Clinical trials: Familial Amyloid Polyneuropathies
Aortic Stenosis Symptomatic, Amyloidosis Cardiac
ATTR Amyloidosis in Elderly Patients With Aortic Stenosis
Severe aortic stenosis is defined with a mean transvalvular pressure gradient (MTPG) > 40mmHg and a calculated aortic valve area of < 1cm2.
- Trial name
- NCT04061213
- Testing
- Tc-99m-DPD scintigraphy
- Where
- Switzerland
- Volunteers
- 489 people
- Sponsor
- Insel Gruppe AG, University Hospital Bern
Amyloid, Cardiac Amyloidosis
[18F]FT8 PET Imaging in Immunoglobulin Light Chain Amyloidosis
[18F]FT8, a derivative of 1-(4-pyridyl)-4-piperazinyl arene ([18F]TPZA), exhibits high affinity for AL amyloid in myocardial tissue sections and shows no significant binding to transthyretin amyloid.
- Conditions
- Amyloid, Cardiac Amyloidosis, Amyloidosis, Healthy Subjects
- Trial name
- NCT07232459
- Testing
- [18F]FT8
- Where
- 3 sites in China
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Tianjin Medical University
Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP), Transthyretin Cardiomyopathy (TTR-CM)
Burden of Disease Study In Patients With Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) orTransthyretin Cardiomyopathy (TTR-CM) And Caregivers
This study is an online (web-based) or paper-based survey for patients with transthyretin familial amyloidosis polyneuropathy (TTR-FAP) and caregivers.
- Conditions
- Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP), Transthyretin Cardiomyopathy (TTR-CM), Familial Amyloid Cardiomyopathy, Senile Systemic Amyloidosis (SSA)
- Trial name
- NCT01604122
- Testing
- No drug
- Volunteers
- 1 people
- Sponsor
- Pfizer
Transthyretin Familial Amyloid Polyneuropathy
Vyndaqel Drug Use Investigation (Regulatory Post Marketing Commitment Plan)
The purpose of this study is to understand safety (e.g., occurrence of adverse drug reactions [ADRs]) and efficacy data on the long-term use of Vyndaqel Capsules (hereinafter referred to as Vyndaqel) in all patients who received…
- Trial name
- NCT02146378
- Testing
- Vyndaqel
- Volunteers
- 500 people
- Sponsor
- Pfizer
Cardiac Amyloidosis
Thalidomide/Dexamethasone Treatment And PET Evaluation In Organ Involvemenet of Cardiac Amyloidosis
To prove the organ-reversing potential of thalidomide for amyloidosis with cardiac involvement
- Trial name
- TaPiOCA (NCT02966522)
- Testing
- Thalidomide, Dexamethasone
- Where
- South Korea
- Volunteers
- 30 people
- Sponsor
- Seoul National University Hospital
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
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
Amyloid, Amyloid Cardiomyopathy
18F-florbetaben PET-CT to Non-invasively Diagnose Cardiac AL Amyloidosis
Amyloidoses are systemic or acquired disorders characterized by the deposition in the extracellular spaces of amyloid fibers formed by proteins codified by mutated genes or non-mutated but misfolded proteins.
- Trial name
- PETAL (NCT06048601)
- Testing
- 18F-florbetaben PET/CT
- Where
- Italy
- Volunteers
- 150 people
- Sponsor
- Fondazione Toscana Gabriele Monasterio
Cardiac Amyloidosis
Hybrid Florbetaben PET/MRI for Imaging of Cardiac Amyloidosis
Exploration of prognostic parameters in 18F-Florbetaben PET/MRI in patients with cardiac amyloidosis. The clinical endpoints are defined as occurence of major adverse cardiac events (MACE) in amyloidosis patients.
- Trial name
- NCT07154381
- Where
- Germany
- Volunteers
- 25 people
- Sponsor
- University Hospital, Essen
Amyloidosis, Amyloidosis Cardiac
NatiOnal Referral cenTEr Study of Transthyretin Amyloid Cardiomyopathy(ATTR) Patients on Tafamidis
All ATTRwt patients on tafamidis 61 mg treatment will be clinically evaluated before treatment initiation and subsequently every six months for the eligibility to continue tafamidis treatment, according to Italian Medicines…
- Conditions
- Amyloidosis, Amyloidosis Cardiac, ATTR Amyloidosis Wild Type
- Trial name
- NOTE-ATTR (NCT06251778)
- Where
- Italy
- Volunteers
- 200 people
- Sponsor
- Paolo Milani
Amyloidosis; Heart (Manifestation), Senile Cardiac Amyloidosis
Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy
The objective of the study is to determine whether the combination of the bile acid TUDCA, and doxycycline will slow the progression of familial and senile amyloidosis.
