Clinical trials: ATTR
TTR-mediated Amyloidosis
Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis
This was a multiple dose, dose escalation study designed to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of patisiran (ALN-TTR02) in participants with transthyretin (TTR) mediated amyloidosis (ATTR).
- Trial name
- NCT01617967
- Testing
- Patisiran
- Where
- 10 sites in United States, Brazil and 5 more
- Volunteers
- 29 people
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
The purpose of this study is to evaluate the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.
- Trial name
- NCT03997383
- Testing
- Patisiran
- Where
- 90 sites in United States, Argentina and 20 more
- Volunteers
- 360 people
- Sponsor
- Alnylam Pharmaceuticals
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
ATTR-CM, TTR-CM
Safety And Efficacy Evaluation Of Fx-1006a In Patients With V122i Or Wild-Type Transthyretin (TTR) Amyloid Cardiomyopathy
Open-label Safety and Efficacy Evaluation of Fx-1006a in Patients with V122i Or Wild-type Transthyretin (ttr) Amyloid Cardiomyopathy.
- Trial name
- NCT00935012
- Testing
- tafamidis
- Where
- 7 sites in United States
- Volunteers
- 31 people
- Sponsor
- Pfizer
Amyloidosis, Hereditary, Transthyretin Amyloidosis
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
The purpose of this study is to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02) in participants with hereditary transthyretin amyloidosis (hATTR amyloidosis).
- Trial name
- NCT03759379
- Testing
- Patisiran, Vutrisiran
- Where
- 64 sites in United States, Argentina and 20 more
- Volunteers
- 164 people
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin Amyloid Cardiomyopathy
SGLT2 Inhibitors in Transthyretin Amyloid Cardiomyopathy
This is a single center, single arm, prospective, 12 week open label pilot trial of the sodium-glucose cotransporter 2 inhibitor (SGLT2i), empagliflozin 10 mg oral daily, in patients with transthyretin amyloid cardiomyopathy…
- Trial name
- NCT05233163
- Testing
- Empagliflozin
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Columbia University
Amyloidosis
A Study to Evaluate Organ Level Uptake Repeatability of 124I AT-01 in Subjects With Systemic Amyloidosis
This study is designed to assess the repeatability of organ-specific quantitation of radiotracer uptake following Positron Emission Tomography/Computed Tomography (PET/CT) imaging of AT- 01 in subjects with amyloid light chain…
- Trial name
- AT01-001 (NCT05235269)
- Testing
- 124I-AT-01
- Where
- 3 sites in United States
- Volunteers
- 33 people
- Sponsor
- Attralus, Inc.
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
Biodistribution of 89Zirconium-labelled GSK2398852 Using PET Imaging
The principal aim of this study is to investigate the cardiac uptake of 89Zr-GSK2398852 in subjects with transthyretin cardiomyopathy amyloidosis (ATTR-CM), and its biodistribution to other organs.
- Trial name
- NCT03417830
- Testing
- GSK2315698 (CPHPC), GSK2398852 (unlabeled anti-SAP mAb), 89Zr-GSK2398852 (89Zr-labeled anti-SAP mAb)
- Where
- Sweden
- Volunteers
- 2 people
- Sponsor
- GlaxoSmithKline
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
Familial ATTR-CM (ATTRm-CM, or FAC), Wild-type ATTR-CM (ATTRwt-CM)
Study of AG10 in Amyloid Cardiomyopathy
This prospective, randomized, multicenter, double-blind, parallel group, placebo-controlled, dose-ranging study will evaluate the safety, tolerability, PK (Pharmacokinetic) and PD (Pharmacodynamic) of AG10 compared to placebo…
- Trial name
- NCT03458130
- Testing
- AG10
- Where
- 13 sites in United States
- Volunteers
- 49 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
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
Transthyretin Gene Mutations, Transthyretin Amyloidosis
Transthyretin Amyloidosis Outcome Survey (THAOS)
THAOS is a global, multi-center, longitudinal observational survey open to all patients with transthyretin amyloidosis (ATTR), including ATTR-PN (polyneuropathy), ATTR-CM (cardiomyopathy) and wild-type ATTR-CM.
- Trial name
- THAOS (NCT00628745)
- Testing
- None. Observational Study.
- Where
- 113 sites in United States, Argentina and 23 more
- Volunteers
- 6,718 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
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
Healthy Volunteers, hATTR Amyloidosis
Evaluate the Safety and Tolerability, as Well as the Pharmacokinetic and Pharmacodynamic Profiles of Single and Multiple Doses of Eplontersen Administered Subcutaneously to Healthy Volunteers and Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR ).
To evaluate the safety and tolerability, as well as the pharmacokinetic and pharmacodynamic profiles of single and multiple doses of Eplontersen administered subcutaneously to healthy volunteers and patients with Hereditary…
- Trial name
- NCT03728634
- Testing
- ION-682884, Vitamin A
- Where
- Canada
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
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