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ClinicalTrials.gov public records Last synced Oct 8, 2026, 10:14 AM EDT

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Showing 1–24 of 40 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Transthyretin Amyloidosis (ATTR) With Cardiomyopathy Clear all
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 8, 2026, 10:14 AM 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 8, 2026, 10:14 AM EDT
No Longer Available No phase listed Expanded access

Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy

NCT05505838
View directory record Official record
Conditions
Transthyretin-mediated Amyloidosis With Cardiomyopathy, ATTR Amyloidosis With Cardiomyopathy
Interventions
Patisiran
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
U.S. locations
20
States / cities
Phoenix, Arizona • Los Angeles, California • San Diego, California + 17 more
Source: ClinicalTrials.gov public record
Updated Feb 16, 2024 · Synced Oct 8, 2026, 10:14 AM EDT
Recruiting No phase listed Observational

Long-term Safety of Eplontersen Treated aTTR Patients and in Liver Transplant and Severely Hepatic Impaired Subpopulations

NCT07654855
View directory record Official record
Conditions
Transthyretin Amyloidosis, Transthyretin Amyloidosis With Cardiomyopathy, Transthyretin Amyloidosis With Polyneuropathy
Interventions
None ( observational study )
Other
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years and older
Enrollment
320 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Oct 8, 2026, 10:14 AM EDT
Completed Phase 2 Interventional

A Research Study to Look at How a New Medicine Called NNC6019-0001 Works and How Safe it is for People Who Have Heart Disease Due to Transthyretin (TTR) Amyloidosis

NCT05442047
View directory record Official record
Conditions
Transthyretin Amyloid Cardiomyopathy (ATTR CM)
Interventions
NNC6019-0001, Placebo (NNC6019-0001)
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
18 Years to 85 Years
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
9
States / cities
Phoenix, Arizona • Beverly Hills, California • Stanford, California + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2026 · Synced Oct 8, 2026, 10:14 AM EDT
Enrolling by invitation Phase 3 Interventional

An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

NCT05667493
View directory record Official record
Conditions
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
Interventions
Eplontersen
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
1,400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
46
States / cities
Scottsdale, Arizona • Beverly Hills, California • La Jolla, California + 35 more
Source: ClinicalTrials.gov public record
Updated Apr 17, 2026 · Synced Oct 8, 2026, 10:14 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 4, 2020 · Synced Oct 8, 2026, 10:14 AM 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 8, 2026, 10:14 AM 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 8, 2026, 10:14 AM EDT
Enrolling by invitation No phase listed Observational

Prospective Evaluation of Artificial Intelligence-enabled Screening for Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

NCT07398950
View directory record Official record
Conditions
ATTR Amyloidosis With Cardiomyopathy, Heart Failure
Interventions
ATTR-CM AI-ECG and AI-Echo Model
Diagnostic Test
Lead sponsor
Yale University
Other
Eligibility
18 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Oct 8, 2026, 10:14 AM EDT
Active, not recruiting Phase 3 Interventional

Study of ALXN2220 Versus Placebo in Adults With ATTR-CM

NCT06183931
View directory record Official record
Conditions
Transthyretin Amyloid Cardiomyopathy
Interventions
ALXN2220, Placebo
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 90 Years
Enrollment
1,181 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
36
States / cities
Irvine, California • La Jolla, California • Palo Alto, California + 30 more
Source: ClinicalTrials.gov public record
Updated Aug 10, 2026 · Synced Oct 8, 2026, 10:14 AM 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 8, 2026, 10:14 AM EDT
Recruiting No phase listed Observational

DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting

NCT07358078
View directory record Official record
Conditions
Transthyretin Amyloidosis With Cardiomyopathy
Interventions
Not listed
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
11
States / cities
Tucson, Arizona • Beverly Hills, California • Naples, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 8, 2026, 10:14 AM EDT
Completed Phase 2 Interventional Results available

