Clinical trials: Amyloid Cardiomyopathy, Transthyretin-Related
Transthyretin Amyloidosis, Cardiomyopathy
Safety and Efficacy Study of Doxycycline/UrsoDeoxyCholicAcid on Disease Progression in ATTR Amyloidosis
The primary objective for this study is to evaluate the efficacy of doxycycline + ursodeoxycholic acid (UDCA) on disease progression in Transthyretin Amyloidosis (ATTR) subjects with cardiomyopathy with or without neuropathy.
- Trial name
- Dox/Urso (NCT02016365)
- Testing
- Doxycycline, Ursodeoxycholic acid
- Where
- 3 sites in Sweden
- Volunteers
- 55 people
- Sponsor
- Umeå University
Amyloidosis, Trigger Finger
Cardiac Amyloidosis Screening at Trigger Finger Release
The investigators will prospectively evaluate for the presence of amyloid deposits in soft tissue samples obtained from patients undergoing trigger finger release surgery.
- Conditions
- Amyloidosis, Trigger Finger, Transthyretin Amyloidosis, Primary Amyloidosis of Light Chain Type
- Trial name
- CAST (NCT03886155)
- Testing
- Biopsy
- Where
- United States
- Volunteers
- 107 people
- Sponsor
- The Cleveland Clinic
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 Amyloidosis, Carpal Tunnel Syndrome
Systemic Transthyretin Amyloidosis: Carpal Tunnel Syndrome in a Portuguese Population
Systemic transthyretin amyloidosis is an aging-related disorder. It is usually associated with cardiac disease but also extends to other organs.
- Trial name
- CarPoS (NCT05409833)
- Testing
- Synovial biopsy and Cardiac scintigraphy with [99mTc]Tc-DPD
- Where
- 2 sites in Portugal
- Volunteers
- 100 people
- Sponsor
- Universidade do Porto
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
Amyloidosis in Transthyretin (TTR), Hereditary Amyloidosis, Transthyretin-Related
Validation of a Questionnaire for Quality of Life Assessment in Patients With Transthyretin Amyloidosis.
Prospective study of development and validation of a questionnaire on quality of life in patients with Hereditary Amyloidosis The questionnaire development process will include 4 medical specialists experts in Transthyretin…
- Trial name
- NCT07080268
- Where
- Argentina
- Sponsor
- Hospital General de Niños Pedro de Elizalde
Transthyretin (TTR) Amyloid Cardiomyopathy, Cardiac Amyloidosis
Liver and Coagulation Disorders in Cardiac Transthyretin Amyloidosis
Transthyretin cardiac amyloidosis (ATTR-CA) is a progressive infiltrative cardiomyopathy caused by the deposition of misfolded transthyretin protein within the myocardium.
- Conditions
- Transthyretin (TTR) Amyloid Cardiomyopathy, Cardiac Amyloidosis, Cardiomyopathies, Wild-Type Transthyretin Cardiac Amyloidosis
- Trial name
- LICA2025 (NCT07766135)
- Where
- Italy
- Volunteers
- 70 people
- Sponsor
- University of Messina
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
Hereditary Transthyretin Amyloidosis
An Interventional Pilot Study to Investigate the Feasibility and Acceptance of a Structured Psychological Support Program for Patients, Caregivers, and Presymptomatic Carriers in Hereditary Transthyretin Amyloidosis With Cardiomyopathy
Hereditary transthyretin amyloidosis (ATTRv) is a genetic, progressive and disabling disease that affects multiple organs and severely compromises the quality of life of patients and their families.
