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ClinicalTrials.gov public records Last synced Oct 22, 2026, 5:09 AM EDT

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Showing 1–24 of 108 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: TTR Clear all
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 22, 2026, 5:09 AM EDT
Terminated No phase listed Observational

carDIo-ttranSSfOrm nucLEar Imaging Study

NCT05259072
View directory record Official record
Conditions
Amyloidosis, Transthyretin Amyloid Cardiomyopathy
Interventions
99m-technetium pyrophosphate scintigraphy
Diagnostic Test
Lead sponsor
Oregon Health and Science University
Other
Eligibility
60 Years to 90 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jan 29, 2025 · Synced Oct 22, 2026, 5:09 AM 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 22, 2026, 5:09 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 6, 2022 · Synced Oct 22, 2026, 5:09 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 17, 2019 · Synced Oct 22, 2026, 5:09 AM EDT
Completed Phase 2 Interventional

Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

NCT01981837
View directory record Official record
Conditions
TTR-mediated Amyloidosis
Interventions
ALN-TTRSC (revusiran) for subcutaneous administration
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 80 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
3
States / cities
Boston, Massachusetts • New York, New York • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 20, 2023 · Synced Oct 22, 2026, 5:09 AM EDT
Withdrawn Phase 2 Interventional

ION-682884 in Patients With TTR Amyloid Cardiomyopathy

NCT04843020
View directory record Official record
Conditions
Transthyretin Amyloid Cardiopathy
Interventions
ION 682884
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
65 Years to 85 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 20, 2026 · Synced Oct 22, 2026, 5:09 AM EDT
Recruiting Phase 3 Interventional

TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

NCT07052903
View directory record Official record
Conditions
Transthyretin Amyloidosis With Cardiomyopathy
Interventions
Nucresiran, Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
Enrollment
1,750 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
45
States / cities
La Jolla, California • Laguna Hills, California • Stanford, California + 38 more
Source: ClinicalTrials.gov public record
Updated Oct 20, 2026 · Synced Oct 22, 2026, 5:09 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 22, 2026, 5:09 AM EDT
Active, not recruiting Phase 2 Interventional

A Study to Evaluate the Efficacy and Safety of Concomitant Use of Eplontersen and ALXN2220 Compared With Eplontersen and Placebo for Adults Participants With ATTR-CM

NCT07608354
View directory record Official record
Conditions
Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Interventions
Eplontersen, ALXN2220, Placebo
Drug · Biological · Other
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 85 Years
Enrollment
326 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
24
States / cities
La Jolla, California • San Francisco, California • Stanford, California + 21 more
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Oct 22, 2026, 5:09 AM EDT
Completed Phase 3 Interventional Results available

NEURO-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 Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

NCT04136184
View directory record Official record
Conditions
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Interventions
Inotersen, Eplontersen
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 82 Years
Enrollment
168 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
12
States / cities
Scottsdale, Arizona • Jacksonville, Florida • Indianapolis, Indiana + 9 more
Source: ClinicalTrials.gov public record
Updated Dec 13, 2024 · Synced Oct 22, 2026, 5:09 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 22, 2026, 5:09 AM 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 22, 2026, 5:09 AM EDT
Completed No phase listed Observational

CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study

NCT06073574
View directory record Official record
Conditions
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
Interventions
Cardiac MRI
Diagnostic Test
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 90 Years
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
2
States / cities
Boston, Massachusetts • Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Oct 22, 2026, 5:09 AM EDT
Completed Phase 2 Interventional Results available

The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)

NCT01961921
View directory record Official record
Conditions
TTR-mediated Amyloidosis
Interventions
ALN-TTR02 (patisiran) administered by intravenous (IV) infusion
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 19, 2024 · Synced Oct 22, 2026, 5:09 AM EDT
Completed No phase listed Observational

Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy

NCT06393465
View directory record Official record
Conditions
Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
Interventions
Tafamidis
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 29, 2026 · Synced Oct 22, 2026, 5:09 AM EDT
Completed Phase 3 Interventional Results available

Safety And Efficacy Evaluation Of Fx-1006A In Subjects With Transthyretin Amyloidosis

NCT00925002
View directory record Official record
Conditions
ATTR-PN
Interventions
Tafamidis
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
93 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2020
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 29, 2021 · Synced Oct 22, 2026, 5:09 AM EDT
Completed Phase 2 Interventional Results available

Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis

NCT01617967
View directory record Official record
Conditions
TTR-mediated Amyloidosis
Interventions
Patisiran
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 19, 2024 · Synced Oct 22, 2026, 5:09 AM EDT
Recruiting Phase 3 Interventional

Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)

NCT07608432
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Duchenne Type, Muscular Dystrophy, Duchenne and Becker Types, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Genetic Disease, Inborn, Genetic Disease, X-Linked, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neuromuscular Diseases (NMD)
Interventions
Zeleciment Rostudirsen (DYNE-251), Placebo
Drug
Lead sponsor
Dyne Therapeutics
Industry
Eligibility
4 Years to 18 Years · Male only
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Hillsborough, North Carolina
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Oct 22, 2026, 5:09 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 22, 2026, 5:09 AM 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 22, 2026, 5:09 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 22, 2026, 5:09 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 22, 2026, 5:09 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 22, 2026, 5:09 AM EDT