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ClinicalTrials.gov public records Last synced Jun 11, 2026, 5:26 AM EDT

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Showing 1–24 of 45 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Hereditary Amyloidosis, Transthyretin-Related Clear all
Recruiting No phase listed Observational

Determining the Association of TTR Stabilizing Therapy With Circulating TTR Amyloid Aggregates Over Time in Patients With ATTR-CM: Longitudinal Biomarker Study

NCT07196839
View directory record Official record
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
Not listed
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
30 Years to 80 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 6, 2026 · Synced Jun 11, 2026, 5:26 AM EDT
Completed No phase listed Observational Results available

Transthyretin Amyloidosis Outcome Survey (THAOS)

NCT00628745
View directory record Official record
Conditions
Transthyretin Gene Mutations, Transthyretin Amyloidosis
Interventions
None. Observational Study.
Other
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
6,718 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2023
U.S. locations
47
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 28 more
Source: ClinicalTrials.gov public record
Updated Nov 21, 2024 · Synced Jun 11, 2026, 5:26 AM EDT
Active, not recruiting Phase 3 Interventional Results available

HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

NCT04153149
View directory record Official record
Conditions
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Interventions
Vutrisiran, Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
Enrollment
655 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
17
States / cities
La Mesa, California • Los Angeles, California • Stanford, California + 13 more
Source: ClinicalTrials.gov public record
Updated Jan 11, 2026 · Synced Jun 11, 2026, 5:26 AM EDT
Completed Phase 3 Interventional Results available

HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

NCT03759379
View directory record Official record
Conditions
Amyloidosis, Hereditary, Transthyretin Amyloidosis
Interventions
Patisiran, Vutrisiran
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
Enrollment
164 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
15
States / cities
La Mesa, California • Aurora, Colorado • Jacksonville, Florida + 12 more
Source: ClinicalTrials.gov public record
Updated Jan 11, 2026 · Synced Jun 11, 2026, 5:26 AM EDT
Completed No phase listed Observational

Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis

NCT03431896
View directory record Official record
Conditions
Amyloidosis, Amyloid, Amyloid Neuropathies, Familial, Amyloid Cardiomyopathy, Amyloid - Primary, Transthyretin Amyloidosis, AL Amyloidosis
Interventions
Not listed
Lead sponsor
The Cleveland Clinic
Other
Eligibility
Not listed
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 11, 2026 · Synced Jun 11, 2026, 5:26 AM EDT
Recruiting Phase 3 Interventional

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

NCT06128629
View directory record Official record
Conditions
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Interventions
NTLA-2001, Placebo
Biological · Drug
Lead sponsor
Intellia Therapeutics
Industry
Eligibility
18 Years to 90 Years
Enrollment
1,200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
38
States / cities
Tucson, Arizona • Los Angeles, California • Palo Alto, California + 29 more
Source: ClinicalTrials.gov public record
Updated Mar 26, 2026 · Synced Jun 11, 2026, 5:26 AM EDT
Active, not recruiting Phase 3 Interventional

Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants

NCT04988386
View directory record Official record
Conditions
Amyloid Cardiomyopathy, Transthyretin-Related
Interventions
Acoramidis (AG10)
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 90 Years
Enrollment
389 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
26
States / cities
Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 21 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2025 · Synced Jun 11, 2026, 5:26 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

NCT01737398
View directory record Official record
Conditions
FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis
Interventions
Inotersen, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 82 Years
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
9
States / cities
Orange, California • Indianapolis, Indiana • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 16, 2019 · Synced Jun 11, 2026, 5:26 AM EDT
Completed Phase 1 Interventional

Parametric Cardiac 18F-flutemetamol PET Imaging in ATTR Cardiomyopathy

NCT05374564
View directory record Official record
Conditions
Cardiomyopathies, Primary
Interventions
(18F)Flutemetamol
Drug
Lead sponsor
Yale University
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Aug 2, 2025 · Synced Jun 11, 2026, 5:26 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Single and Multiple Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of AG10

NCT03294707
View directory record Official record
Conditions
Amyloid Cardiomyopathy, Transthyretin-Related
Interventions
AG10 oral tablet, Placebo Oral Tablet
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 55 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Tempe, Arizona
Source: ClinicalTrials.gov public record
Updated Sep 18, 2024 · Synced Jun 11, 2026, 5:26 AM EDT
Completed No phase listed Observational

Screening for Systemic Amyloidosis Via the Ligamentum Flavum

NCT03923920
View directory record Official record
Conditions
Amyloid, Amyloidosis, Spinal Stenosis, Transthyretin Amyloidosis, Primary Amyloidosis of Light Chain Type
Interventions
Biopsy
Procedure
Lead sponsor
The Cleveland Clinic
Other
Eligibility
50 Years and older
Enrollment
200 participants
Timeline
2019 – 2021
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 21, 2021 · Synced Jun 11, 2026, 5:26 AM EDT
Completed Phase 1 Interventional

A Study to Characterize the Biodistribution of 124I-Labeled AT-03 in Patients With Systemic Amyloidosis

NCT05201911
View directory record Official record
Conditions
Amyloidosis; Systemic
Interventions
124I-AT03
Drug
Lead sponsor
Attralus, Inc.
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Knoxville, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 14, 2022 · Synced Jun 11, 2026, 5:26 AM EDT
Recruiting No phase listed Observational

Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis

NCT06465810
View directory record Official record
Conditions
Transthyretin Amyloidosis, ATTR-CM, ATTRv-PN, ATTR, ATTR-Mixed, hATTR
Interventions
Treatment of transthyretin (ATTR) amyloidosis in observational study setting
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years
Enrollment
1,850 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
30
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 22 more
Source: ClinicalTrials.gov public record
Updated May 25, 2026 · Synced Jun 11, 2026, 5:26 AM EDT
Completed Early Phase 1 Interventional

Prevalence of Transthyretin Cardiac Amyloidosis in Clinically Significant Aortic Stenosis

NCT04899180
View directory record Official record
Conditions
Aortic Stenosis, Transthyretin Amyloidosis, Transthyretin Amyloid Cardiomyopathy, Transthyretin Cardiac Amyloidosis
Interventions
99mTc-PYP
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
75 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 6, 2024 · Synced Jun 11, 2026, 5:26 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Early Detection of Neuropathy in ATTRv

NCT05311488
View directory record Official record
Conditions
Hereditary Amyloidosis, Transthyretin-Related
Interventions
neurofilament light chain, In-vivo Meissner Corpuscle imaging, Nerve conduction study
Diagnostic Test
Lead sponsor
University of Pennsylvania
Other
Eligibility
30 Years to 90 Years
Enrollment
47 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2026
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 11, 2026 · Synced Jun 11, 2026, 5:26 AM EDT
Completed No phase listed Observational Results available

A Study to Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis

NCT05139680
View directory record Official record
Conditions
Hereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed Phenotype
Interventions
tafamidis
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 18, 2024 · Synced Jun 11, 2026, 5:26 AM EDT
Completed Phase 3 Interventional Results available

APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

NCT03997383
View directory record Official record
Conditions
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Interventions
Placebo, Patisiran
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
Enrollment
360 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
19
States / cities
Los Angeles, California • Chicago, Illinois • Skokie, Illinois + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 19, 2026 · Synced Jun 11, 2026, 5:26 AM EDT
Completed Phase 2Phase 3 Interventional Results available

The Effect of Diflunisal on Familial Amyloidosis

NCT00294671
View directory record Official record
Conditions
Familial Amyloid Polyneuropathy, Familial Amyloidosis
Interventions
diflunisal, placebo
Drug · Other
Lead sponsor
Boston University
Other
Eligibility
18 Years to 75 Years
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
3
States / cities
Boston, Massachusetts • Rochester, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 16, 2017 · Synced Jun 11, 2026, 5:26 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Evuzamitide in PET/CT to Measure Potential Therapeutic Response in ATTR

NCT05635045
View directory record Official record
Conditions
Transthyretin Amyloidosis
Interventions
I 124-Evuzamitide
Drug
Lead sponsor
Columbia University
Other
Eligibility
50 Years to 105 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 17, 2025 · Synced Jun 11, 2026, 5:26 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis

NCT00409175
View directory record Official record
Conditions
Familial Amyloid Polyneuropathy
Interventions
Fx-1006A, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 75 Years
Enrollment
128 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 16, 2012 · Synced Jun 11, 2026, 5:26 AM EDT
Completed No phase listed Observational

A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

NCT04201418
View directory record Official record
Conditions
Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, Polyneuropathy
Interventions
Patisiran
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
27
States / cities
Los Angeles, California • San Diego, California • Aurora, Colorado + 22 more
Source: ClinicalTrials.gov public record
Updated Jun 5, 2022 · Synced Jun 11, 2026, 5:26 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy

NCT01855360
View directory record Official record
Conditions
Amyloidosis; Heart (Manifestation), Senile Cardiac Amyloidosis
Interventions
Tauroursodeoxycholic Acid and Doxycycline
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 3, 2020 · Synced Jun 11, 2026, 5:26 AM EDT
Active, not recruiting Phase 4 Interventional

Safety, Efficacy, and Pharmacokinetics of Tafamidis in Patients With Transthyretin-mediated Amyloidosis Post Orthotopic Heart Transplantation

NCT05489523
View directory record Official record
Conditions
Transthyretin Cardiac Amyloidosis
Interventions
Tafamidis 61 MG
Drug
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years to 90 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
4
States / cities
Beverly Hills, California • New York, New York • Cleveland, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Jun 11, 2026, 5:26 AM EDT
Recruiting No phase listed Observational

Cardiovascular Multimodality Imaging Study

NCT06610019
View directory record Official record
Conditions
Non-ischemic Cardiomyopathy, Cardiomyopathies, Hypertrophic Cardiomyopathy, Right Ventricular Arrhythmogenic Cardiomyopathy, Cardiac Amyloidosis, Anderson Fabry Disease, Sarcoidosis, Cancer Therapy-related Cardiac Dysfunction, Ventricular Arrythmia, Heart Failure
Interventions
Not listed
Lead sponsor
Montefiore Medical Center
Other
Eligibility
18 Years and older
Enrollment
5,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
1
States / cities
The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Feb 13, 2025 · Synced Jun 11, 2026, 5:26 AM EDT