- Trial name
- NCT01855360
- Testing
- Tauroursodeoxycholic Acid and Doxycycline
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Brigham and Women's Hospital
Amyloidosis, Amyloid
Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis
This study measures circulating, misfolded ATTR oligomers in asymptomatic ATTRm amyloidosis genetic carriers longitudinally over five years.
- Conditions
- Amyloidosis, Amyloid, Amyloid Neuropathies, Familial, Amyloid Cardiomyopathy
- Trial name
- MED-hATTR (NCT03431896)
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- The Cleveland Clinic
Amyloid Cardiomyopathy, Transthyretin Amyloid Cardiomyopathy
Artificial Intelligence to Assist the Echocardiographic Identification of Transthyretin Cardiac Amyloidosis
The goal of this study is to develop an algorithm using artificial intelligence (AI) to assist identification of potential ATTR-CM cases using routine transthoracic echocardiography.
- Conditions
- Amyloid Cardiomyopathy, Transthyretin Amyloid Cardiomyopathy, Transthyretin Cardiac Amyloidosis
- Trial name
- AI-ATTR-ECHO (NCT06328075)
- Testing
- non interventional study
- Where
- France
- Volunteers
- 15,000 people
- Sponsor
- Algalarrondo Vincent
Amyloidosis Cardiac, Heart Failure
Cardiac Contractility Modulation Therapy in Amyloid Cardiomyopathy Patients With Heart Failure
The primary aim of this observational registry is to evaluate the efficacy of CCM in patients with heart failure with mid-range or reduced EF and diagnosis of TTR amyloidosis.
- Trial name
- AMY-CCM (NCT05167799)
- Testing
- Cardiac Contractility Modulation (CCM)
- Where
- Italy
- Volunteers
- 25 people
- Sponsor
- Ospedale C & G Mazzoni
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
FAP, Familial Amyloid Polyneuropathy
Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy
The purpose of this study is to evaluate the efficacy and safety of inotersen given for 65 weeks in participants with Familial Amyloid Polyneuropathy (FAP).
- Conditions
- FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin
- Trial name
- NCT01737398
- Testing
- Inotersen
- Where
- 24 sites in United States, Argentina and 8 more
- Volunteers
- 173 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
AL Amyloidosis (AL), Cardiac Amyloidosis
Multimodal Radiomics Model (18F-FAPI PET/CT + CMR) for AL Cardiac Amyloidosis Prognosis
1. Goal of the Study: The goal of this prospective observational study is to develop and validate a novel, non-invasive method for predicting the prognosis of patients with light-chain cardiac amyloidosis (AL-CA).
- Trial name
- AL-CA (NCT07103863)
- Where
- China
- Volunteers
- 49 people
- Sponsor
- Beijing Anzhen Hospital
Cardiac Amyloidosis
AI Echocardiographic Screening of Cardiac Amyloidosis
Recent advances in machine learning and image processing techniques have shown that machine learning models can identify features unrecognized by human experts and accurately assess common measurements made in clinical practice.
- Trial name
- NCT06664866
- Testing
- EchoNet-LVH Assessment
- Where
- 4 sites in United States
- Volunteers
- 500 people
- Sponsor
- Cedars-Sinai Medical Center
Supraventricular Arrhythmia, Left Atrial Abnormality
Left Atrial Strain and Supraventricular Arrhythmia Burden in Cardiac Light Chain Amyloidosis Following Chemotherapy
The purpose of this study to assess the longitudinal changes in left atrial strain and supraventricular arrhythmia burden after chemotherapeutic strategies in cardiac light chain amyloidosis.
- Conditions
- Supraventricular Arrhythmia, Left Atrial Abnormality, Amyloid Cardiomyopathy, Congestive Heart Failure
- Trial name
- NCT05448716
- Testing
- Left Atrial Strain Imaging, Electrocardiography
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Istanbul University - Cerrahpasa
Cardiac Amyloidosis
Cardiac Uptake of 18F Florbetapir in Patients Undergoing Chemotherapy
This is a pilot study designed to assess for relative change in cardiac 18F Florbetapir uptake in patients with AL cardiac amyloidosis after appropriate chemotherapy.
- Trial name
- NCT03333551
- Testing
- F18 Florbetapir (amyvid) cardiac PET/CT imaging
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Medical College of Wisconsin