Clinical trials: Amyloid Cardiomyopathy
Transthyretin Amyloidosis Polyneuropathy, Transthyrexin Amyloidosis Cardiomyopathy
A Phase I/IIa，Open-label, Single Ascending Dose and Dose-expansion Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of YOLT-201 in Patients With Transthyretin Amyloidosis Polyneuropathy (ATTR-PN) or Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
This study will be conducted to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of YOLT-201 in participants with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and…
- Trial name
- NCT06539208
- Testing
- YOLT-201
- Where
- 3 sites in China
- Volunteers
- 31 people
- Sponsor
- YolTech Therapeutics Co., Ltd
Transthyretin-type Cardiac Amyloidosis
Real-Life Clinical Efficacy of Acoramidis in Participants With ATTR-CM and Association With Cardiac Biomarkers
The purpose of this study is to confirm that the treatment with acoramidis prevents the deterioration of the ATTR-CM disease progression index and that these indexes are surrogate markers of disease progression.
- Trial name
- ATTR-CM (NCT07306949)
- Testing
- Acoramidis
- Where
- 16 sites in Japan
- Volunteers
- 200 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
ATTR-CM
A Retrospective Validation Study To Identify Chart-Based Clinical Diagnosis Of Wild-Type Transthyretin Amyloid Cardiomyopathy (Attrwt-CM) And Non-Amyloid Heart Failure Among Patients With Heart Failure (HF).
This is an observational, retrospective non-inferiority study with a study sample from a large national database. A machine learning (ML) model will use a national database to predict the clinical diagnosis of ATTRwt-CM among HF…
- Trial name
- NCT06029452
- Testing
- Machine learning algorithm
- Where
- United States
- Volunteers
- 558 people
- Sponsor
- Pfizer
Hereditary Transthyretin Amyloidosis (ATTRv), Polyneuropathy
Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv) Patients Included in B3461028 and B3461045.
A study of patients with hereditary transthyretin amyloidosis (ATTRv) and wild-type transthyretin amyloidosis (ATTRwt) that have been enrolled in B3461028 and B3461045 studies in Spain - exposed to tafamidis 61mg for ≥12 months…
- Trial name
- TRAMA (NCT05560555)
- Testing
- Tafamidis
- Where
- 3 sites in Spain
- Volunteers
- 5 people
- Sponsor
- Pfizer
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
Transthyretin Amyloid Cardiomyopathy
A Study to Learn More About the Change in the Blood Levels of Transthyretin When Participants With Transthyretin Amyloidosis With Cardiomyopathy Switch From Tafamidis to Acoramidis
Transthyretin (TTR) is a protein made by the liver that helps transport thyroid hormone and vitamin A in the blood. In some people, this protein breaks down and forms harmful clumps called amyloid.
- Trial name
- ACO-SWITCH (NCT07298044)
- Testing
- Acoramidis
- Where
- 26 sites in Austria, Belgium and 3 more
- Volunteers
- 50 people
- Sponsor
- Bayer
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
Transthyretin Amyloid Cardiomyopathy
Study of ALXN2220 Versus Placebo in Adults With ATTR-CM
The primary objective of this study is to access the efficacy of ALXN2220 in the treatment of adult participants with ATTR-CM by evaluating the difference between the ALXN2220 and placebo groups as assessed by the total…
- Trial name
- DepleTTR-CM (NCT06183931)
- Testing
- ALXN2220
- Where
- 213 sites in United States, Argentina and 25 more
- Volunteers
- 1,182 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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
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
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
- Trial name
- NCT06128629
- Testing
- NTLA-2001
- Where
- 132 sites in United States, Argentina and 24 more
- Volunteers
- 1,200 people
- Sponsor
- Intellia Therapeutics
Amyloidosis, Amyloid Cardiomyopathy
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque-like substance that accumulates in different organs in the body and can cause…
- Conditions
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies
- Trial name
- NCT06563895
- Testing
- Acoramidis
- Where
- 104 sites in United States, Argentina and 22 more
- Volunteers
- 587 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
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 Cardiac Amyloidosis
Multicenter Study for the Validation of an AI-based ECG Platform for Early Cardiac Amyloidosis Diagnosis
CONCERTO is a retrospective, observational, multicentric and single-arm study to perform an external validation of the cloud-based and AI-powered electrocardiogram (ECG) analysis platform, named Willem™, to detect Transthyretin…
- Trial name
- CONCERTO (NCT06978660)
- Where
- 8 sites in United States, France and 3 more
- Volunteers
- 2,200 people
- Sponsor
- Idoven 1903 S.L.
Transthyretin Amyloidosis With Cardiomyopathy
TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
The purpose of this study is to: Evaluate the efficacy of nucresiran compared to placebo on reducing all-cause mortality and cardiovascular (CV) events Evaluate the efficacy of nucresiran compared to placebo on additional…
- Trial name
- NCT07052903
- Testing
- Nucresiran, Sterile Normal Saline (0.9% NaCl)
- Where
- 266 sites in United States, Argentina and 34 more
- Volunteers
- 1,750 people
- Sponsor
- Alnylam Pharmaceuticals
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