- Trial name
- AMH (NCT06907186)
- Testing
- Psychological Support
- Where
- Italy
- Volunteers
- 150 people
- Sponsor
- Fondazione IRCCS Policlinico San Matteo di Pavia
Transthyretin Amyloidosis, Heart Failure
Prevalence and Prediction of ATTR in Ambulatory Patients With HFpEF
Recent studies have shown that transthyretin amyloidosis (ATTR) can sometimes cause a type of heart failure where the pumping function of the heart is normal, also known as Heart Failure with Preserved Ejection Fraction (HFpEF)…
- Conditions
- Transthyretin Amyloidosis, Heart Failure, Heart Failure, Diastolic, Amyloidosis
- Trial name
- TTRinHFpEF (NCT05577819)
- Testing
- 99mTc-pyrophosphate Scintigraphy
- Where
- United States
- Volunteers
- 515 people
- Sponsor
- Massachusetts General Hospital
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
Amyloidosis, Cardiomyopathy
Carpal Tunnel Syndrome and Amyloid Cardiomyopathy
The investigators will prospectively evaluate tissue samples obtained from patients undergoing carpal tunnel release surgery for amyloid in the soft tissue that is removed.
- Conditions
- Amyloidosis, Cardiomyopathy, Transthyretin Amyloidosis, Senile Systemic Amyloidosis
- Trial name
- NCT02792790
- Where
- United States
- Volunteers
- 96 people
- Sponsor
- The Cleveland Clinic
Amyloid Cardiomyopathy, Transthyretin-Related
Screening for Cardiac Amyloidosis With Nuclear Imaging for Minority Populations
In this study, the investigators recruited a cohort of elderly Black and Hispanic patients with heart failure to define the number of patients who have cardiac amyloidosis by utilizing highly sensitive heart imaging and blood…
- Trial name
- SCAN-MP (NCT03812172)
- Testing
- 99mTc-PYP or 99m Tc-HDP
- Where
- 4 sites in United States
- Volunteers
- 646 people
- Sponsor
- Mathew S. Maurer, MD
Carpal Tunnel, Transthyretin Amyloidosis
Concordance for Transthyretin Amyloidosis Between Synovial Biopsy and Anterior Carpal Ligament Biopsy
Transthyretin amyloidosis (ATTR) is an underdiagnosed condition that can present early as carpal tunnel syndrome, sometimes preceding cardiac involvement by several years.
- Trial name
- ATTR2026 (NCT07644325)
- Testing
- Annular ligament biopsy
- Where
- France
- Volunteers
- 500 people
- Sponsor
- Clinique Saint Jean, France
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
Cardiac Amyloidosis, Transthyretin Amyloidosis
Retrospective Identification of Scintigraphic Cardiac Amyloidosis (RISCA)
This study aims to determine the positive predictive value of incidental cardiac uptake on bone scintigraphy for diagnosing transthyretin cardiac amyloidosis, in patients who underwent scintigraphy for reasons other than…
- Trial name
- RISCA (NCT06921408)
- Volunteers
- 57 people
- Sponsor
- University Hospital, Lille
Neuromuscular Disease, Neuromuscular Diseases (NMD)
A Phase 3 Study of NTLA-2001 in ATTRv-PN
This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.
- Conditions
- Neuromuscular Disease, Neuromuscular Diseases (NMD), Neurodegenerative Disease, Neurodegenerative Disease, Hereditary
- Trial name
- NCT06672237
- Testing
- nexiguran ziclumeran
- Where
- 14 sites in Argentina, Brazil and 4 more
- Volunteers
- 67 people
- Sponsor
- Intellia Therapeutics
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
The purpose of this study is to evaluate the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.
- Trial name
- NCT03997383
- Testing
- Patisiran
- Where
- 90 sites in United States, Argentina and 20 more
- Volunteers
- 360 people
- Sponsor
- Alnylam Pharmaceuticals
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, Transthyretin Amyloidosis
ATTRACT-52: Primary Care Cardiac Amyloidosis Screening in Ordu, Turkey
Cardiac amyloidosis is a progressive infiltrative cardiomyopathy, most commonly related to transthyretin (ATTR) misfolding. Although considered rare, emerging data suggest higher prevalence in specific regions, including the…
- Trial name
- ATTRACT-52 (NCT07238426)
- Where
- Turkey (Türkiye)
- Volunteers
- 800 people
- Sponsor
- Kotyora Family Medicine Health Management and Education Association