Clinical trials: Transthyretin Amyloidosis
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
Cardiac Amyloidosis
CAPACITY (Cardiac Amyloidosis and Physical ACtivITY) Study
Exercise training in patients with heart failure and preserved ejection fraction (HFpEF) has been associated with an improvement in cardiorespiratory fitness and quality of life.
- Trial name
- CAPACITY (NCT06096675)
- Testing
- • Cardiac Rehabilitation
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Wake Forest University Health Sciences
Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy, Pregnancy
TEGSEDI Pregnancy Surveillance Program
This is a worldwide safety surveillance study of pregnancy outcomes in women with hATTR-PN who may have been exposed or were not exposed to TEGSEDI prior to or during the pregnancy and of pediatric outcomes up to 1 year of age.
- Trial name
- NCT04270058
- Testing
- inotersen
- Volunteers
- 20 people
- Sponsor
- Akcea Therapeutics
Cardiac Amyloidosis
Description of a Retrospective Cohort of Patients With Transthyretin Cardiac Amyloidosis (ATTR-CA) in Internal Medicine
In 2024, the prevalence of TTR cardiac amyloidosis is difficult to determine precisely. Indeed, the disease has long been underdiagnosed due to a lack of effective treatment and non-invasive diagnostic methods.
- Trial name
- ATTR-CA (NCT06907836)
- Where
- France
- Volunteers
- 30 people
- Sponsor
- University Hospital, Strasbourg, France
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
Light-chain Cardiac Amyloidosis
Selinexor, Daratumumab, and Dexamethasone (XDd) for Light-Chain Cardiac Amyloidosis
The goal of this clinical trial is to evaluate whether selinexor combined with daratumumab and dexamethasone (XDd) can treat patients with advanced light-chain cardiac amyloidosis.
- Trial name
- NCT07743957
- Testing
- XDd
- Volunteers
- 63 people
- Sponsor
- Beijing Anzhen Hospital
Amyloidosis, Hereditary, Amyloidosis Cardiac
Subclinical Transthyretin Cardiac Amyloidosis in V122I TTR Carriers
Approximately 1.5 million of the 44 million Blacks in the United States are carriers of the valine-to-isoleucine substitution at position 122 (V122I) in the transthyretin (TTR) protein.
- Conditions
- Amyloidosis, Hereditary, Amyloidosis Cardiac, Amyloidosis, Familial, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
- Trial name
- NCT05489549
- Where
- 3 sites in United States
- Volunteers
- 500 people
- Sponsor
- University of Texas Southwestern Medical Center
Cardiac Amyloidosis
Exercise Training and Rehabilitation In Cardiac Amyloidosis
Notwithstanding the dramatic improvement associated with Tafamidis in Heart Failure (HF) due to wild-type transthyretin cardiac amyloidosis (ATTRwt-CA), remarkable morbidity and mortality still burden this disease.
- Trial name
- ERICA (NCT06412432)
- Testing
- Exercise training (ET1+ET2), Optimal Medical Therapy (No-ET)
- Where
- Italy
- Volunteers
- 34 people
- Sponsor
- Federico II University
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
Cardiac Amyloidosis
Prevalence of Cardiac Thrombi in Cardiac Amyloidosis
Cardiac amyloidosis (CA) is an infiltrative disease characterized by deposits of amyloid proteins of genetic or acquired origin (often in elderly patients), leading to heart failure and arrhythmias.
- Trial name
- CATICA (NCT07648303)
- Testing
- Cardiac CT scan with contrast
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Centre Hospitalier Universitaire Dijon
Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
The purpose of this study is to: Describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients Characterize the safety and effectiveness of patisiran and…
- Trial name
- ConTTRibute (NCT04561518)
- Where
- 40 sites in United States, Brazil and 10 more
- Volunteers
- 1,500 people
- Sponsor
- Alnylam Pharmaceuticals
Amyloid Neuropathies, Transthyretin Amyloidosis
Expanding the Biomarkers in Familial Amyloid Neuropathy: MRI and Motor Unit Estimation by Electrophysiological Study
Familial amyloid neuropathies (FAP) are hereditary disease due to a mutation of the tranthyretin gene (TTR). These neuropathies are severe and life frightening.
- Trial name
- NCT03588468
- Testing
- Electrophysiological biomarkers, MRI biomarkers
- Where
- France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Assistance Publique Hopitaux De Marseille
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
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
Amyloidosis
The Effect of an Antisense Oligonucleotide to Lower Transthyretin (TTR) Levels on the Progression of -Wild-type TTR Involving the Heart
ATTRwt (also known as senile systemic, or senile cardiac amyloidosis) is a progressive heart disease, causing congestive heart failure.
- Trial name
- NCT02627820
- Testing
- Isis 420915/GSK 299872
- Sponsor
- Brigham and Women's Hospital
Amyloid Cardiomyopathy
Genistein in trAnSthyretin recePtor Amyloid caRdiomyopathy
This Phase 1b/2a study aims to investigate the safety and efficacy of genistein in patients with Transthyretin (TTR) Amyloidosis.
- Trial name
- GASPAR (NCT06634108)
- Testing
- Genistein
- Where
- Canada
- Volunteers
- 20 people
- Sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
Cardiomyopathies, Primary
Parametric Cardiac 18F-flutemetamol PET Imaging in ATTR Cardiomyopathy
18F-Flutemetamol (Vizamyl) is a radioactive diagnostic agent indicated and FDA-approved for Positron Emission Tomography (PET) imaging of the brain to estimate β-amyloid neuritic plaque density in adult patients with cognitive…
- Trial name
- NCT05374564
- Testing
- (18F)Flutemetamol
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Yale University
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
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
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