Clinical trials: Amyloid Cardiomyopathy, Transthyretin-Related
Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, Polyneuropathy
A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.
- Trial name
- NCT04201418
- Testing
- Patisiran
- Where
- 27 sites in United States
- Volunteers
- 67 people
- Sponsor
- Alnylam Pharmaceuticals
Amyloidosis, Amyloid
Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis
This study measures circulating, misfolded ATTR oligomers in asymptomatic ATTRm amyloidosis genetic carriers longitudinally over five years.
- Conditions
- Amyloidosis, Amyloid, Amyloid Neuropathies, Familial, Amyloid Cardiomyopathy
- Trial name
- MED-hATTR (NCT03431896)
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- The Cleveland Clinic
Transthyretin Amyloidosis, Carpal Tunnel Syndrome
Transthyretin Cardiac Amyloidosis in Patients With Idiopathic Carpal Tunnel Syndrome Referred for Release Surgery
This study will determine the prevalence of transthyretin amyloidosis in pathology of the transverse carpal ligament and cardiac involvement using natriuretic peptides, electrocardiography and echocardiography in patients…
- Trial name
- PRATHYC (NCT04611204)
- Testing
- Descriptive cohort
- Where
- France
- Volunteers
- 6 people
- Sponsor
- University Hospital, Toulouse
Hereditary Amyloidosis, Transthyretin-Related
Early Detection of Neuropathy in ATTRv
The purpose of the study is to evaluate and compare different tools that are used to detect evidence of peripheral neuropathy in patients with TTRv.
- Trial name
- EDONA (NCT05311488)
- Testing
- neurofilament light chain, In-vivo Meissner Corpuscle imaging, Nerve conduction study
- Where
- United States
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
Transthyretin Amyloidosis
New Biomarkers and Plasma Prothrombotic Potential in Cardiac Transthyretin Amyloidosis
The development of cardiac amyloidosis is caused by the deposition of misfolded, insoluble proteins in the extracellular matrix of tissues.
- Trial name
- NCT06345235
- Testing
- blood test
- Where
- Poland
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- John Paul II Hospital, Krakow
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 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
Transthyretin Amyloidosis, Amyloidosis, Leptomeningeal, Transthyretin-Related
Short-term Effects of TOLCAPONE on Transthyretin Stability in Subjects With Leptomeningeal TTR Amyloidosis (ATTR)
The purpose of this study is to determine whether Tolcapone crosses from the blood stream into the fluid around the brain and stabilizes the protein that makes leptomeningeal amyloid.
- Trial name
- NCT03591757
- Testing
- Tolcapone
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Boston University
Transthyretin Cardiomyopathy (TTR-CM), Transthyretin (TTR) Amyloid Cardiomyopathy
PL_GNT01_ISR_Grant 53234273
We carry out a prospective non-interventional, longitudinal, observational multicentre registry designed to improve our understanding of the epidemiology of TTR amyloidosis in our country.
- Conditions
- Transthyretin Cardiomyopathy (TTR-CM), Transthyretin (TTR) Amyloid Cardiomyopathy, Transthyretin Amyloidosis
- Trial name
- NCT06714019
- Where
- Poland
- Volunteers
- 100 people
- Sponsor
- National Institute of Cardiology, Warsaw, Poland
Amyloidosis, Aortic Valve Stenosis
Screening for Amyloidosis Before Aortic Valve Elective Replacement
Previous studies detected that up to 15% of patients undergoing aortic valve replacement (AVR) for degenerative aortic stenosis have concomitant transthyretin amyloidosis (ATTR) cardiomyopathy (Castano, 2017).
- Trial name
- SAVER (NCT04869631)
- Testing
- The outcome of an intervention is not evaluated, but aortic stenosis and additional amyloidosis are compared.
- Where
- Germany
- Volunteers
- 1,000 people
- Sponsor
- Charite University, Berlin, Germany
Transthyretin Amyloidosis, Amyloidosis, Hereditary
Myocardial Effects in Patients With ATTRv With Polyneuropathy Treated With Patisiran or Vutrisiran
ATTRv amyloidosis is a systemic disease with two clinical forms, neurological and cardiological, which are sometimes combined (so-called mixed forms).
