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ClinicalTrials.gov public records Last synced May 22, 2026, 4:16 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 38 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, 4:16 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, 4:16 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, 4:16 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, 4:16 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, 4:16 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, 4:16 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, 4:16 AM EDT
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 11, 2026 · Synced May 22, 2026, 4:16 AM EDT
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 Apr 19, 2026 · Synced May 22, 2026, 4:16 AM EDT
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 26, 2026 · Synced May 22, 2026, 4:16 AM EDT
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 2, 2025 · Synced May 22, 2026, 4:16 AM EDT
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 Apr 23, 2026 · Synced May 22, 2026, 4:16 AM EDT
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 17, 2018 · Synced May 22, 2026, 4:16 AM EDT
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
11
States / cities
San Francisco, California • New Haven, Connecticut • Detroit, Michigan + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 13, 2025 · Synced May 22, 2026, 4:16 AM EDT
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 11, 2026 · Synced May 22, 2026, 4:16 AM EDT
Conditions
Transthyretin Amyloidosis, ATTR-CM, ATTRv-PN, ATTR, ATTR-Mixed, hATTR
Interventions
Treatment of transthyretin (ATTR) amyloidosis in observational study setting
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years
Enrollment
1,850 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
29
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 21 more
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced May 22, 2026, 4:16 AM EDT
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 13, 2025 · Synced May 22, 2026, 4:16 AM EDT
Conditions
Familial Amyloidotic Cardiomyopathy (FAC)
Interventions
Not listed
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
1,010 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
43
States / cities
Little Rock, Arkansas • Bakersfield, California • La Mesa, California + 32 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2016 · Synced May 22, 2026, 4:16 AM EDT
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 18, 2025 · Synced May 22, 2026, 4:16 AM EDT
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 23, 2019 · Synced May 22, 2026, 4:16 AM EDT
Conditions
Amyloidosis
Interventions
Inotersen
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years to 85 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 19, 2026 · Synced May 22, 2026, 4:16 AM EDT
Completed Phase 2Phase 3 Interventional Results available
Conditions
Familial Amyloid Polyneuropathy, Familial Amyloidosis
Interventions
diflunisal, placebo
Drug · Other
Lead sponsor
Boston University
Other
Eligibility
18 Years to 75 Years
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
3
States / cities
Boston, Massachusetts • Rochester, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 16, 2017 · Synced May 22, 2026, 4:16 AM EDT
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 18, 2024 · Synced May 22, 2026, 4:16 AM EDT
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 2, 2021 · Synced May 22, 2026, 4:16 AM EDT