Clinical trials: Hereditary Amyloidosis, Transthyretin-Related
Hereditary Amyloidosis, Transthyretin-Related, Asymptomatic Carrier State
Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathy
This is a single-center observational study evaluating the potential value of NfL as a biomarker for diagnosis, detection of disease onset, monitoring of disease progression, and treatment response in asymptomatic carriers of TTR…
- Trial name
- NCT06360289
- Testing
- Standard of Care
- Where
- France
- Volunteers
- 346 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Amyloid Neuropathies, Transthyretin Amyloidosis
Expanding the Biomarkers in Familial Amyloid Neuropathy: MRI and Motor Unit Estimation by Electrophysiological Study
Familial amyloid neuropathies (FAP) are hereditary disease due to a mutation of the tranthyretin gene (TTR). These neuropathies are severe and life frightening.
- Trial name
- NCT03588468
- Testing
- Electrophysiological biomarkers, MRI biomarkers
- Where
- France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Assistance Publique Hopitaux De Marseille
Hereditary Amyloidosis, Transthyretin-Related
Early Detection of Neuropathy in ATTRv
The purpose of the study is to evaluate and compare different tools that are used to detect evidence of peripheral neuropathy in patients with TTRv.
- Trial name
- EDONA (NCT05311488)
- Testing
- neurofilament light chain, In-vivo Meissner Corpuscle imaging, Nerve conduction study
- Where
- United States
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
Transthyretin Amyloidosis, Transthyretin Amyloid Cardiomyopathy
Phenotyping and Characterization of wtATTR-CM (TRACE 1)
Descriptive cross-sectional study on 100 consecutive ATTRwt-CM patients reflecting all NAC stages aiming primarily to investigate ATTRwt-CM patient's quality of life (QoL) measures and their relation to ATTRwt-CM severity.
- Conditions
- Transthyretin Amyloidosis, Transthyretin Amyloid Cardiomyopathy, Wild-Type Transthyretin-Related (ATTR)Amyloidosis, Quality of Life
- Trial name
- NCT06291805
- Where
- Denmark
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Steen Hvitfeldt Poulsen
Transthyretin Amyloidosis
Evuzamitide in PET/CT to Measure Potential Therapeutic Response in ATTR
This was a single center, prospective cohort study that is evaluating the ability of 124I-evuzamitide PET scanning to detect potential therapeutic changes in subjects under treatment for ATTR after one year had elapsed since…
- Trial name
- NCT05635045
- Testing
- I 124-Evuzamitide
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Columbia University
Hereditary Transthyretin Amyloidosis
Exploring Biomarkers in Hereditary Transthyretin Amyloidosis
Hereditary transthyretin amyloidosis (ATTRv, v for variant) is a severe and heterogeneous systemic condition due to mutations in the transthyretin (TTR) gene.
- Trial name
- ELBA (NCT05929209)
- Testing
- Assessment of disease biomarkers
- Where
- Italy
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Aortic Stenosis, Transthyretin Amyloidosis
Positron Emission Tomography / Magnetic Resonance Imaging in Aortic Stenosis
Aortic stenosis is the most common valve disease requiring surgery in the Western world. It is defined by progressive calcification and fibrosis of the valve leaflets and restricted valve opening.
- Trial name
- PASS (NCT03352089)
- Where
- United Kingdom
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- University of Edinburgh
Transthyretin Amyloidosis
New Biomarkers and Plasma Prothrombotic Potential in Cardiac Transthyretin Amyloidosis
The development of cardiac amyloidosis is caused by the deposition of misfolded, insoluble proteins in the extracellular matrix of tissues.
- Trial name
- NCT06345235
- Testing
- blood test
- Where
- Poland
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- John Paul II Hospital, Krakow
Transthyretin Amyloidosis
The Regional Scintigraphic DPD Uptake in Cardiac Transthyretin Amyloidosis.
Cardiac transthyretin (ATTR) amyloidosis is an infiltrative cardiomyopathy with an inexorably progressive clinical course and poor prognosis.
- Trial name
- AMYLOIDOZA (NCT05814380)
- Testing
- 99mTc-DPD scintigraphy, Collection of blood samples and echocardiography
- Where
- Poland
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Katarzyna Holcman
Amyloidosis, Hereditary, Transthyretin-Related, Microbial Colonization
Analysis of the Instestinal Microbiome of Patients With Transthyretin Amyloidosis
Amyloidosis is a serious systemic disease. Cardiac involvement has a great impact on prognosis and can occur in its three main forms: acquired monoclonal light chain, hereditary transthyretinal and senile form.
- Trial name
- AMIAT (NCT05196594)
- Testing
- collection of stools for genetic analysis
- Where
- Brazil
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo General Hospital
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