Clinical trials: Transthyretin Amyloidosis, open to volunteers
- Condition Transthyretin Amyloidosis
- Status Open to volunteers
- Funder type Industry (pharma, biotech, devices)
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
The purpose of this study is to obtain safety, efficacy, and pharmacodynamic data on the use of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy who continued on extended use of vutrisiran, or switched from…
- Trial name
- NCT06679946
- Testing
- Vutrisiran
- Where
- 87 sites in United States, Argentina and 15 more
- Volunteers
- 700 people
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin Amyloidosis With Cardiomyopathy
TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
The purpose of this study is to: Evaluate the efficacy of nucresiran compared to placebo on reducing all-cause mortality and cardiovascular (CV) events Evaluate the efficacy of nucresiran compared to placebo on additional…
- Trial name
- NCT07052903
- Testing
- Nucresiran, Sterile Normal Saline (0.9% NaCl)
- Where
- 266 sites in United States, Argentina and 34 more
- Volunteers
- 1,750 people
- Sponsor
- Alnylam Pharmaceuticals
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
Transthyretin Amyloidosis With Cardiomyopathy
DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting
The purpose of this study is to: Describe the clinical characteristics of adult patients with transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM) treated with vutrisiran in routine clinical care Describe treatment…
- Trial name
- DemonsTTRate (NCT07358078)
- Where
- 27 sites in United States, Germany and 1 more
- Volunteers
- 2,000 people
- Sponsor
- Alnylam Pharmaceuticals
Hereditary Transthyretin-mediated (hATTR) Amyloidosis, Polyneuropathy
Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal/neonatal/infant outcomes in women exposed to patisiran-LNP.
- Trial name
- NCT05040373
- Where
- 7 sites in United States, France and 5 more
- Volunteers
- 10 people
- Sponsor
- Alnylam Pharmaceuticals
Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy, hATTR-PN
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
The purpose of this study is to: Determine the efficacy of nucresiran in patients with hATTR-PN by evaluating the effect on neurologic impairment, quality of life, nutritional status, disability, and gait speed Demonstrate…
- Trial name
- TRITON-PN (NCT07223203)
- Testing
- Nucresiran, Vutrisiran
- Where
- 61 sites in United States, Argentina and 20 more
- Volunteers
- 125 people
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
A Study to Learn About the Use of Acoramidis in Patients With a Heart Condition Called Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in a Real-world Setting
Transthyretin Amyloid Cardiomyopathy (ATTR-CM) is a serious and life-threatening condition where a protein called transthyretin (TTR) misfolds and builds up as amyloid fibrils in the heart muscle.
- Trial name
- ACO-REAL (NCT07235462)
- Testing
- Acoramidis (356 mg film-coated tablets)
- Where
- 17 sites in Austria, Belgium and 15 more
- Volunteers
- 2,000 people
- Sponsor
- Bayer
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
Transthyretin Amyloidosis, Transthyretin Amyloidosis With Cardiomyopathy
Long-term Safety of Eplontersen Treated aTTR Patients and in Liver Transplant and Severely Hepatic Impaired Subpopulations
The aim of this observational cohort study is to characterise use of eplontersen in patients with prior liver transplant or with pre-existing severe hepatic impairment, as well as to assess long-term safety among all new users of…
- Conditions
- Transthyretin Amyloidosis, Transthyretin Amyloidosis With Cardiomyopathy, Transthyretin Amyloidosis With Polyneuropathy
- Trial name
- sTTRing (NCT07654855)
- Testing
- None ( observational study )
- Where
- 6 sites in United States, Canada and 4 more
- Volunteers
- 320 people
- Sponsor
- AstraZeneca
Transthyretin Amyloidosis
The Eplontersen Pregnancy and Lactation Outcomes Study
The overall objective of this DPSS is to describe the occurrence of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant associated with exposure to…
- Trial name
- EPPRO (NCT07205666)
- Where
- Germany
- Volunteers
- 10 people
- Sponsor
- AstraZeneca
Amyloidosis in Transthyretin (TTR), Amyloidosis, Familial
To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE)
The AG10-504 study is an open-label extension study of acoramidis in participants with newly diagnosed transthyretin amyloid cardiomyopathy (ATTR-CM) or both ATTR-CM and transthyretin amyloid polyneuropathy (ATTR-PN).
- Conditions
- Amyloidosis in Transthyretin (TTR), Amyloidosis, Familial, Amyloid Cardiomyopathy, Amyloid Cardiomyopathy, Transthyretin-Related
- Trial name
- NCT07116473
- Testing
- Acoramidis (AG10)
- Volunteers
- 250 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Amyloidosis, Amyloid Cardiomyopathy
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque-like substance that accumulates in different organs in the body and can cause…
- Conditions
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies
- Trial name
- NCT06563895
- Testing
- Acoramidis
- Where
- 104 sites in United States, Argentina and 22 more
- Volunteers
- 587 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
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 Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis
Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis
The study aims to evaluate the effect of acoramidis on cardiac functional and structural improvement in participants with ATTR-CM.
- Conditions
- Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis
- Trial name
- ASCEND-ATTR (NCT07695701)
- Testing
- Acoramidis (AG10) oral tablet
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Transthyretin Cardiac Amyloidosis
Multicenter Study for the Validation of an AI-based ECG Platform for Early Cardiac Amyloidosis Diagnosis
CONCERTO is a retrospective, observational, multicentric and single-arm study to perform an external validation of the cloud-based and AI-powered electrocardiogram (ECG) analysis platform, named Willem™, to detect Transthyretin…
- Trial name
- CONCERTO (NCT06978660)
- Where
- 8 sites in United States, France and 3 more
- Volunteers
- 2,200 people
- Sponsor
- Idoven 1903 S.L.
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.
- Trial name
- NCT05667493
- Testing
- Eplontersen
- Where
- 134 sites in United States, Argentina and 18 more
- Volunteers
- 1,400 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
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
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
- Trial name
- NCT06128629
- Testing
- NTLA-2001
- Where
- 132 sites in United States, Argentina and 24 more
- Volunteers
- 1,200 people
- Sponsor
- Intellia Therapeutics
Transthyretin-type Cardiac Amyloidosis
Real-Life Clinical Efficacy of Acoramidis in Participants With ATTR-CM and Association With Cardiac Biomarkers
The purpose of this study is to confirm that the treatment with acoramidis prevents the deterioration of the ATTR-CM disease progression index and that these indexes are surrogate markers of disease progression.
- Trial name
- ATTR-CM (NCT07306949)
- Testing
- Acoramidis
- Where
- 16 sites in Japan
- Volunteers
- 200 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF
US Benchmarking Clinical Study
The goal of this observational study is to measure shear wave velocity (SWV) in patients with non-reduced left ventricular ejection fraction (LVEF) heart failure and left ventricular hypertrophy, with or without transthyretin…
- Conditions
- HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF, ATTR-CM (Transthyretin Amyloid Cardiomyopathy), Left Ventricular Hypertrophy
- Trial name
- EMS-CT-1 (NCT07215715)
- Where
- 3 sites in United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- eMyosound SAS