Clinical trials: Amyloid Neuropathies
Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)
A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.
- Trial name
- NCT02797847
- Testing
- ALN-TTRSC02, Sterile Normal Saline (0.9% NaCl)
- Where
- United Kingdom
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Cardiac Amyloidosis
Validation of Different Diagnostic Modalities in the Detection of Cardiac Amyloidosis Among Patients With "Red Flags"
The study aims to test and compare the diagnostic yield of different diagnostic modalities (speckle tracking echocardiography, CMR, serum and urine protein analysis and bone tracer scintigraphy) in the detection of cardiac…
- Trial name
- NCT06175858
- Testing
- CMR
- Volunteers
- 30 people
- Sponsor
- Assiut University
Transthyretin (TTR) Amyloid Cardiomyopathy
An Observational Study Using Artificial Intelligence (AI) Algorithms on Electrocardiography (ECG), Point-of-care Ultrasound (POCUS), and Transthoracic Echocardiophy (TTE) to Estimate the Under-diagnosis of Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Across a Diverse Range of US Health Systems.
This is a multi-center, observational study with the overall objective to examine the scale of under-diagnosis for transthyretin amyloid cardiomyopathy (ATTR-CM) across a broad range of diverse health systems in the US using a…
- Trial name
- TRACE Network (NCT07062848)
- Testing
- AI Toolkit for ATTR-CM Diagnosis
- Where
- 12 sites in United States
- Volunteers
- 1,500,000 people
- Sponsor
- Yale University
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
Amyloid Cardiomyopathy, Block, AV
Screening of ATTRwt in Patient With Advanced AV-Block Undergoing Pacemaker Implantation
The investigators will nationally investigate the prevalence of Transthyretin Amyloidosis wildtype (ATTRwt) in patients of ≥ 65 years with left ventricular hypertrophy who present with high degree atrioventricular block…
- Trial name
- ATTRAB (NCT05699044)
- Testing
- Amyloidosis screening (bloodtest, DPD-scintigraphy, potentially endomyocardial biopsy)
- Where
- Denmark
- Volunteers
- 170 people
- Sponsor
- Steen Hvitfeldt Poulsen
Transthyretin Amyloid Polyneuropathy (ATTR-PN)
The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients
This is a single-arm, open-label, multicenter study designed to evaluate the efficacy, safety, tolerability as well as pharmacodynamics of tafamidis meglumine in ATTR-PN participants in China.
- Trial name
- NCT04828993
- Testing
- tafamidis meglumine
- Where
- 9 sites in China
- Volunteers
- 15 people
- Sponsor
- Pfizer
Transthyretin Amyloidosis Polyneuropathy, Transthyrexin Amyloidosis Cardiomyopathy
A Phase I/IIa，Open-label, Single Ascending Dose and Dose-expansion Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of YOLT-201 in Patients With Transthyretin Amyloidosis Polyneuropathy (ATTR-PN) or Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
This study will be conducted to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of YOLT-201 in participants with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and…
- Trial name
- NCT06539208
- Testing
- YOLT-201
- Where
- 3 sites in China
- Volunteers
- 31 people
- Sponsor
- YolTech Therapeutics Co., Ltd
Transthyretin (TTR) Amyloid Cardiomyopathy
Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy
This Phase 3 study will investigate the efficacy, safety and tolerability of an oral daily dose of 20 mg or 80 mg tafamidis meglumine capsules compared to placebo in subjects with either transthyretin genetic variants or…
- Trial name
- ATTR-ACT (NCT01994889)
- Testing
- Tafamidis
- Where
- 80 sites in United States, Belgium and 11 more
- Volunteers
- 441 people
- Sponsor
- Pfizer
Cardiac Amyloidosis
International Cardiac Amyloidosis Registry
Cardiac amyloidosis is a relatively rare disease. However, with the newer imaging techniques that have become available to us in recent years, determining the diagnosis is becoming more common. There are several variants.
- Trial name
- ICAR (NCT06894290)
- Where
- 6 sites in Belgium, Italy and 3 more
- Volunteers
- 300 people
- Sponsor
- Leiden University Medical Center
Cardiac Amyloidosis, Spinal Stenosis
Prevalence of Cardiac Amyloidosis Among Patients With a History of Lumbar Spinal Stenosis.
The study investigates the prevalence of cardiac amyloidosis among patients with a history of lumbar spinal stenosis within the last ten years in the region of central Denmark.
