NCT02641145

Brief Summary

Cardiac amyloidosis is a major cause of early treatment-related death and poor overall survival in individuals with systemic light chain amyloidosis. This project will develop a novel approach to visualize cardiac amyloid deposits using advanced imaging methods. The long-term goal of this work is to identify the mechanisms of cardiac dysfunction, in order to guide the development of novel life-saving treatments.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
171

participants targeted

Target at P75+ for not_applicable

Timeline
32mo left

Started Apr 2016

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress79%
Apr 2016Dec 2028

First Submitted

Initial submission to the registry

December 22, 2015

Completed
7 days until next milestone

First Posted

Study publicly available on registry

December 29, 2015

Completed
3 months until next milestone

Study Start

First participant enrolled

April 1, 2016

Completed
11.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2027

Expected
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2028

Last Updated

November 14, 2025

Status Verified

November 1, 2025

Enrollment Period

11.8 years

First QC Date

December 22, 2015

Last Update Submit

November 13, 2025

Conditions

Keywords

amyloidosisF-18 florbetapirPositron emission tomographycardiac magnetic resonance imaging

Outcome Measures

Primary Outcomes (4)

  • Change in F-18 florbetapir myocardial retention index from baseline to 6 months and 12 months

    quantitative measure of F-18 florbetapir uptake by the heart muscle

    Baseline, 6 and 12 months

  • Change in Serum oxidative stress markers from baseline to 6 months and 12 months

    serum F-2 isoprostane and peroxynitrite levels

    Baseline, 6 and 12 months

  • Change in Myocardial oxidative metabolism markers from baseline to 6 months

    K mono and coronary flow reserve obtained by C-11 acetate PET/CT at rest and stress

    Baseline and 6 months

  • Change in Magnetic resonance imaging markers from baseline to 6 months and 12 months

    Extracellular volume index, T-1 mapping, late gadolinium enhancement, global strain, left ventricular mass

    Baseline, 6 and 12 months

Secondary Outcomes (3)

  • Change in Myocardial energy efficiency from baseline to 6 months

    Baseline and 6 months

  • Light Chain Toxicity

    Baseline

  • Understand the role of gut microbiota and heavy metals in the pathogenesis of AL Amyloidosis

    Baseline

Study Arms (5)

Active AL cardiac amyloidosis

EXPERIMENTAL

75 individuals with light chain systemic amyloidosis with active plasma cell dyscrasia and cardiac involvement will undergo a research F-18 florbetapir PET, C-11 acetate PET, and MRI of blood of the heart, as well as the heavy metal analysis of the blood at baseline, 6 months and 12 months after initiation of chemotherapy. 25 of these individuals will also undergo a N-13 ammonia PET scan of the heart following supine bicycle stress at baseline and at 6 months after initiation of chemotherapy.

Radiation: F-18 florbetapir/C-11 acetate PETDevice: MRIRadiation: N-13 ammonia PET

Remission AL cardiac amyloidosis

ACTIVE COMPARATOR

25 individuals with light chain systemic amyloidosis with cardiac involvement and plasma cell dyscrasia in hematological remission (complete hematological remission or very good partial response-differential free light chain (dFLC)\<40 mg/dL for \> 1 year prior to enrollment) will undergo a research F-18 florbetapir PET, C-11 acetate PET, and MRI scan of the heart as well as a heavy metal analysis of the blood at baseline.

Radiation: F-18 florbetapir/C-11 acetate PETDevice: MRI

Active AL Pre-CMP

EXPERIMENTAL

36 individuals with light chain systemic amyloidosis with active plasma cell dyscrasia and without cardiac involvement will undergo a research F-18 florbetapir PET, C-11 acetate PET, and MRI of the heart, as well as a heavy metal analysis of the blood at baseline. At 6 months they will undergo a research MRI of the heart and at 12 months they will have a clinical follow up. Subjects with contraindications to Cardiac MRI or gadolinium contrast may still be eligible for study participation.

Radiation: F-18 florbetapir/C-11 acetate PETDevice: MRI

Multiple Myeloma Controls

NO INTERVENTION

25 individuals with diagnosis of multiple myeloma without concomitant amyloidosis by standard criteria will undergo urine and blood testing only.

Heart Failure

EXPERIMENTAL

10 individuals with diagnosis of heart failure without amyloidosis by standard criteria will undergo a research F-18 florbetapir PET, C-11 acetate PET, and MRI of the heart, as well as a heavy metal analysis of the blood at baseline..

Radiation: F-18 florbetapir/C-11 acetate PETDevice: MRI

Interventions

F-18 florbetapir PET scan, C-11 acetate PET scan

Active AL Pre-CMPActive AL cardiac amyloidosisHeart FailureRemission AL cardiac amyloidosis
MRIDEVICE

Cardiac MRI with gadolinium contrast.

Active AL Pre-CMPActive AL cardiac amyloidosisHeart FailureRemission AL cardiac amyloidosis

N-13 ammonia PET scan following supine bicycle stress.

