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ClinicalTrials.gov public records Last synced Oct 2, 2026, 4:03 PM EDT

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Showing 1–17 of 17 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Transthyretin Amyloidosis With Polyneuropathy Clear all
Completed Phase 2 Interventional Results available

The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis

NCT00630864
View directory record Official record
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 18, 2013 · Synced Oct 2, 2026, 4:03 PM 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 2, 2026, 4:03 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 2, 2026, 4:03 PM 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 2, 2026, 4:03 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 2, 2026, 4:03 PM 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 2, 2026, 4:03 PM EDT
Completed Early Phase 1 Interventional

Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy

NCT05023889
View directory record Official record
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 May 22, 2026 · Synced Oct 2, 2026, 4:03 PM 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 2, 2026, 4:03 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis

NCT00409175
View directory record Official record
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 17, 2012 · Synced Oct 2, 2026, 4:03 PM 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 2, 2026, 4:03 PM EDT
Recruiting No phase listed Observational

Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis

NCT06465810
View directory record Official record
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
33
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 24 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Oct 2, 2026, 4:03 PM EDT
Completed Phase 3 Interventional Results available

The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)

NCT02510261
View directory record Official record
Conditions
Amyloidosis
Interventions
Patisiran
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
Enrollment
211 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
14
States / cities
La Mesa, California • Aurora, Colorado • Jacksonville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Dec 6, 2023 · Synced Oct 2, 2026, 4:03 PM EDT
Recruiting No phase listed Observational

ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)

NCT04561518
View directory record Official record
Conditions
Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis
Interventions
Not listed
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2033
U.S. locations
13
States / cities
La Jolla, California • Los Angeles, California • Jacksonville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 2, 2026, 4:03 PM 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 2, 2026, 4:03 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

NCT05071300
View directory record Official record
Conditions
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Interventions
Eplontersen
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
151 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
8
States / cities
Scottsdale, Arizona • Indianapolis, Indiana • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2025 · Synced Oct 2, 2026, 4:03 PM EDT
Withdrawn Phase 3 Interventional

Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy

NCT04418024
View directory record Official record
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 19, 2024 · Synced Oct 2, 2026, 4:03 PM EDT
Completed No phase listed Observational Results available

A Study to Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis

NCT05139680
View directory record Official record
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 19, 2024 · Synced Oct 2, 2026, 4:03 PM EDT