Analysis of Lumbar Spine Stenosis Specimens for Identification of Transthyretin Cardiac Amyloidosis
NCT ID: NCT06034405
Last Updated: 2025-11-12
Study Results
Results pending
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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Recruitment Status
RECRUITING
Total Enrollment
1663 participants
Study Classification
OBSERVATIONAL
Study Start Date
2023-09-19
Study Completion Date
2028-05-31
Brief Summary
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Primary objective:
To identify older adults with transthyretin cardiac amyloidosis (ATTR-CA) early in the course of the illness, at a time when disease modifying therapies are most effective.
The specific aims of this epidemiologic investigation include:
1. To identify subjects with previous lumbar spinal stenosis (LSS) Surgery who have evidence of transthyretin (TTR) amyloid deposits in spinal specimens and could be at risk for ATTR cardiac amyloidosis.
2. To evaluate for ATTR-CA among those with localized TTR in the spinal tissue.
The study will also explore the following:
1. The prevalence of amyloid in lumbar spinal stenosis specimens by Congo Red staining.
2. The prevalence of TTR deposits among subjects with amyloid as determined by mass spectrometry.
3. Evaluation of a novel artificial intelligence technique for that can identify amyloid histologically with standard H\&E staining.
4. Difference in ATTR-CA prevalence between subjects with TTR and indeterminate amyloid deposits in subject's spine by myocardial uptake of technetium pyrophosphate scan (Tc99-PYP).
To identify older adults with transthyretin cardiac amyloidosis (ATTR-CA) early in the course of the illness, at a time when disease modifying therapies are most effective.
The specific aims of this epidemiologic investigation include:
1. To identify subjects with previous lumbar spinal stenosis (LSS) Surgery who have evidence of transthyretin (TTR) amyloid deposits in spinal specimens and could be at risk for ATTR cardiac amyloidosis.
2. To evaluate for ATTR-CA among those with localized TTR in the spinal tissue.
The study will also explore the following:
1. The prevalence of amyloid in lumbar spinal stenosis specimens by Congo Red staining.
2. The prevalence of TTR deposits among subjects with amyloid as determined by mass spectrometry.
3. Evaluation of a novel artificial intelligence technique for that can identify amyloid histologically with standard H\&E staining.
4. Difference in ATTR-CA prevalence between subjects with TTR and indeterminate amyloid deposits in subject's spine by myocardial uptake of technetium pyrophosphate scan (Tc99-PYP).
Detailed Description
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This is a multicenter, prospective cohort study aimed at facilitating identification of individuals with ATTR-CA. Investigators will identify subjects with previous LSS surgery who have evidence of TTR amyloid deposits in spinal specimens. Subjects with localized TTR in spinal tissue will be invited to an on-site visit and be evaluated for the presence of clinical manifestations of ATTR cardiac amyloidosis (ATTR-CA).
Subjects will undergo nuclear scintigraphy, TTR genetic test, a clinical evaluation, electrocardiogram, and echocardiogram. Quality of life questionnaires will also be administered. Subjects who have myocardial retention of the imaging radiotracer with a Perugini score equal to 2 (uptake equal to bone) or 3 (uptake greater than bone) and confirmation by SPECT or SPECT/CT that the uptake is myocardial in nature and have no evidence of monoclonal proteins will be diagnosed with ATTR-CA.
This study is to be conducted at five study sites: Boston Medical Center, Cedars Sinai Medical Center, Cleveland Clinic, Columbia University Irving Medical Center, and Tufts University Medical Center. The Mayo Clinic will serve a Pathology research core to analyze spine specimens.
Most of the subjects are expected to complete all the study procedures and questionnaires on a single day.
Up to 1663 patients will be consented in this study to have spinal specimens examined for the presence of amyloid by Congo Red at the Mayo Clinic core lab. From the pool of subjects with amyloid detected (which anticipate will be 33% or at least 544), the investigators expect to recruit 163 participants to undergo cardiac phenotyping (Tc99m and cardiac evaluation). Among the 163 participants who undergo cardiac phenotyping, 98 will have TTR as the precursor protein identified by mass spectrometry and 65 will have an indeterminant precursor protein by mass spectrometry.
Subjects will undergo nuclear scintigraphy, TTR genetic test, a clinical evaluation, electrocardiogram, and echocardiogram. Quality of life questionnaires will also be administered. Subjects who have myocardial retention of the imaging radiotracer with a Perugini score equal to 2 (uptake equal to bone) or 3 (uptake greater than bone) and confirmation by SPECT or SPECT/CT that the uptake is myocardial in nature and have no evidence of monoclonal proteins will be diagnosed with ATTR-CA.
