Clinical trials: Amyloidosis Transthyretin
Transthyretin Amyloid Cardiomyopathy
Retinal Fundus Imaging and OCT Imaging for Ocular Detection of ATTR-CM
This multicenter pilot study will enroll adult participants with a confirmed clinical diagnosis of transthyretin amyloid cardiomyopathy (ATTR-CM) and heart failure (HF) controls with documentation within the past 2 years that…
- Trial name
- NCT07690436
- Where
- 20 sites in United States, Germany and 3 more
- Volunteers
- 500 people
- Sponsor
- AstraZeneca
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
Transthyretin Familial Amyloid Polyneuropathy
The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin
Tafamidis has been developed as an oral specific stabilizer of transthyretin tetramer.
- Trial name
- NCT01435655
- Testing
- tafamidis
- Where
- 2 sites in Japan
- Volunteers
- 10 people
- Sponsor
- Pfizer
Hereditary Transthyretin Amyloidosis, Carpal Tunnel Syndrome
Hereditary Transthyretin Amyloidosis Polyneuropathy in Patients With Carpal Tunnel Syndrome in Russia
This is a multicenter observational study consisting of retrospective and prospective phases. The retrospective phase will entail secondary data collection from electronic or paper medical records of patients who underwent…
- Conditions
- Hereditary Transthyretin Amyloidosis, Carpal Tunnel Syndrome, Polyneuropathy, Amyloidosis
- Trial name
- LOCUS (NCT06414746)
- Where
- 13 sites in Russia
- Volunteers
- 721 people
- Sponsor
- AstraZeneca
Amyloidosis
Multiple Treatment Session Study to Assess GSK2398852 Administered Following and Along With GSK2315698
The study is intended to evaluate whether monthly repeated courses of administration of GSK2315698 followed by GSK2398852 is associated with a reduction in cardiac amyloid load in patients with cardiac amyloidosis, monitored by…
- Trial name
- NCT03044353
- Testing
- GSK2315698 (CPHPC), GSK2398852 (anti-SAP mAb)
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 7 people
- Sponsor
- GlaxoSmithKline
Transthyretin Amyloid Cardiomyopathy ("ATTR-CM")
A Prospective Study to Assess the Utility of CA-4F in a Canadian Cardiology Setting
This study will assess whether CA-4F can improve diagnosis of ATTR-CM in a community cardiology setting when used to screen electronic medical record data for patients suitable for follow-up investigations.
- Trial name
- NCT05210010
- Testing
- CA-4F
- Where
- 3 sites in Canada
- Volunteers
- 8 people
- Sponsor
- Ensho Health Intelligent Systems Inc.
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 Amyloidosis With Cardiomyopathy
DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting
The purpose of this study is to: Describe the clinical characteristics of adult patients with transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM) treated with vutrisiran in routine clinical care Describe treatment…
- Trial name
- DemonsTTRate (NCT07358078)
- Where
- 27 sites in United States, Germany and 1 more
- Volunteers
- 2,000 people
- Sponsor
- Alnylam Pharmaceuticals
Amyloidosis, Hereditary
ATTR Expanded Access Program (EAP) by Ionis
The purpose of this program is to provide expanded access to Inotersen for up to 100 Patients with Hereditary Transthyretin Amyloidosis (hTTR).
- Trial name
- NCT03400098
- Testing
- Inotersen
- Sponsor
- Ionis Pharmaceuticals, Inc.
Transthyretin Amyloid Cardiomyopathy, Heart Failure With Preserved Ejection Fraction
Global Prevalence of ATTR-CM in Participants With HFpEF
This study is a global, multi-center study designed to estimate the global prevalence of transthyretin amyloid cardiomyopathy (ATTR-CM) within a clinically at risk population [participants with heart failure with preserved…
- Trial name
- NCT04424914
- Testing
- Scintigraphy
- Where
- 51 sites in United States, Canada and 6 more
- Volunteers
- 347 people
- Sponsor
- Pfizer
Transthyretin-Mediated Amyloidosis
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects
The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single ascending doses of ALN-TTRSC04.
- Trial name
- NCT05661916
- Testing
- ALN-TTRSC04
- Where
- United Kingdom
- Volunteers
- 96 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
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
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 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 Transthyretin Cardiomyopathy
First-in-Human Study of NI006 in Patients With Amyloid Transthyretin Cardiomyopathy
A phase 1, randomized, placebo-controlled, double-blind, dose escalation trial combining single-ascending dose and multiple-ascending dose phases of NI006 or placebo, followed by an open-label extension phase in subjects with…
- Trial name
- NCT04360434
- Testing
- NI006
- Where
- 6 sites in France, Germany and 2 more
- Volunteers
- 46 people
- Sponsor
- Neurimmune AG
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
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
Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
A Study to Learn About the Use of Acoramidis in Patients With a Heart Condition Called Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in a Real-world Setting
Transthyretin Amyloid Cardiomyopathy (ATTR-CM) is a serious and life-threatening condition where a protein called transthyretin (TTR) misfolds and builds up as amyloid fibrils in the heart muscle.
- Trial name
- ACO-REAL (NCT07235462)
- Testing
- Acoramidis (356 mg film-coated tablets)
- Where
- 17 sites in Austria, Belgium and 15 more
- Volunteers
- 2,000 people
- Sponsor
- Bayer
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.
- Trial name
- NCT05667493
- Testing
- Eplontersen
- Where
- 134 sites in United States, Argentina and 18 more
- Volunteers
- 1,400 people
- Sponsor
- Ionis Pharmaceuticals, Inc.