Clinical trials: Transthyretin Amyloidosis With Polyneuropathy
Charcot-Marie-Tooth Neuropathy Type 1A, Hereditary Transthyretin Amyloid Neuropathy
Longitudinal Quantitative Neuromuscular MRI in Neuropathic Patients
The pathophysiological process common to neuropathies is fatty replacement of muscle tissue, more commonly known as intramuscular fat fraction (F.F).
- Conditions
- Charcot-Marie-Tooth Neuropathy Type 1A, Hereditary Transthyretin Amyloid Neuropathy, Acquired Demyelinating Neuropathy
- Trial name
- IMAGINERV (NCT06845644)
- Testing
- Quantitative neuromuscular MRI, Neuromuscular ultrasound
- Where
- France
- Volunteers
- 120 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Transthyretin Familial Amyloid Polyneuropathy
The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin
Tafamidis has been developed as an oral specific stabilizer of transthyretin tetramer.
- Trial name
- NCT01435655
- Testing
- tafamidis
- Where
- 2 sites in Japan
- Volunteers
- 10 people
- Sponsor
- Pfizer
Hereditary Transthyretin Amyloidosis, Carpal Tunnel Syndrome
Hereditary Transthyretin Amyloidosis Polyneuropathy in Patients With Carpal Tunnel Syndrome in Russia
This is a multicenter observational study consisting of retrospective and prospective phases. The retrospective phase will entail secondary data collection from electronic or paper medical records of patients who underwent…
- Conditions
- Hereditary Transthyretin Amyloidosis, Carpal Tunnel Syndrome, Polyneuropathy, Amyloidosis
- Trial name
- LOCUS (NCT06414746)
- Where
- 13 sites in Russia
- Volunteers
- 721 people
- Sponsor
- AstraZeneca
Amyloidosis Cardiac, Amyloid Polyneuropathy
Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM) in Patients With Idiopathic Peripheral Neuropathy
The goal of the present study is to determine the occurrence of wild-type and hereditary transthyretin amyloidosis cardiomyopathy among patients with the diagnosis of idiopathic peripheral neuropathy in the setting of a…
- Conditions
- Amyloidosis Cardiac, Amyloid Polyneuropathy, Amyloidosis, Heart Failure
- Trial name
- NCT04985734
- Testing
- Specific diagnostic work-up for ATTR-CM patients
- Where
- Belgium
- Volunteers
- 200 people
- Sponsor
- University Hospital, Antwerp
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy
See updated study design under NCT04882735. Phase 3 efficacy and safety of AG10 compared with placebo in subjects with symptomatic Transthyretin Amyloid Polyneuropathy (ATTR-PN)
- Trial name
- ATTRibute-PN (NCT04418024)
- Testing
- AG10
- Where
- United States
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Transthyretin Amyloidosis, ATTR-CM
Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis
The MaesTTRo study aims to enroll a global cohort of patients with transthyretin (ATTR) amyloidosis to longitudinally observe the natural course of the disease and describe real-world treatment patterns and outcomes.
- Conditions
- Transthyretin Amyloidosis, ATTR-CM, ATTRv-PN, ATTR
- Trial name
- MaesTTRo (NCT06465810)
- Testing
- Treatment of transthyretin (ATTR) amyloidosis in observational study setting
- Where
- 89 sites in United States, Canada and 4 more
- Volunteers
- 1,850 people
- Sponsor
- AstraZeneca
FAP, Familial Amyloid Polyneuropathy
Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)
This study evaluates the safety and tolerability of extended dosing with IONIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.
- Conditions
- FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin
- Trial name
- NCT02175004
- Testing
- Inotersen
- Where
- 22 sites in United States, Argentina and 7 more
- Volunteers
- 135 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Efficacy and Safety of Acoramidis (AG10) in Subjects with Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)
Phase 3 efficacy and safety of acoramidis in subjects with symptomatic Transthyretin Amyloid Polyneuropathy (ATTR-PN)
- Trial name
- NCT04882735
- Testing
- Acoramidis
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Transthyretin (TTR)-Mediated Amyloidosis, Familial Amyloidotic Polyneuropathy (FAP)
The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant
The purpose of this study was to evaluate the safety and effectiveness of revusiran (ALN-TTRSC) in adults with transthyretin-mediated amyloidosis (ATTR), whose disease has continued to worsen after liver transplantation.
