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ClinicalTrials.gov public records Last synced Oct 9, 2026, 4:58 AM EDT

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Showing 25–47 of 47 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Hereditary Amyloidosis, Transthyretin-Related Clear all
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 20, 2026 · Synced Oct 9, 2026, 4:58 AM EDT
Recruiting Phase 4 Interventional

Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis

NCT07695701
View directory record Official record
Conditions
Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis
Interventions
Acoramidis (AG10) oral tablet
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 80 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 9, 2026, 4:58 AM EDT
Recruiting No phase listed Observational

Subclinical Transthyretin Cardiac Amyloidosis in V122I TTR Carriers

NCT05489549
View directory record Official record
Conditions
Amyloidosis, Hereditary, Amyloidosis Cardiac, Amyloidosis, Familial, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Transthyretin Gene Mutation
Interventions
Not listed
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
30 Years to 80 Years
Enrollment
500 participants
Timeline
2022 – 2027
U.S. locations
3
States / cities
New York, New York • Cleveland, Ohio • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 9, 2026, 4:58 AM EDT
Completed Phase 3 Interventional Results available

The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)

NCT02510261
View directory record Official record
Conditions
Amyloidosis
Interventions
Patisiran
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
Enrollment
211 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
14
States / cities
La Mesa, California • Aurora, Colorado • Jacksonville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Dec 6, 2023 · Synced Oct 9, 2026, 4:58 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 14, 2025 · Synced Oct 9, 2026, 4:58 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 19, 2024 · Synced Oct 9, 2026, 4:58 AM EDT
Recruiting Phase 3 Interventional

TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy

NCT07223203
View directory record Official record
Conditions
Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy, hATTR-PN
Interventions
Nucresiran, Vutrisiran
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
11
States / cities
Aurora, Colorado • Chicago, Illinois • Baltimore, Maryland + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 9, 2026, 4:58 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 17, 2012 · Synced Oct 9, 2026, 4:58 AM EDT
Completed Phase 3 Interventional Results available

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

NCT01960348
View directory record Official record
Conditions
TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related
Interventions
patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
13
States / cities
La Mesa, California • Orange, California • Denver, Colorado + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 22, 2024 · Synced Oct 9, 2026, 4:58 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 12, 2026 · Synced Oct 9, 2026, 4:58 AM EDT
Completed Phase 3 Interventional Results available

Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

NCT02175004
View directory record Official record
Conditions
FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis
Interventions
Inotersen
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
9
States / cities
Orange, California • Indianapolis, Indiana • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Nov 18, 2023 · Synced Oct 9, 2026, 4:58 AM EDT
Completed No phase listed Observational

Carpal Tunnel Syndrome and Amyloid Cardiomyopathy

NCT02792790
View directory record Official record
Conditions
Amyloidosis, Cardiomyopathy, Transthyretin Amyloidosis, Senile Systemic Amyloidosis, Carpal Tunnel Syndrome
Interventions
Not listed
Lead sponsor
The Cleveland Clinic
Other
Eligibility
50 Years and older
Enrollment
96 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 22, 2021 · Synced Oct 9, 2026, 4:58 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 19, 2024 · Synced Oct 9, 2026, 4:58 AM EDT
Recruiting Phase 2 Interventional

Characterizing Iodine-124 Evuzumitide (AT-01) in Systemic Amyloidosis

NCT05758493
View directory record Official record
Conditions
Amyloidosis, Transthyretin Amyloidosis
Interventions
124I-Evuzamitide
Diagnostic Test
Lead sponsor
Oregon Health and Science University
Other
Eligibility
40 Years to 90 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 9, 2026, 4:58 AM EDT
Completed Early Phase 1 Interventional

Short-term Effects of TOLCAPONE on Transthyretin Stability in Subjects With Leptomeningeal TTR Amyloidosis (ATTR)

NCT03591757
View directory record Official record
Conditions
Transthyretin Amyloidosis, Amyloidosis, Leptomeningeal, Transthyretin-Related
Interventions
Tolcapone
Drug
Lead sponsor
Boston University
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 14, 2019 · Synced Oct 9, 2026, 4:58 AM EDT
Recruiting Not applicable Interventional

Prevalence and Prediction of ATTR in Ambulatory Patients With HFpEF

NCT05577819
View directory record Official record
Conditions
Transthyretin Amyloidosis, Heart Failure, Heart Failure, Diastolic, Amyloidosis
Interventions
99mTc-pyrophosphate Scintigraphy
Diagnostic Test
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
65 Years and older
Enrollment
515 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Oct 9, 2026, 4:58 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
10,000 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 Aug 10, 2026 · Synced Oct 9, 2026, 4:58 AM EDT
Recruiting Phase 3 Interventional

Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

NCT06563895
View directory record Official record
Conditions
Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases, Polyneuropathies
Interventions
Acoramidis, Placebo oral tablet
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 75 Years
Enrollment
587 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
40
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 31 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Oct 9, 2026, 4:58 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 12, 2026 · Synced Oct 9, 2026, 4:58 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 7, 2024 · Synced Oct 9, 2026, 4:58 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 27, 2026 · Synced Oct 9, 2026, 4:58 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 3, 2025 · Synced Oct 9, 2026, 4:58 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 4, 2026 · Synced Oct 9, 2026, 4:58 AM EDT