Ultrasound Therapy In Cardiac Amyloidosis

NCT04667494 | Recruiting Phase 4 FDA Device

• 1 other identifier: 2020P003179
• Study Type: interventional
• Target: 70
• Locations: 1 country
• Sites: 1

Brief Summary

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

Outcome Measures

Primary Outcomes (3)

• Myocardial Blood Flow

  N-13 ammonia or Rubidium-82 PET based measure of myocardial blood flow in ml/gm/min

  1 day

• Systolic Function

  Global Longitudinal Strain by echocardiography

  1 day

• Diastolic Function

  Echo measured mitral e' velocity

  1 day

Study Arms (1)

Sonotherapy

EXPERIMENTAL

All participants will undergo sonotherapy

Combination Product: Sonotherapy

Interventions

Sonotherapy COMBINATION_PRODUCT

Use of high mechanical index ultrasound therapy with intravenous Definity contrast infusion

Sonotherapy

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Brigham and Women's Hospital: lead

Study Sites (1)

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

MeSH Terms

Conditions

Amyloid Neuropathies, Familial

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Amyloid Neuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Amyloidosis, Familial; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Amyloidosis; Proteostasis Deficiencies

Study Officials

• Sharmila Dorbala, MD

  Brigham and Women's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Sharmila Dorbala, MD

CONTACT

617-732-6290; sdorbala@bwh.harvard.edu

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Nuclear Cardiology

Model Details: Outcome measures will be evaluated before and after intervention with sonotherapy.

Study Record Dates

• First Submitted: December 3, 2020
• First Posted: December 14, 2020
• Study Start: August 31, 2023
• Primary Completion: April 30, 2026
• Study Completion (Estimated): April 30, 2027
• Last Updated: November 14, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)