- Conditions
- Transthyretin (TTR)-Mediated Amyloidosis, Familial Amyloidotic Polyneuropathy (FAP), ATTR Amyloidosis, Familial Amyloid Neuropathies
- Trial name
- NCT02595983
- Testing
- Revusiran
- Where
- 6 sites in France, Germany and 4 more
- Volunteers
- 12 people
- Sponsor
- Alnylam Pharmaceuticals
Polyneuropathy, Amyloidosis
Prevalence of Wild-type TTR Cardiac Amyloidosis in Patients With Polyneuropathy of Unknown Cause.
To investigate to what extent chronic axonal length-dependent polyneuropathy (CAP) and/or small-fiber neuropathy (SFN) is part of early non-cardiac manifestations of wild-type TTR cardiac amyloidosis (wtTTR-CA).
- Trial name
- NCT05950867
- Testing
- ECG + echocardiography, Answering questionnaires
- Where
- Belgium
- Volunteers
- 150 people
- Sponsor
- Universitair Ziekenhuis Brussel
Amyloidosis
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)
The purpose of this study is to evaluate the safety and efficacy of long-term dosing with ALN-TTR02 (patisiran) in participants with transthyretin (TTR) mediated amyloidosis (ATTR).
- Trial name
- NCT02510261
- Testing
- Patisiran
- Where
- 56 sites in United States, Argentina and 19 more
- Volunteers
- 211 people
- Sponsor
- Alnylam Pharmaceuticals
Familial Amyloid Polyneuropathy, ATTR-PN
An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy
This study is designed to determine the long-term safety and tolerability of Fx-1006A as well as the effects of Fx-1006A on clinical outcomes in patients with ATTR-PN.
- Trial name
- NCT00791492
- Testing
- Fx-1006A
- Where
- 11 sites in Argentina, Brazil and 4 more
- Volunteers
- 86 people
- Sponsor
- Pfizer
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)
This study will be conducted to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NTLA-2001 in participants with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and…
- Conditions
- Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis
- Trial name
- NCT04601051
- Testing
- NTLA-2001
- Where
- 4 sites in France, New Zealand and 2 more
- Volunteers
- 72 people
- Sponsor
- Intellia Therapeutics
TTR-mediated Amyloidosis, Amyloidosis, Hereditary
Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).
- Conditions
- TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies
- Trial name
- NCT02939820
- Testing
- patisiran (ALN-TTR02)
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001
This is a follow-up study of subjects who received NTLA-2001 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.
- Conditions
- Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis
- Trial name
- NCT05697861
- Where
- 4 sites in France, New Zealand and 2 more
- Volunteers
- 72 people
- Sponsor
- Intellia Therapeutics
Transthyretin-associated Amyloidosis With Polyneuropathy
The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis
This is an open-label, multicenter, international study designed to determine TTR stabilization as well as Fx-1006A safety and tolerability, and its effects on clinical outcomes in patients with non-V30M TTR amyloidosis.
- Trial name
- NCT00630864
- Testing
- Fx-1006A
- Where
- 4 sites in United States, France and 2 more
- Volunteers
- 21 people
- Sponsor
- Pfizer
Neuromuscular Disease, Neuromuscular Diseases (NMD)
A Phase 3 Study of NTLA-2001 in ATTRv-PN
This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.
- Conditions
- Neuromuscular Disease, Neuromuscular Diseases (NMD), Neurodegenerative Disease, Neurodegenerative Disease, Hereditary
- Trial name
- NCT06672237
- Testing
- nexiguran ziclumeran
- Where
- 14 sites in Argentina, Brazil and 4 more
- Volunteers
- 60 people
- Sponsor
- Intellia Therapeutics
Hereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed Phenotype
A Study to Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis
This study will examine the clinical effectiveness of Tafamidis in patients with Mixed Phenotype Hereditary Transthyretin Amyloidosis using data that already exist in patients' medical records.
- Trial name
- NCT05139680
- Testing
- tafamidis
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Pfizer
Hereditary Transthyretin Amyloidosis
hATTR Polyneuropathy in Russia
A multicenter observational retrosPective Registry of patIents with transthyretin aMyloid polynEuropathy (hATTR-PN) and chRonic idiopathic axonal polyneuropathy (CIAP) in the population of the Russian Federation (PRIMER) There…
- Trial name
- PRIMER (NCT06365593)
- Where
- 3 sites in Russia
- Volunteers
- 100 people
- Sponsor
- AstraZeneca
Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
The purpose of this study is to: Describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients Characterize the safety and effectiveness of patisiran and…
- Trial name
- ConTTRibute (NCT04561518)
- Where
- 40 sites in United States, Brazil and 10 more
- Volunteers
- 1,500 people
- Sponsor
- Alnylam Pharmaceuticals