- Trial name
- MyocardON-TTR (NCT05873868)
- Testing
- Six minutes walk test, Kansas City questionnaire, COMPASS31 self questionnaire
- Where
- 5 sites in France
- Volunteers
- 20 people
- Sponsor
- Rennes University Hospital
Hereditary Transthyretin Amyloidosis
Exploring Biomarkers in Hereditary Transthyretin Amyloidosis
Hereditary transthyretin amyloidosis (ATTRv, v for variant) is a severe and heterogeneous systemic condition due to mutations in the transthyretin (TTR) gene.
- Trial name
- ELBA (NCT05929209)
- Testing
- Assessment of disease biomarkers
- Where
- Italy
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Familial Amyloid Neuropathy, Transthyretin Amyloidosis
Assessment of the Prevalence of TTR Amyloid Neuropathy in a Population of Patients With Neuropathy of Unknown Aetiology
Familial amyloid neuropathy due to transthyretin gene mutations (TTR-FAP) is a rare autosomal dominant inherited disease resulting in the abnormal multi-system deposition of amyloid proteins.
- Trial name
- PRE-TRANS (NCT03190577)
- Testing
- blood sample
- Where
- 12 sites in France
- Volunteers
- 400 people
- Sponsor
- Nantes University Hospital
Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis
Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis
The study aims to evaluate the effect of acoramidis on cardiac functional and structural improvement in participants with ATTR-CM.
- Conditions
- Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis
- Trial name
- ASCEND-ATTR (NCT07695701)
- Testing
- Acoramidis (AG10) oral tablet
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Amyloid, Amyloidosis
Screening for Systemic Amyloidosis Via the Ligamentum Flavum
The investigators will prospectively evaluate for the presence of amyloid deposits in ligamentum flavum (yellow ligament) tissue samples obtained from patients undergoing spinal stenosis surgery.
- Conditions
- Amyloid, Amyloidosis, Spinal Stenosis, Transthyretin Amyloidosis
- Trial name
- ALF (NCT03923920)
- Testing
- Biopsy
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- The Cleveland Clinic
Transthyretin (TTR) Amyloid Cardiomyopathy
Determining the Association of TTR Stabilizing Therapy With Circulating TTR Amyloid Aggregates Over Time in Patients With ATTR-CM: Longitudinal Biomarker Study
The objective of this study is to determine the association of clinically prescribed, on-label, TTR stabilizing therapy (e.g. tafamidis or acoramidis) with levels of circulating transthyretin amyloid aggregates (TAAs, a surrogate…
- Trial name
- NCT07196839
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Texas Southwestern Medical Center
Transthyretin Amyloidosis, Transthyretin Amyloidosis With Cardiomyopathy
Long-term Safety of Eplontersen Treated aTTR Patients and in Liver Transplant and Severely Hepatic Impaired Subpopulations
The aim of this observational cohort study is to characterise use of eplontersen in patients with prior liver transplant or with pre-existing severe hepatic impairment, as well as to assess long-term safety among all new users of…
- Conditions
- Transthyretin Amyloidosis, Transthyretin Amyloidosis With Cardiomyopathy, Transthyretin Amyloidosis With Polyneuropathy
- Trial name
- sTTRing (NCT07654855)
- Testing
- None ( observational study )
- Where
- 6 sites in United States, Canada and 4 more
- Volunteers
- 320 people
- Sponsor
- AstraZeneca
Transthyretin Amyloidosis
The Effect of Tafamidis on Transthyretin Stabilization, Safety, Tolerability and Efficacy in Transthyretin Amyloid Polyneuropathy Patients
Transthyretin amyloid polyneuropathy (ATTR-PN) is a fatal illness resulting from autosomal dominantly inherited single-point mutations on the transthyretin gene.
- Trial name
- NCT04963985
- Testing
- Tafamidis Pill
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Peking University Third Hospital
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
Amyloid Cardiomyopathy, Transthyretin-Related
Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants
Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)
- Trial name
- AG10 (NCT04988386)
- Testing
- Acoramidis (AG10)
- Where
- 72 sites in United States, Australia and 15 more
- Volunteers
- 389 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company