Active AL cardiac amyloidosis

Eligibility Criteria

Age18 Years - 99 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Age \> 18 years
  • Diagnosis of light chain amyloidosis by standard criteria (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)
  • For subjects traveling from out of town referred for systemic AL therapy based on clinical evaluation and laboratory testing, but, pending biopsy results, study enrollment and procedures may begin before official confirmation of biopsy results. If biopsy is negative for AL amyloidosis, subject will be considered a screen failure. There will be no more than 10 subjects who fall under this screen failure for the duration of the study.
  • Subjects with localized amyloid deposition and non-systemic AL disease will be eligible for enrollment in group D.
  • Willing and able to provide consent

You may not qualify if:

  • Hemodynamic instability
  • Decompensated heart failure (unable to lie flat for 1 hour)
  • Concomitant non-ischemic non-amyloid heart disease (valvular heart disease or dilated cardiomyopathy)
  • Known obstructive epicardial coronary artery disease with stenosis \> 50% in any single territory
  • Severe claustrophobia despite use of sedatives
  • Presence of MRI contraindications such as metallic implants (pacemaker or ICD) at the time of study enrollment except for Control Heart Failure subjects. Control HF subjects with no devices, or, with strictly MR compatible devices will be eligible to undergo MRI.
  • Significant renal dysfunction with estimated glomerular filtration rate \< 30 ml/min/m2 within 14 days of each cardiac MRI study. Subjects who develop renal dysfunction over the course of the study, meeting criteria listed above, will be excluded from the cardiac MRI scan except for control HF subjects. These subjects with eGFR \< 30 ml/min/1.73 m2 will undergo MRI without gadolinium contrast.
  • Subjects on dialysis will be excluded
  • Pregnant state. For women in child bearing age, a urine pregnancy test will be performed prior to the PET and the cardiac MRI studies
  • Documented allergy to F-18 florbetapir, C-11 acetate or gadolinium.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Brigham and Womens' Hospital

Boston, Massachusetts, 02421, United States

RECRUITING

Related Publications (5)

  • Benz DC, Clerc OF, Cuddy SAM, Singh V, Kijewski MF, Bianchi G, Yee AJ, Ruberg FL, Jerosch-Herold M, Kwong RY, Di Carli MF, Liao R, Falk RH, Dorbala S. Changes in Myocardial Light Chain Amyloid Burden After Plasma Cell Therapy. JACC Cardiovasc Imaging. 2025 Dec;18(12):1363-1374. doi: 10.1016/j.jcmg.2025.07.017. Epub 2025 Sep 12.

  • Clerc OF, Cuddy SAM, Jerosch-Herold M, Benz DC, Katznelson E, Canseco Neri J, Taylor A, Kijewski MF, Bianchi G, Ruberg FL, Di Carli MF, Liao R, Kwong RY, Falk RH, Dorbala S. Myocardial Characteristics, Cardiac Structure, and Cardiac Function in Systemic Light-Chain Amyloidosis. JACC Cardiovasc Imaging. 2024 Nov;17(11):1271-1286. doi: 10.1016/j.jcmg.2024.05.004. Epub 2024 Jul 10.

  • Katznelson E, Jerosch-Herold M, Cuddy SAM, Clerc OF, Benz DC, Taylor A, Rao S, Kijewski MF, Liao R, Landau H, Yee AJ, Ruberg FL, Di Carli MF, Falk RH, Kwong RY, Dorbala S. Mechanisms of left ventricular systolic dysfunction in light chain amyloidosis: a multiparametric cardiac MRI study. Front Cardiovasc Med. 2024 May 30;11:1371810. doi: 10.3389/fcvm.2024.1371810. eCollection 2024.

  • Cuddy SAM, Jerosch-Herold M, Falk RH, Kijewski MF, Singh V, Ruberg FL, Sanchorawala V, Landau H, Maurer MS, Yee AJ, Bianchi G, Di Carli MF, Liao R, Kwong RY, Dorbala S. Myocardial Composition in Light-Chain Cardiac Amyloidosis More Than 1 Year After Successful Therapy. JACC Cardiovasc Imaging. 2022 Apr;15(4):594-603. doi: 10.1016/j.jcmg.2021.09.032. Epub 2021 Dec 15.

  • Cuddy SAM, Bravo PE, Falk RH, El-Sady S, Kijewski MF, Park MA, Ruberg FL, Sanchorawala V, Landau H, Yee AJ, Bianchi G, Di Carli MF, Cheng SC, Jerosch-Herold M, Kwong RY, Liao R, Dorbala S. Improved Quantification of Cardiac Amyloid Burden in Systemic Light Chain Amyloidosis: Redefining Early Disease? JACC Cardiovasc Imaging. 2020 Jun;13(6):1325-1336. doi: 10.1016/j.jcmg.2020.02.025. Epub 2020 May 13.

MeSH Terms

Conditions

Immunoglobulin Light-chain AmyloidosisCardiomyopathiesAmyloidosis

Condition Hierarchy (Ancestors)

Neoplasms, Plasma CellNeoplasms by Histologic TypeNeoplasmsProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic DiseasesLymphoproliferative DisordersImmunoproliferative DisordersImmune System DiseasesParaproteinemiasHeart DiseasesCardiovascular Diseases

Study Officials

  • Sharmila Dorbala, MD

    Brigham and Women's Hospital (AHA and NIH Studies)

    PRINCIPAL INVESTIGATOR
  • Rodney Falk, MD

    Brigham and Women's Hospital (NIH Study)

    PRINCIPAL INVESTIGATOR
  • Ronglih Liao, PhD

    Stanford School of Medicine (AHA Study)

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Sharmila Dorbala, MD, MPH

CONTACT

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
DIAGNOSTIC
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Director, Nuclear Cardiology

Study Record Dates

First Submitted

December 22, 2015

First Posted

December 29, 2015

Study Start

April 1, 2016

Primary Completion (Estimated)

December 31, 2027

Study Completion (Estimated)

December 31, 2028

Last Updated

November 14, 2025

Record last verified: 2025-11

Data Sharing

IPD Sharing
Will not share

We do not have a plan to share individual participant data with other researchers

Locations