- Trial name
- CASS-I (NCT05701410)
- Testing
- DPD scintigraphy
- Where
- Denmark
- Volunteers
- 200 people
- Sponsor
- Steen Hvitfeldt Poulsen
Aortic Valve Stenosis, Cardiac Amyloidosis
Targeted Testing for ATTR Among Aortic Stenosis Patients-Pilot
Our ultimate goal is to design a multi-center randomized trial to test the hypothesis that targeted testing for transthyretin cardiac amyloid (ATTR) will improve survival and health status among aortic stenosis patients who…
- Trial name
- NCT04363294
- Testing
- PYP scan
- Sponsor
- University of Texas Southwestern Medical Center
Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Biomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP)
International, multicenter, observational, longitudinal study to identify biomarker/s for the development of a new MS-based biomarker for the early and sensitive diagnosis of Transthyretin-Related Familial Amyloidotic…
- Conditions
- Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin Amyloid Cardiopathy
- Trial name
- BioTRAP (NCT02713880)
- Where
- 2 sites in Germany, India
- Sponsor
- CENTOGENE GmbH Rostock
Carpal Tunnel, Transthyretin Amyloidosis
Concordance for Transthyretin Amyloidosis Between Synovial Biopsy and Anterior Carpal Ligament Biopsy
Transthyretin amyloidosis (ATTR) is an underdiagnosed condition that can present early as carpal tunnel syndrome, sometimes preceding cardiac involvement by several years.
- Trial name
- ATTR2026 (NCT07644325)
- Testing
- Annular ligament biopsy
- Where
- France
- Volunteers
- 500 people
- Sponsor
- Clinique Saint Jean, France
Cardiac Amyloidosis
Identification of Prognostic Parameters in Patients With " Senile " Amyloid Cardiomyopathy
The aging of the population is a reality in our society, with a strong increase in the number of elderly patients hospitalized for heart failure in our institutions.
- Trial name
- EVAMYLOSE (NCT04225754)
- Where
- France
- Volunteers
- 142 people
- Sponsor
- University Hospital, Grenoble
Cardiac Amyloidosis
Assessment of 18F-Florbetaben Whole-body PET for the Detection of Cardiac and Extracardiac Sites of Amyloid Deposits
Although being classified as a rare disease, cardiac amyloidosis constitutes an increasing cause of heart failure, which is often overlooked and thus poorly managed.
- Trial name
- CAPRI (NCT03616496)
- Testing
- Neuraceq PET/CT imaging
- Where
- France
- Volunteers
- 67 people
- Sponsor
- Central Hospital, Nancy, France
Amyloid Cardiomyopathy, Transthyretin Cardiac Amyloidosis
Exercise Training in Transthyretin Cardiac Amyloidosis
Transthyretin cardiac amyloidosis causes debilitating heart failure in older adults. The proposed research will develop a personalized exercise training program to improve functional capacity in patients on optimal treatment for…
- Trial name
- NCT05797857
- Testing
- Exercise Training
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Brigham and Women's Hospital
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 Amyloidosis
The Effect of Tafamidis on Transthyretin Stabilization, Safety, Tolerability and Efficacy in Transthyretin Amyloid Polyneuropathy Patients
Transthyretin amyloid polyneuropathy (ATTR-PN) is a fatal illness resulting from autosomal dominantly inherited single-point mutations on the transthyretin gene.
- Trial name
- NCT04963985
- Testing
- Tafamidis Pill
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Peking University Third Hospital
Cardiac Amyloidosis
Diagnosis of Cardiac Amyloidosis With 99mTc-PYP; Comparison Between Planar Imaging, SPECT/CT and Cardiac-dedicated CZT Camera
1. Patients will undergo clinical and laboratory assessment, as well as imaging tests, at the discretion of the attending cardiologist and will be referred at their discretion to perform cardiac mapping using 99mTc-PYP. 2.
- Trial name
- NCT04146415
- Testing
- Spect scan with mTc99-PYP
- Volunteers
- 30 people
- Sponsor
- Tel-Aviv Sourasky Medical Center
Transthyretin (TTR) Amyloid Cardiomyopathy
Vyndaqel Capsules Special Investigation (ATTR-CM)
Secondary Data Collection : To confirm the safety and effectiveness profiles under the actual medical practice of Vyndaqel in Japan.
- Trial name
- NCT04108091
- Testing
- Treatment for TTR amyloidosis
- Where
- Japan
- Volunteers
- 1,214 people
- Sponsor
- Pfizer