Clinical trials: Hereditary Amyloidosis
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
Hereditary Transthyretin Amyloidosis With Polyneuropthy
A Non-interventional Cohort Safety Study of Patients With hATTR-PN
This is a prospective, non-interventional, Long-term, multinational cohort safety study of patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy (hATTR-PN).
- Trial name
- NCT04850105
- Testing
- Data Collection
- Where
- 26 sites in Bulgaria, Cyprus and 6 more
- Volunteers
- 202 people
- Sponsor
- Akcea Therapeutics
Chronic Inflammatory Demyelinating Polyneuropathy, Guillain-Barre Syndrome
RWE-based Treatment Patterns and Outcomes in CIDP
To describe the demographics, clinical characteristics, treatment patterns and clinical outcomes of chronic inflammatory demyelinating polyneuropathy (CIDP), Guillain-Barre Syndrome (GBS), and heredofamilial amyloidosis (hATTR)…
- Conditions
- Chronic Inflammatory Demyelinating Polyneuropathy, Guillain-Barre Syndrome, Hereditary Amyloidosis
- Trial name
- NCT05940922
- Testing
- No intervention. Retrospective observation only.
- Where
- United Kingdom
- Volunteers
- 170 people
- Sponsor
- RwHealth
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
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
Secondary (AA) Amyloidosis, Rheumatoid Arthritis
Study of the Safety and Efficacy of NC-503 in Secondary (AA) Amyloidosis
The main objective of this study is to evaluate the safety and efficacy of NC-503 compared to placebo in patients with secondary (AA) amyloidosis using a composite assessment of clinical improvement/worsening of both renal and…
- Conditions
- Secondary (AA) Amyloidosis, Rheumatoid Arthritis, Nephrotic Syndrome, Familial Mediterranean Syndrome
- Trial name
- NCT00035334
- Testing
- NC-503 (Anti-amyloidotic (AA) Agent)
- Where
- 26 sites in United States, Finland and 10 more
- Volunteers
- 150 people
- Sponsor
- Bellus Health Inc. - a GSK company
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
Cardiac Amyloidosis
Test-retest Study With [18F]FBB in Cardiac Amyloidosis
This study is an open-label study to evaluate and characterize test-retest reliability of [18F]florbetaben PET in subjects with AL-CA and ATTR-CA compared to non-CA subjects.
- Trial name
- CATI (NCT06790394)
- Testing
- Florbetaben F18
- Where
- 2 sites in United Kingdom
- Volunteers
- 15 people
- Sponsor
- Lantheus Germany GmbH
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
Amyloidosis; Systemic
A Study to Characterize the Biodistribution of 124I-Labeled AT-03 in Patients With Systemic Amyloidosis
This is a single center Phase 1 study is to evaluate the biodistribution of radiolabeled AT-03 in patients with systemic amyloidosis.
- Trial name
- AT03-001 (NCT05201911)
- Testing
- 124I-AT03
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Attralus, Inc.
Hereditary Transthyretin Amyloidosis
hATTR Polyneuropathy in Russia
A multicenter observational retrosPective Registry of patIents with transthyretin aMyloid polynEuropathy (hATTR-PN) and chRonic idiopathic axonal polyneuropathy (CIAP) in the population of the Russian Federation (PRIMER) There…
- Trial name
- PRIMER (NCT06365593)
- Where
- 3 sites in Russia
- Volunteers
- 100 people
- Sponsor
- AstraZeneca
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
Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy, hATTR-PN
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
The purpose of this study is to: Determine the efficacy of nucresiran in patients with hATTR-PN by evaluating the effect on neurologic impairment, quality of life, nutritional status, disability, and gait speed Demonstrate…
- Trial name
- TRITON-PN (NCT07223203)
- Testing
- Nucresiran, Vutrisiran
- Where
- 61 sites in United States, Argentina and 20 more
- Volunteers
- 125 people
- Sponsor
- Alnylam Pharmaceuticals
Hereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed Phenotype
A Study to Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis
This study will examine the clinical effectiveness of Tafamidis in patients with Mixed Phenotype Hereditary Transthyretin Amyloidosis using data that already exist in patients' medical records.
- Trial name
- NCT05139680
- Testing
- tafamidis
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Pfizer
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
Transthyretin Amyloid Polyneuropathy
To Evaluate the Efficacy and Safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients With Transthyretin Amyloid Polyneuropathy
The purpose of this study is to evaluate the efficacy and safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients with Transthyretin Amyloid Polyneuropathy
- Trial name
- NCT06940336
- Testing
- Tafamidis Meglumine Soft Capsules
- Where
- China
- Volunteers
- 15 people
- Sponsor
- Qilu Pharmaceutical Co., Ltd.
Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Biomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP)
International, multicenter, observational, longitudinal study to identify biomarker/s for the development of a new MS-based biomarker for the early and sensitive diagnosis of Transthyretin-Related Familial Amyloidotic…
- Conditions
- Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin Amyloid Cardiopathy
- Trial name
- BioTRAP (NCT02713880)
- Where
- 2 sites in Germany, India
- Sponsor
- CENTOGENE GmbH Rostock
HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF
US Benchmarking Clinical Study
The goal of this observational study is to measure shear wave velocity (SWV) in patients with non-reduced left ventricular ejection fraction (LVEF) heart failure and left ventricular hypertrophy, with or without transthyretin…
- Conditions
- HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF, ATTR-CM (Transthyretin Amyloid Cardiomyopathy), Left Ventricular Hypertrophy
- Trial name
- EMS-CT-1 (NCT07215715)
- Where
- 3 sites in United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- eMyosound SAS
Hereditary Corneal Dystrophy, Corneal Abnormality
Safety and Efficacy Study of GEB-101 Injection in Subjects With TGFBI-related Corneal Dystrophy
The goal of this clinical trial is to evaluate the safety and efficacy of GEB-101 injection in subjects with TGFBI-related Corneal Dystrophy
- Conditions
- Hereditary Corneal Dystrophy, Corneal Abnormality, Corneal Dystrophies, Corneal Dystrophies, Hereditary
- Trial name
- CLARITY (NCT07750678)
- Testing
- GEB-101 Injection
- Where
- 2 sites in United States, China
- Volunteers
- 30 people
- Sponsor
- GenEditBio Limited
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