Amyloidosis CardiacPhase 4UTICANCT04667494
Ultrasound Therapy In Cardiac Amyloidosis
A Phase 4 study testing Sonotherapy in people with Amyloidosis Cardiac, run by Brigham and Women's Hospital at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 90 yearsHealthy volunteers can take part
- Conditions the study is for
- Amyloidosis Cardiac
- What is being tested?
- Sonotherapy
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Aug 2023to 30 Apr 2027
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 90 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Sharmila Dorbala, MBBS, MPH, 617-732-6290, sdorbala@partners.org
Sharmila Dorbala, MBBS, MPH
About this study
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.
Read the detailed description
70 subjects will be enrolled in this study in 3 study groups: A) 30 subjects with light chain cardiac amyloidosis B) 30 subjects with transthyretin cardiac amyloidosis and C) 10 subjects without amyloidosis. All subjects will undergo rest 2D echocardiography, rest N-13 ammonia or rubidium-82 perfusion PET, Definity infusion with high mechanical index (MI) imaging defined as sonotherapy in this study protocol.
- Conditions
- Amyloidosis Cardiac
- Being tested
- Sonotherapy
- Medical subject headings
- Amyloid Neuropathies, Familial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SonotherapyCombination product
Use of high mechanical index ultrasound therapy with intravenous Definity contrast infusion
| Study group | Role | What they receive |
|---|---|---|
| Sonotherapy | Experimental | All participants will undergo sonotherapy Receives: Combination Product: Sonotherapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Myocardial Blood Flow
N-13 ammonia or Rubidium-82 PET based measure of myocardial blood flow in ml/gm/min
Measured: 1 day
Systolic Function
Global Longitudinal Strain by echocardiography
Measured: 1 day
Diastolic Function
Echo measured mitral e' velocity
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Dec 2020
- Study start31 Aug 2023
- Primary completion30 Apr 2027 (estimated)
- Study completion30 Apr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyloidosis CardiacStudies of the same condition, open and under way.
- More trials run by Brigham and Women's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Outcome measures will be evaluated before and after intervention with sonotherapy.
- Masking
- None (open label)
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Brigham and Women's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Sharmila Dorbala, Brigham and Women's Hospital
- Investigators
- Sharmila Dorbala, MD, Principal investigator, Brigham and Women's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04667494
- Sponsor's protocol ID
- 2020P003179
- Acronym
- UTICA
- Status last verified
- Aug 2026
- First submitted
- 3 Dec 2020
- Last updated
- 21 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- No: There is no plan to share IRD
Study at a glance
From the registry record, last updated by the sponsor on 21 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ultrasound Therapy In Cardiac Amyloidosis
- ClinicalTrials.gov ID
- NCT04667494
- Acronym / sponsor ID
- UTICA / 2020P003179
- Conditions
- Amyloidosis Cardiac
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- Brigham and Women's HospitalUniversity, hospital or other
- Study start
- 31 Aug 2023 (actual)
- Primary completion
- 30 Apr 2027 (estimated)
- Study completion
- 30 Apr 2027 (estimated)
- First posted
- 14 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
70 subjects will be enrolled in this study in 3 study groups: A) 30 subjects with light chain cardiac amyloidosis B) 30 subjects with transthyretin cardiac amyloidosis and C) 10 subjects without amyloidosis. All subjects will undergo rest 2D echocardiography, rest N-13 ammonia or rubidium-82 perfusion PET, Definity infusion with high mechanical index (MI) imaging defined as sonotherapy in this study protocol.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Outcome measures will be evaluated before and after intervention with sonotherapy.
| Arm | Type | Receives |
|---|---|---|
| Sonotherapy | Experimental | Combination Product: SonotherapyAll participants will undergo sonotherapy |
Who may qualify
- Ages
- 18 to 90 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- 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)
- Diagnosis of transthyretin cardiac amyloidosis by standard criteria
Key exclusion criteria (23 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SonotherapyUse of high mechanical index ultrasound therapy with intravenous Definity contrast infusion | Combination product | Sonotherapy |
Primary outcome
Myocardial Blood Flow
Time frame: 1 day
N-13 ammonia or Rubidium-82 PET based measure of myocardial blood flow in ml/gm/min
Systolic Function
Time frame: 1 day
Global Longitudinal Strain by echocardiography
Diastolic Function
Time frame: 1 day
Echo measured mitral e' velocity
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Aug 2026.
- Individual participant dataSharing planned: No. There is no plan to share IRD
Related trials for Amyloidosis Cardiac
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EPIC-ATTR: A Study to Evaluate the Effect of Eplontersen on the Transthyretin Reduction and Long-term Safety in Chinese Subjects With Transthyretin Amyloid Cardiomyopathy
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Essen Amyloidosis Registry
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MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Sharmila Dorbala, MD principal investigator
Brigham and Women's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Sonotherapy in people with Amyloidosis Cardiac, run by Brigham and Women's Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 21 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sharmila Dorbala, MD (617-732-6290, sdorbala@bwh.harvard.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Myocardial Blood Flow (time frame: 1 day). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Aug 2026.