Clinical trials: Transthyretin Amyloidosis
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
Hereditary Amyloidosis, Transthyretin-Related, Asymptomatic Carrier State
Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathy
This is a single-center observational study evaluating the potential value of NfL as a biomarker for diagnosis, detection of disease onset, monitoring of disease progression, and treatment response in asymptomatic carriers of TTR…
- Trial name
- NCT06360289
- Testing
- Standard of Care
- Where
- France
- Volunteers
- 346 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
A Study to Learn About the Study Medicine Called Tafamidis 61mg in People Diagnosed With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
The purpose of this clinical trial is to learn about the effects of the study medicine (called Tafamidis 61milligrams (mg)) for the potential treatment of Transthyretin amyloid cardiomyopathy (ATTR-CM).
- Trial name
- NCT06321523
- Testing
- Tafamidis 61 milligrams
- Where
- 4 sites in South Korea
- Volunteers
- 13 people
- Sponsor
- Pfizer
Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Study of Acoramidis in Patients With Transthyretin Amyloid Cardiomyopathy
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive heart disease caused by the buildup of an abnormal protein, called transthyretin (TTR), in the heart.
- Trial name
- MOSAIC-TTR (NCT07791160)
- Testing
- Acoramidis
- Where
- France
- Volunteers
- 500 people
- Sponsor
- Bayer
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
The CARDIO-TTRansform Scintigraphy Sub-study
The purpose of this study is to examine the changes in amyloid myocardial burden in a subset of the population participating in the ION682884-CS2 (NCT04136171) study, up to 150 participants, after treatment with eplontersen or…
- Trial name
- NCT06073587
- Testing
- Scintigraphy scan
- Where
- 5 sites in United States, Spain
- Volunteers
- 163 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
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 (ATTR CM)
A Research Study to Look at Long-term Treatment With a Medicine Called NNC6019-0001 for People Who Have Heart Failure Due to Transthyretin Amyloidosis
This study will test a medicine, NNC6019-0001, for people who have a heart disease due to TTR amyloidosis. It will look at how safe this medicine is in the long term and if it can reduce symptoms of a heart disease due to TTR…
- Trial name
- NCT06260709
- Testing
- NNC6019-0001
- Where
- 38 sites in United States, Canada and 8 more
- Volunteers
- 88 people
- Sponsor
- Novo Nordisk A/S
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 Amyloidosis (ATTR) With Cardiomyopathy
A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
The purpose of this study is to obtain safety, efficacy, and pharmacodynamic data on the use of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy who continued on extended use of vutrisiran, or switched from…
- Trial name
- NCT06679946
- Testing
- Vutrisiran
- Where
- 87 sites in United States, Argentina and 15 more
- Volunteers
- 700 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
Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy
This study will examine the clinical effectiveness of Tafamidis in patients with Mixed Phenotype Transthyretin Amyloidosis using data that already exist in patients' medical records
- Trial name
- NCT06393465
- Testing
- Tafamidis
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Pfizer
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
To evaluate the efficacy of eplontersen compared to placebo in participants with ATTR-CM receiving available standard of care (SoC).
- Trial name
- NCT04136171
- Testing
- Eplontersen
- Where
- 139 sites in United States, Argentina and 19 more
- Volunteers
- 1,438 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Symptomatic Transthyretin Amyloid Cardiomyopathy
A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM
This prospective study is designed to evaluate the efficacy, safety, and tolerability of ALXN2060 (also known as AG10), as well as to establish its pharmacokinetic and pharmacodynamic profile in Japanese participants with…
- Trial name
- NCT04622046
- Testing
- ALXN2060
- Where
- 11 sites in Japan
- Volunteers
- 25 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Transthyretin-mediated Amyloidosis
A Study to Assess the PK, PD, Safety and Tolerability of Eplontersen in Healthy Chinese Volunteers
This is a Phase 1 study to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of eplontersen following single dosing in healthy Chinese participants.
- Trial name
- NCT06527755
- Testing
- Eplontersen Solution for Injection
- Where
- China
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Hereditary Transthyretin Amyloidosis With Polyneuropthy
A Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Participants With hATTR-PN
The objective of the study was to characterize adverse events (AEs) occurring within one day of TEGSEDI administration to adult participants with hATTR-PN overall and in individual participants with respect to time course of AE…
- Trial name
- NCT04306510
- Testing
- TEGSEDI
- Where
- 2 sites in United States, Canada
- Volunteers
- 8 people
- Sponsor
- Akcea Therapeutics
Transthyretin Amyloid Cardiomyopathy
EPIC-ATTR: A Study to Evaluate the Effect of Eplontersen on the Transthyretin Reduction and Long-term Safety in Chinese Subjects With Transthyretin Amyloid Cardiomyopathy
The purpose of this study is to investigate the effect of eplontersen compared to placebo on the reduction of serum TTR concentration and long-term safety in Chinese participants with hereditary or wild-type transthyretin amyloid…
- Trial name
- EPIC-ATTR (NCT06194825)
- Testing
- Eplontersen
- Where
- 26 sites in China
- Volunteers
- 64 people
- Sponsor
- AstraZeneca
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
Transthyretin Amyloidosis Cardiomyopathy, Heart Failure With Preserved Ejection Fraction
Transthyretin Amyloidosis Cardiomyopathy in Patients With HFpEF in Russia
A multicenter observational retrospective-prospective study of prevalence and clinical characteristics of transthyretin amyloidosis (ATTR) cardiomyopathy (CM) in Russian patients with heart failure with preserved ejection…
- Trial name
- TETRAMER (NCT06338839)
- Where
- 21 sites in Russia
- Volunteers
- 1,770 people
- Sponsor
- AstraZeneca
Familial Amyloidotic Cardiomyopathy (FAC)
DISCOVERY: A Study Examining the Prevalence of TTR Mutations in Subjects Suspected of Having Cardiac Amyloidosis
The purpose of this study is to characterize the frequency of TTR mutations in subjects suspected of having cardiac amyloidosis
- Trial name
- NCT02252653
- Where
- 58 sites in United States, Belgium and 4 more
- Volunteers
- 1,010 people
- Sponsor
- Alnylam Pharmaceuticals