This study is to be conducted at five study sites: Boston Medical Center, Cedars Sinai Medical Center, Cleveland Clinic, Columbia University Irving Medical Center, and Tufts University Medical Center. The Mayo Clinic will serve a Pathology research core to analyze spine specimens.
Most of the subjects are expected to complete all the study procedures and questionnaires on a single day.
Up to 1663 patients will be consented in this study to have spinal specimens examined for the presence of amyloid by Congo Red at the Mayo Clinic core lab. From the pool of subjects with amyloid detected (which anticipate will be 33% or at least 544), the investigators expect to recruit 163 participants to undergo cardiac phenotyping (Tc99m and cardiac evaluation). Among the 163 participants who undergo cardiac phenotyping, 98 will have TTR as the precursor protein identified by mass spectrometry and 65 will have an indeterminant precursor protein by mass spectrometry.
Conditions
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Lumbar Spinal Stenosis
Cardiac Amyloidosis
ATTR Amyloidosis Wild Type
ATTR Gene Mutation
ATTRV122I Amyloidosis
Cardiomyopathy, Hypertrophic
Study Design
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Observational Model Type
COHORT
Study Time Perspective
PROSPECTIVE
Study Groups
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ATTR Negative
Participants who have undergone previous lumbar spinal, and/or carpel tunnel surgery will provide medical record data and spinal specimen for analysis of presence of ATTR amyloid.
No interventions assigned to this group
ATTR Positive
Participants who have undergone previous lumbar spinal, and/or carpel tunnel surgery will provide medical record data, spinal specimen, prospective data from cardiac workup with confirmed ATTR amyloid in spinal specimen then undergo cardiac workup for cardiac phenotyping/genotyping.
Tc99-PYP or Tc99-HDMP Scan
Intervention Type
DIAGNOSTIC_TEST
Difference in ATTR-CA prevalence between subjects with TTR and indeterminate amyloid deposits in their spine by myocardial uptake of Tc99-PYP.
Interventions
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Tc99-PYP or Tc99-HDMP Scan
Difference in ATTR-CA prevalence between subjects with TTR and indeterminate amyloid deposits in their spine by myocardial uptake of Tc99-PYP.
Intervention Type
DIAGNOSTIC_TEST
Eligibility Criteria
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Inclusion Criteria
1. Clinically indicated spinal decompressive surgery within 20 years prior to enrollment.
2. Age ≥50 years at the time of the surgery.
3. Able to understand and sign the informed consent document after the nature of the study has been fully explained.
2. Age ≥50 years at the time of the surgery.
3. Able to understand and sign the informed consent document after the nature of the study has been fully explained.
Exclusion Criteria
The presence of any of the following excludes eligibility for enrollment in this study:
1. Confirmed primary amyloidosis (AL) or secondary amyloidosis (AA).
2. Known TTR amyloidosis.
3. Other reason that would make the subject inappropriate for entry into this study.
1. Confirmed primary amyloidosis (AL) or secondary amyloidosis (AA).
2. Known TTR amyloidosis.
3. Other reason that would make the subject inappropriate for entry into this study.
Minimum Eligible Age
50 Years
Eligible Sex
ALL
Accepts Healthy Volunteers
Yes
Sponsors
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National Institute on Aging (NIA)
NIH
Sponsor Role
collaborator
Columbia University
OTHER
Sponsor Role
lead
Responsible Party
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Mathew S. Maurer, MD
Arnold and Arlene Goldstein Professor of Cardiology (in Medicine, Dept of Medicine Cardiology)
Responsibility Role
PRINCIPAL_INVESTIGATOR
Principal Investigators
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Mathew S Maurer, MD
Role: PRINCIPAL_INVESTIGATOR
Columbia University
Locations
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Cedars-Sinai Medical Center
Beverly Hills, California, United States
Site Status
RECRUITING
Tufts Medical Center
Boston, Massachusetts, United States
Site Status
RECRUITING
Boston Medical Center
Boston, Massachusetts, United States
Site Status
RECRUITING
Mayo Clinic
Rochester, Minnesota, United States
Site Status
ACTIVE_NOT_RECRUITING
Columbia University Irving Medical Center
New York, New York, United States
Site Status
RECRUITING
Cleveland Clinic
Cleveland, Ohio, United States
Site Status
RECRUITING
Countries
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United States
Central Contacts
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Facility Contacts
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Shruti Shantharam
Role: primary
Christine Sumbi
Role: backup
Ayan Patel, MD
Role: primary
James Kryzanski, MD
Role: backup
Frederick Ruberg, MD
Role: primary
Denise Fine
Role: backup
Jeffeny DeLosSantos, MHA
Role: primary
Samantha L Guadalupe, MHA
Role: backup
Mazen Hanna, MD
Role: primary
Lauren Ives, RN
Role: backup
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Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
AAAU4704
Identifier Type: -
Identifier Source: org_study_id