAL_INVESTIGATOR, Brigham and Women's Hospital

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

Study Start Date2023-08-31

Study Completion Date2027-04-30

Study Record Updates

Study Start Date2023-08-31

Study Completion Date2027-04-30

Terms related to this study

Additional Relevant MeSH Terms

Amyloidosis Cardiac