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ClinicalTrials.gov public records Last synced May 22, 2026, 3:55 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 101 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
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 13, 2024 · Synced May 22, 2026, 3:55 AM EDT
Completed Phase 2 Interventional Results available
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 15, 2022 · Synced May 22, 2026, 3:55 AM EDT
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 13, 2019 · Synced May 22, 2026, 3:55 AM EDT
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 Dec 8, 2024 · Synced May 22, 2026, 3:55 AM EDT
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 May 22, 2026, 3:55 AM EDT
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 12, 2025 · Synced May 22, 2026, 3:55 AM EDT
Conditions
Transthyretin-associated Amyloidosis With Polyneuropathy
Interventions
Fx-1006A
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 75 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 17, 2013 · Synced May 22, 2026, 3:55 AM EDT
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 Jun 3, 2025 · Synced May 22, 2026, 3:55 AM EDT
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 17, 2023 · Synced May 22, 2026, 3:55 AM EDT
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
4
States / cities
Naples, Florida • Boston, Massachusetts • New Brunswick, New Jersey + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 23, 2026 · Synced May 22, 2026, 3:55 AM EDT
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 19, 2023 · Synced May 22, 2026, 3:55 AM EDT
Completed No phase listed Observational Results available
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 May 22, 2026, 3:55 AM EDT
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 18, 2024 · Synced May 22, 2026, 3:55 AM EDT
Conditions
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Interventions
AG10, Placebo
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 90 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Sep 18, 2024 · Synced May 22, 2026, 3:55 AM EDT
Conditions
Amyloid, Cardiac Amyloidosis, Amyloidosis Cardiac, Systemic Amyloidosis, AL Amyloidosis, Infiltrative Cardiomyopathy, Amyloid, ATTR Amyloidosis Wild Type
Interventions
Chest Wall Fat Tissue Collection
Procedure
Lead sponsor
Midwest Heart & Vascular Specialists
Other
Eligibility
40 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
1
States / cities
Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated Feb 19, 2024 · Synced May 22, 2026, 3:55 AM EDT
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 17, 2025 · Synced May 22, 2026, 3:55 AM EDT
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 28, 2021 · Synced May 22, 2026, 3:55 AM EDT
Conditions
Transthyretin Amyloidosis
Interventions
Computer algorithm for ATTR
Device
Lead sponsor
Yale University
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Sep 17, 2025 · Synced May 22, 2026, 3:55 AM EDT
Conditions
Polyneuropathies, Wild Type ATTR Amyloidosis, Wild-Type Transthyretin-Related (ATTR)Amyloidosis, Wild-Type Transthyretin Cardiac Amyloidosis, Transthyretin Amyloidosis
Interventions
patisiran
Drug
Lead sponsor
Austin Neuromuscular Center
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced May 22, 2026, 3:55 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available
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 May 22, 2026, 3:55 AM EDT
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 May 22, 2026, 3:55 AM EDT
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 12, 2024 · Synced May 22, 2026, 3:55 AM EDT
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 9, 2025 · Synced May 22, 2026, 3:55 AM EDT
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 12, 2026 · Synced May 22, 2026, 3:55 AM EDT