Study of AG10 in Amyloid Cardiomyopathy

NCT03458130
View directory record Official record
Conditions
Familial ATTR-CM (ATTRm-CM, or FAC), Wild-type ATTR-CM (ATTRwt-CM)
Interventions
AG10, Placebo Oral Tablet
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 90 Years
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
13
States / cities
Beverly Hills, California • Palo Alto, California • San Francisco, California + 9 more
Source: ClinicalTrials.gov public record
Updated Nov 16, 2022 · Synced Oct 8, 2026, 10:14 AM EDT
Completed Phase 3 Interventional Results available

Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy

NCT02791230
View directory record Official record
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
Tafamidis
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
1,733 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
41
States / cities
Birmingham, Alabama • Beverly Hills, California • La Jolla, California + 20 more
Source: ClinicalTrials.gov public record
Updated Feb 3, 2025 · Synced Oct 8, 2026, 10:14 AM EDT
Terminated Not applicable Interventional Results available

Global Prevalence of ATTR-CM in Participants With HFpEF

NCT04424914
View directory record Official record
Conditions
Transthyretin Amyloid Cardiomyopathy, Heart Failure With Preserved Ejection Fraction
Interventions
Scintigraphy
Diagnostic Test
Lead sponsor
Pfizer
Industry
Eligibility
60 Years and older
Enrollment
347 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
22
States / cities
Fairhope, Alabama • Huntsville, Alabama • Miami, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2024 · Synced Oct 8, 2026, 10:14 AM EDT
Enrolling by invitation Phase 3 Interventional

A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

NCT06679946
View directory record Official record
Conditions
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Interventions
Vutrisiran
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
700 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
24
States / cities
Phoenix, Arizona • Beverly Hills, California • La Jolla, California + 19 more
Source: ClinicalTrials.gov public record
Updated Feb 20, 2026 · Synced Oct 8, 2026, 10:14 AM EDT
Active, not recruiting Phase 2 Interventional

A Research Study to Look at Long-term Treatment With a Medicine Called NNC6019-0001 for People Who Have Heart Failure Due to Transthyretin Amyloidosis

NCT06260709
View directory record Official record
Conditions
Transthyretin Amyloid Cardiomyopathy (ATTR CM)
Interventions
NNC6019-0001
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
18 Years to 85 Years
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
8
States / cities
Phoenix, Arizona • Los Angeles, California • Stanford, California + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 30, 2026 · Synced Oct 8, 2026, 10:14 AM EDT
Not yet recruiting Phase 3 Interventional

An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of ALXN2220 in Adults With Transthyretin Amyloid Cardiomyopathy

NCT07789483
View directory record Official record
Conditions
Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Interventions
ALXN2220
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
1,180 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027 – 2030
U.S. locations
35
States / cities
Irvine, California • La Jolla, California • Palo Alto, California + 29 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2026 · Synced Oct 8, 2026, 10:14 AM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy

NCT03860935
View directory record Official record
Conditions
Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases
Interventions
acoramidis, Placebo Oral Tablet
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 90 Years
Enrollment
632 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
40
States / cities
Los Angeles, California • Santa Monica, California • Aurora, Colorado + 32 more
Source: ClinicalTrials.gov public record
Updated Jun 27, 2024 · Synced Oct 8, 2026, 10:14 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 22, 2024 · Synced Oct 8, 2026, 10:14 AM EDT
Completed Phase 2 Interventional Results available

A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

NCT02292186
View directory record Official record
Conditions
TTR-mediated Amyloidosis
Interventions
Revusiran (ALN-TTRSC)
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 90 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
3
States / cities
Boston, Massachusetts • New York, New York • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 9, 2020 · Synced Oct 8, 2026, 10:14 AM EDT
Completed Phase 3 Interventional

CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

NCT04136171
View directory record Official record
Conditions
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
Interventions
Eplontersen, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 90 Years
Enrollment
1,438 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
51
States / cities
Phoenix, Arizona • La Jolla, California • Los Angeles, California + 39 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 8, 2026, 10:14 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 20, 2026 · Synced Oct 8, 2026, 10:14 AM EDT