Ultrasound therapy for cardiac amyloidosis
Ultrasound Therapy In Cardiac Amyloidosis
This study is testing whether ultrasound therapy can improve heart blood flow and function in people with cardiac amyloidosis.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 70 (estimated) |
| Ages | 18 Years to 90 Years |
| Sex | All |
| Sponsor | Brigham and Women's Hospital Academic / other |
| Locations | 1 site (Boston, Massachusetts) |
| Trial ID | NCT04667494 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates include adults over 18 years with a confirmed diagnosis of light chain or transthyretin cardiac amyloidosis in hematological remission.
Not a fit: Patients without cardiac amyloidosis or those who do not meet the specific diagnostic criteria will not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could improve cardiac function and quality of life for patients with cardiac amyloidosis.
How similar studies have performed: While this approach is novel in the context of cardiac amyloidosis, similar ultrasound therapies have shown promise in other cardiac conditions.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Boston, Massachusetts
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.