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ClinicalTrials.gov public records Last synced Oct 4, 2026, 5:06 PM EDT

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Showing 1–24 of 39 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Familial Transthyretin Cardiac Amyloidosis Clear all
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 4, 2020 · Synced Oct 4, 2026, 5:06 PM 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 4, 2026, 5:06 PM 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 12, 2026 · Synced Oct 4, 2026, 5:06 PM 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 4, 2026, 5:06 PM EDT
Active, not recruiting Phase 2 Interventional

Open-Label Study of AG10 in Patients with Cardiomyopathy

NCT03536767
View directory record Official record
Conditions
Amyloid Cardiomyopathy
Interventions
AG10
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 90 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
9
States / cities
Palo Alto, California • San Francisco, California • New Haven, Connecticut + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 13, 2025 · Synced Oct 4, 2026, 5:06 PM 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 12, 2026 · Synced Oct 4, 2026, 5:06 PM EDT
Active, not recruiting No phase listed Observational

An Observational Study Using Artificial Intelligence (AI) Algorithms on Electrocardiography (ECG), Point-of-care Ultrasound (POCUS), and Transthoracic Echocardiophy (TTE) to Estimate the Under-diagnosis of Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Across a Diverse Range of US Health Systems.

NCT07062848
View directory record Official record
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
AI Toolkit for ATTR-CM Diagnosis
Diagnostic Test
Lead sponsor
Yale University
Other
Eligibility
50 Years to 95 Years
Enrollment
1,500,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
12
States / cities
San Francisco, California • New Haven, Connecticut • Chicago, Illinois + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 4, 2026, 5:06 PM 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 4, 2026, 5:06 PM EDT
Not listed No phase listed Observational

Exercise Effect on Transthyretin Stability

NCT04986826
View directory record Official record
Conditions
Amyloidosis Transthyretin, Athlete Heart
Interventions
Not listed
Lead sponsor
Oregon Health and Science University
Other
Eligibility
18 Years and older
Enrollment
24 participants
Timeline
2021
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Aug 3, 2021 · Synced Oct 4, 2026, 5:06 PM 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 4, 2026, 5:06 PM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy

NCT01994889
View directory record Official record
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
Tafamidis, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 90 Years
Enrollment
441 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
50
States / cities
Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 24 more
Source: ClinicalTrials.gov public record
Updated Apr 24, 2019 · Synced Oct 4, 2026, 5:06 PM 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 Sep 15, 2026 · Synced Oct 4, 2026, 5:06 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Analysis of Lumbar Spine Stenosis Specimens for Identification of Transthyretin Cardiac Amyloidosis

NCT06034405
View directory record Official record
Conditions
Lumbar Spinal Stenosis, Cardiac Amyloidosis, ATTR Amyloidosis Wild Type, ATTR Gene Mutation, ATTRV122I Amyloidosis, Cardiomyopathy, Hypertrophic
Interventions
Tc99-PYP or Tc99-HDMP Scan
Diagnostic Test
Lead sponsor
Columbia University
Other
Eligibility
50 Years and older
Enrollment
1,663 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2028
U.S. locations
6
States / cities
Beverly Hills, California • Boston, Massachusetts • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Oct 4, 2026, 5:06 PM EDT
Not yet recruiting No phase listed Observational

PET Imaging Study Using Evuzamitide to Detect Cardiac Amyloidosis in Patients With Inconclusive Nuclear Scans and Elevated TAD1 Levels

NCT07538518
View directory record Official record
Conditions
Amyloidogenic Transthyretin (ATTR) Amyloidosis, Cardiac Amyloidosis
Interventions
Evuzamitide
Drug
Lead sponsor
Lorena Saelices
Other
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
Boston, Massachusetts • New York, New York • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 4, 2026, 5:06 PM EDT
Completed No phase listed Observational Results available

Screening for Cardiac Amyloidosis With Nuclear Imaging for Minority Populations

NCT03812172
View directory record Official record
Conditions
Amyloid Cardiomyopathy, Transthyretin-Related
Interventions
99mTc-PYP or 99m Tc-HDP
Drug
Lead sponsor
Mathew S. Maurer, MD
Other
Eligibility
60 Years and older
Enrollment
646 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
4
States / cities
New Haven, Connecticut • Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 19, 2025 · Synced Oct 4, 2026, 5:06 PM EDT
Recruiting Not applicable Interventional

Exercise Training in Transthyretin Cardiac Amyloidosis

NCT05797857
View directory record Official record
Conditions
Amyloid Cardiomyopathy, Transthyretin Cardiac Amyloidosis
Interventions
Exercise Training
Other
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
60 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 18, 2025 · Synced Oct 4, 2026, 5:06 PM EDT
Terminated Phase 4 Interventional Results available

A Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Participants With hATTR-PN

NCT04306510
View directory record Official record
Conditions
Hereditary Transthyretin Amyloidosis With Polyneuropthy
Interventions
TEGSEDI
Drug
Lead sponsor
Akcea Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Rosedale, New York
Source: ClinicalTrials.gov public record
Updated Apr 10, 2025 · Synced Oct 4, 2026, 5:06 PM EDT
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 7, 2026 · Synced Oct 4, 2026, 5:06 PM EDT
Recruiting No phase listed Observational

Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

NCT05040373
View directory record Official record
Conditions
Hereditary Transthyretin-mediated (hATTR) Amyloidosis, Polyneuropathy
Interventions
Not listed
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
Female only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 4, 2026, 5:06 PM 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 4, 2026, 5:06 PM EDT
Not listed No phase listed Observational

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

NCT06539169
View directory record Official record
Conditions
Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
Interventions
Not listed
Lead sponsor
xCures
Industry
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Los Altos, California
Source: ClinicalTrials.gov public record
Updated Nov 14, 2024 · Synced Oct 4, 2026, 5:06 PM 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 4, 2026, 5:06 PM 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 4, 2026, 5:06 PM EDT
Completed Phase 3 Interventional Results available

ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

NCT02319005
View directory record Official record
Conditions
Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related, Familial Transthyretin Cardiac Amyloidosis
Interventions
Revusiran (ALN-TTRSC), Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 90 Years
Enrollment
206 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
41
States / cities
Bakersfield, California • Beverly Hills, California • La Mesa, California + 30 more
Source: ClinicalTrials.gov public record
Updated Jul 18, 2018 · Synced Oct 4, 2026, 5:06 PM EDT