Clinical trials: Amyloidosis Transthyretin
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
ATTR-CM (Transthyretin Amyloid Cardiomyopathy)
Korean Post-marketing Surveillance Vyndamax® Capsules for the Treatment of Transthyretin Amyloid Cardiomyopathy
This non-interventioal study will be performed by design of post-marketing surveillance (PMS) as an additional pharmacovigilance activity of the Risk Management Plan (RMP) for Vyndamax® Capsules, which is required by the Ministry…
- Trial name
- NCT04801329
- Testing
- Vyndamax (tafamidis 61mg)
- Where
- South Korea
- Volunteers
- 110 people
- Sponsor
- Pfizer
Cardiac Amyloidosis
PATHWAY-RCT: Preventing Admission To Hospital With Attr cardiomyopathY
Cardiac amyloidosis is a condition where the heart muscle, amongst other tissues, is infiltrated by the abnormal build-up of proteins called amyloid.
- Trial name
- PATHWAY-RCT (NCT05098665)
- Testing
- Telemonitoring service
- Where
- United Kingdom
- Volunteers
- 320 people
- Sponsor
- Richmond Research Institute
Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
TRAMmoniTTR Study Genetic Screening of an At-risk Population for hATTR and Monitoring of TTR Positive Subjects
National, multicenter, epidemiological, longitudinal protocol to investigate the hATTR prevalence in an at-risk population for Hereditary Transthyretin Amyloidosis (hATTR) and subjects diagnosed with hATTR, to monitor the…
- Conditions
- Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Polyneuropathies
- Trial name
- TRAMmoniTTR (NCT03237494)
- Where
- 84 sites in Austria, Germany and 1 more
- Volunteers
- 5,028 people
- Sponsor
- CENTOGENE GmbH Rostock
Cardiac Amyloidosis, AL Amyloidosis
Efficacy of [18F]Florbetaben PET for Diagnosis of Cardiac AL Amyloidosis
This is an open-label, multi-center pivotal Phase 3 study to visually and quantitatively assess PET images obtained after single application of 300 MBq [18F]florbetaben and PET scanning of patients with suspected cardiac…
- Conditions
- Cardiac Amyloidosis, AL Amyloidosis, ATTR Amyloidosis
- Trial name
- CArdiag (NCT05184088)
- Testing
- [18F]florbetaben
- Where
- 14 sites in United States, Germany and 2 more
- Volunteers
- 244 people
- Sponsor
- Lantheus Germany GmbH
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.
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
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 Amyloid Cardiomyopathy
A Study to Assess the Safety and Efficacy Of Tafamidis In Chinese Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
This is a national, multi-center, single-arm study, open-label to patients with symptomatic Transthyretin amyloid cardiomyopathy (ATTR-CM) who are tafamidis naïve.
- Trial name
- NCT04814186
- Testing
- Tafamidis
- Where
- 9 sites in China
- Volunteers
- 53 people
- Sponsor
- Pfizer
Transthyretin-type Cardiac Amyloidosis
Real-Life Clinical Efficacy of Acoramidis in Participants With ATTR-CM and Association With Cardiac Biomarkers
The purpose of this study is to confirm that the treatment with acoramidis prevents the deterioration of the ATTR-CM disease progression index and that these indexes are surrogate markers of disease progression.
- Trial name
- ATTR-CM (NCT07306949)
- Testing
- Acoramidis
- Where
- 16 sites in Japan
- Volunteers
- 200 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Polyneuropathy, Amyloid, Neuropathic Pain
Screening for the Transthyretin-Related Familial Amyloidotic Polyneuropathy (TTR FAP)
An International, multicenter, epidemiological observational study investigating the prevalence of Transthyretin-Related Familial Amyloidotic Polyneuropathy (TTR-FAP) in participants with small fiber polyneuropathy of no obvious…
- Conditions
- Polyneuropathy, Amyloid, Neuropathic Pain, Cardiac Failure, Orthostatic Hypotension
- Trial name
- TRAP2-1 (NCT01705626)
- Where
- 11 sites in Austria, Hungary and 4 more
- Volunteers
- 500 people
- Sponsor
- CENTOGENE GmbH Rostock
TTR-mediated Amyloidosis
The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)
The purpose of this study is to evaluate the safety and tolerability of long-term dosing with ALN-TTR02 (patisiran) in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
- Trial name
- NCT01961921
- Testing
- ALN-TTR02 (patisiran) administered by intravenous (IV) infusion
- Where
- 9 sites in United States, Brazil and 5 more
- Volunteers
- 27 people
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
- Trial name
- NCT06128629
- Testing
- NTLA-2001
- Where
- 138 sites in United States, Argentina and 24 more
- Volunteers
- 1,200 people
- Sponsor
- Intellia Therapeutics
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
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
ATTR-PN
Safety And Efficacy Evaluation Of Fx-1006A In Subjects With Transthyretin Amyloidosis
This is a Phase 3, open-label study designed to obtain additional long-term safety and efficacy data for oral tafamidis (20 mg soft gelatin capsule) administered once daily (QD).
- Trial name
- NCT00925002
- Testing
- Tafamidis
- Where
- 9 sites in United States, Argentina and 6 more
- Volunteers
- 93 people
- Sponsor
- Pfizer
Amyloidosis, Familial, Transthyretin Amyloidosis
Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant
The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of patisiran in participants with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with disease progression after liver…
- Trial name
- NCT03862807
- Testing
- Patisiran
- Where
- 9 sites in France, Germany and 5 more
- Volunteers
- 24 people
- Sponsor
- Alnylam Pharmaceuticals
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
ATTR-CM (Transthyretin Amyloid Cardiomyopathy)
A Retrospective Observational Study of Epidemiology and Outcomes in Transthyretin Amyloid Cardiomyopathy in Germany
This observational study aims to describe the characteristics, health outcomes, and epidemiology of individuals diagnosed with transthyretin amyloid cardiomyopathy (ATTR-CM) in Germany.
- Trial name
- NCT07494656
- Testing
- tafamidis 61mg (based on prescription data)
- Where
- Germany
- Volunteers
- 1 people
- Sponsor
- Pfizer
TTR-mediated Amyloidosis
Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis
The purpose of this study is to determine the pharmacokinetics, pharmacodynamics and exploratory clinical activity of ALN-TTRSC (revusiran) in Patients with Transthyretin (TTR) Cardiac Amyloidosis.
- Trial name
- NCT01981837
- Testing
- ALN-TTRSC (revusiran) for subcutaneous administration
- Where
- 4 sites in United States, United Kingdom
- Volunteers
- 26 people
- Sponsor
- Alnylam Pharmaceuticals