Clinical trials: Amyloid Neuropathies
Transthyretin (TTR) Amyloid Cardiomyopathy
Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy
Open label study to evaluate tafamidis for the treatment of transthyretin cardiomyopathy
- Trial name
- NCT02791230
- Testing
- Tafamidis
- Where
- 80 sites in United States, Argentina and 15 more
- Volunteers
- 1,733 people
- Sponsor
- Pfizer
Amyloid Cardiomyopathy
Open-Label Study of AG10 in Patients With Cardiomyopathy
This prospective, multicenter open-label study will evaluate the long-term safety, tolerability, PK and PD of acoramidis administered on a background of stable heart failure therapy.
- Trial name
- NCT03536767
- Testing
- Acoramidis
- Where
- 9 sites in United States
- Volunteers
- 47 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Amyloidosis, Amyloid Cardiomyopathy
Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy
Phase 3 efficacy and safety study to evaluate acoramidis (AG10) HCl 800 mg administered orally twice a day compared to placebo in subjects with symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM).
- Conditions
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies
- Trial name
- ATTRibute-CM (NCT03860935)
- Testing
- acoramidis
- Where
- 104 sites in United States, Australia and 16 more
- Volunteers
- 632 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
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
The Role of F-18 Florbetapir in the Early Detection of Cardiac Amyloidosis
The investigators postulate that F-18 florbetapir will show improved detection of cardiac amyloidosis over conventional non-invasive imaging techniques, particularly in early disease.
- Trial name
- NCT03040427
- Testing
- F-18 florbetapir
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- The Cleveland Clinic
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
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 (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
Amyloidosis
The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)
The purpose of this study is to evaluate the safety and efficacy of long-term dosing with ALN-TTR02 (patisiran) in participants with transthyretin (TTR) mediated amyloidosis (ATTR).
- Trial name
- NCT02510261
- Testing
- Patisiran
- Where
- 56 sites in United States, Argentina and 19 more
- Volunteers
- 211 people
- Sponsor
- Alnylam Pharmaceuticals
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
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
Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary
ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)
The purpose of this study was to evaluate the safety and efficacy of revusiran (ALN-TTRSC) in patients with transthyretin (TTR) mediated Familial Amyloidotic Cardiomyopathy.
- Conditions
- Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Amyloid Neuropathies
- Trial name
- NCT02319005
- Testing
- Revusiran (ALN-TTRSC), Sterile Normal Saline (0.9% NaCl)
- Where
- 61 sites in United States, Belgium and 7 more
- Volunteers
- 206 people
- Sponsor
- Alnylam Pharmaceuticals
Familial Amyloid Polyneuropathy, ATTR-PN
An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy
This study is designed to determine the long-term safety and tolerability of Fx-1006A as well as the effects of Fx-1006A on clinical outcomes in patients with ATTR-PN.
- Trial name
- NCT00791492
- Testing
- Fx-1006A
- Where
- 11 sites in Argentina, Brazil and 4 more
- Volunteers
- 86 people
- Sponsor
- Pfizer
Cardiac Amyloidosis
Imaging Cardiac Amyloidosis: A Pilot Study Using F-18 Florbetapir Positron Emission Tomography
The primary aim of this pilot study is to determine whether amyloid deposits in the heart can be measured non-invasively by F-18 florbetapir (Trade Name: Amyvid) positron emission tomography (PET) in 30 individuals with…
- Trial name
- NCT01683825
- Testing
- F-18 florbetapir PET
- Where
- United States
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- Brigham and Women's Hospital
Familial Amyloid Polyneuropathy
Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis
This study will examine whether Fx-1006A is effective in halting the progression of Familial Amyloid Polyneuropathy (FAP). Deposition of TTR amyloid is associated with a variety of human diseases.
- Trial name
- NCT00409175
- Testing
- Fx-1006A
- Where
- 10 sites in United States, Argentina and 7 more
- Volunteers
- 128 people
- Sponsor
- Pfizer
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.
TTR-mediated Amyloidosis, Amyloidosis, Hereditary
APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis
The purpose of this study is to evaluate the safety and efficacy of patisiran (ALN-TTR02) in patients with transthyretin (TTR) mediated amyloidosis.
- Conditions
- TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies
- Trial name
- NCT01960348
- Testing
- patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl)
- Where
- 52 sites in United States, Argentina and 19 more
- Volunteers
- 225 people
- Sponsor
- Alnylam Pharmaceuticals
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.
Transthyretin (TTR)-Mediated Amyloidosis, Familial Amyloidotic Polyneuropathy (FAP)
The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant
The purpose of this study was to evaluate the safety and effectiveness of revusiran (ALN-TTRSC) in adults with transthyretin-mediated amyloidosis (ATTR), whose disease has continued to worsen after liver transplantation.
- Conditions
- Transthyretin (TTR)-Mediated Amyloidosis, Familial Amyloidotic Polyneuropathy (FAP), ATTR Amyloidosis, Familial Amyloid Neuropathies
- Trial name
- NCT02595983
- Testing
- Revusiran
- Where
- 6 sites in France, Germany and 4 more
- Volunteers
- 12 people
- Sponsor
- Alnylam Pharmaceuticals
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