Clinical trials: Amyloidosis, Familial
Secondary (AA) Amyloidosis, Rheumatoid Arthritis
Study of the Safety and Efficacy of NC-503 in Secondary (AA) Amyloidosis
The main objective of this study is to evaluate the safety and efficacy of NC-503 compared to placebo in patients with secondary (AA) amyloidosis using a composite assessment of clinical improvement/worsening of both renal and…
- Conditions
- Secondary (AA) Amyloidosis, Rheumatoid Arthritis, Nephrotic Syndrome, Familial Mediterranean Syndrome
- Trial name
- NCT00035334
- Testing
- NC-503 (Anti-amyloidotic (AA) Agent)
- Where
- 26 sites in United States, Finland and 10 more
- Volunteers
- 150 people
- Sponsor
- Bellus Health Inc. - a GSK company
Amyloidosis Transthyretin, Athlete Heart
Exercise Effect on Transthyretin Stability
The goal of this study to investigate if exercise predisposes to transthyretin instability. The investigators will evaluate the effect of exercise on transthyretin biochemistry.
- Trial name
- NCT04986826
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Oregon Health and Science University
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
Cardiac Amyloidosis, Heart Diseases
N-of-1 for Beta-Blockers in Cardiac Amyloidosis
The study team will generate preliminary data on whether patients with cardiac amyloidosis feel better when their beta-blocker is stopped. To achieve this objective, 20 N-of-1 trials (on vs.
- Conditions
- Cardiac Amyloidosis, Heart Diseases, TTR Cardiac Amyloidosis
- Trial name
- NCT05019027
- Testing
- Beta blocker
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Weill Medical College of Cornell University
Heart Failure, Amyloid Cardiomyopathy
EOSS-ATTR Study (eHealth Based Operative Support System in ATTR-CM)
Unicenter, proof-of-concept, prospective, randomised, controlled, open-label and blinded end-point adjudication trial to assess the effect on patient-reported outcomes measures (PROMs), patient-reported experience measures…
- Conditions
- Heart Failure, Amyloid Cardiomyopathy, ATTR Amyloidosis Wild Type, ATTR Gene Mutation
- Trial name
- eOSS-ATTR (NCT06499064)
- Testing
- mHealth
- Volunteers
- 50 people
- Sponsor
- Hospital Universitari de Bellvitge
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
Amyloidosis
24 Month Open Label Study of the Tolerability and Efficacy of Inotersen in TTR Amyloid Cardiomyopathy Patients
Transthyretin is a protein produced in the liver that transports thyroid hormone and vitamin A. A single substitution of an amino acid in the structure of TTR can result in a relatively unstable protein, the breakdown products of…
- Trial name
- NCT03702829
- Testing
- Inotersen
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Brigham and Women's Hospital
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.
Amyloid Cardiomyopathy, Transthyretin-Related
Screening for Cardiac Amyloidosis With Nuclear Imaging for Minority Populations
In this study, the investigators recruited a cohort of elderly Black and Hispanic patients with heart failure to define the number of patients who have cardiac amyloidosis by utilizing highly sensitive heart imaging and blood…
- Trial name
- SCAN-MP (NCT03812172)
- Testing
- 99mTc-PYP or 99m Tc-HDP
- Where
- 4 sites in United States
- Volunteers
- 646 people
- Sponsor
- Mathew S. Maurer, MD
Cardiac Amyloidosis
Cardiac Amyloidosis Discovery Trial
This is a single center, diagnostic clinical trial in which the investigators aim to prospectively validate a deep learning model that identifies patients with features suggestive of cardiac amyloidosis, including transthyretin…
- Trial name
- NCT06469372
- Testing
- Cardiac amyloidosis deep learning model
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Pierre Elias
Amyloidosis
Safety and Effect of Doxycycline in Patients With Amyloidosis
The tetracycline antibiotic doxycycline disrupts A beta amyloid fibrils (AB) in Alzheimer's disease, transthyretin (ATTR) amyloid fibrils in familial amyloidotic polyneuropathy, and immunoglobulin light chain (AL) amyloid fibrils…
- Trial name
- NCT01677286
- Testing
- Doxycycline 100 mg po bid x 12 months
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Boston University
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
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
Familial Amyloid Polyneuropathy, Familial Amyloidosis
The Effect of Diflunisal on Familial Amyloidosis
The purpose of this study is to determine if diflunisal can prevent progressive lower leg nerve damage in patients with familial amyloidosis polyneuropathy.
- Trial name
- NCT00294671
- Testing
- diflunisal
- Where
- 8 sites in United States, Italy and 3 more
- Volunteers
- 130 people
- Sponsor
- Boston University
Alzheimer Disease, Family Members
Gamma Light and Sound Stimulation to Prevent Dementia in Cognitively Normal People at Risk for Alzheimer's Disease
Alzheimer's disease (AD) is characterized by significant memory loss, toxic protein deposits (amyloid and tau) in the brain, and changes in the gamma frequency band on EEG.
- Trial name
- NCT05776641
- Testing
- GENUS
- Where
- 2 sites in United States
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Massachusetts General 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
Hereditary Corneal Dystrophy, Corneal Abnormality
Safety and Efficacy Study of GEB-101 Injection in Subjects With TGFBI-related Corneal Dystrophy
The goal of this clinical trial is to evaluate the safety and efficacy of GEB-101 injection in subjects with TGFBI-related Corneal Dystrophy
- Conditions
- Hereditary Corneal Dystrophy, Corneal Abnormality, Corneal Dystrophies, Corneal Dystrophies, Hereditary
- Trial name
- CLARITY (NCT07750678)
- Testing
- GEB-101 Injection
- Where
- 2 sites in United States, China
- Volunteers
- 30 people
- Sponsor
- GenEditBio Limited
Amyloid Cardiomyopathy, Congestive Heart Failure
Longitudinal Changes in Left and Right Ventricular Global Strain After Chemotherapy in Cardiac Light Chain Amyloidosis
The purpose of this study to assess the longitudinal changes in left and right ventricular global strain after chemotherapeutic strategies in cardiac light chain amyloidosis.
- Conditions
- Amyloid Cardiomyopathy, Congestive Heart Failure, Chemotherapy Effect
- Trial name
- NCT05452850
- Testing
- Left and Right Ventricular Global Strain Imaging
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Istanbul University - Cerrahpasa
Amyloidosis
The Effect of Diflunisal on Familial Transthyretin Amyloidosis
An ongoing trial of diflunisal has been closed for enrollment, thus, patients suitable for the study can no longer participate or receive treatment by diflunisal; and patients, who have participated in the trial can not continue…
- Trial name
- DFNS01 (NCT01432587)
- Testing
- Diflunisal
- Where
- 3 sites in Sweden
- Volunteers
- 55 people
- Sponsor
- Umeå University