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A Phase 1/2, First-in-Human, Open-Label, Multicenter, Single-Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of NVC-001, a Novel Adeno-Associated Virus (Serotype 9) Vector Expressing a Dominant Negative SUN1 Transgene, in Patients With LMNA-Related Dilated Cardiomyopathy (SUNBEAM-LMNA)
This study will be conducted to evaluate the safety and tolerability, and assess exploratory efficacy of NVC-001 in adults participants with LMNA-Related Dilat…
AI-Enabled Direct-from-ECG Ejection Fraction (EF) Severity Using COR ECG Wearable Monitor
This prospective, multicenter, cluster-randomized controlled study aims to evaluate the accuracy of an investigational artificial intelligence (AI) Software as…
Characteristics And Clinical Course Of Disease In Participants With Cardiomyopathy Associated With Friedreich Ataxia (CLARITY-FA)
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
Mavacamten to Aficamten Transition in Patients With Obstructive Hypertrophic Cardiomyopathy
This is an investigator-initiated two-center study. The goal of this study is to investigate the feasibility, safety and efficacy outcomes of a seamless transi…
Iodine-124 Evuzamitide PET/CT Imaging in Carriers of TTR Mutations
The purpose of this study is to determine if TTR gene carriers have early signs of a type of heart disease called amyloidosis using a new radiotracer dye (iodi…
A Phase 1/2, Multicenter, Open-label, Dose Escalation and Expansion Clinical Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ASP2957 in Male Participants With Invasive Ventilator-dependent X-linked Myotubular Myopathy
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no approved t…
Analysis of Lumbar Spine Stenosis Specimens for Identification of Transthyretin Cardiac Amyloidosis
Primary objective: To identify older adults with transthyretin cardiac amyloidosis (ATTR-CA) early in the course of the illness, at a time when disease modify…
A Phase 2b/3, Adaptive, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Assess the Efficacy and Safety of Danicamtiv in Participants With Symptomatic Genetic and Familial Dilated Cardiomyopathy
The Sponsor is studying an investigational medication called danicamtiv to determine if it can help people with genetic and familial dilated cardiomyopathy (DC…
A Global Phase 2b, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Ninerafaxstat in Patients With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy - FORTITUDE-HCM
FORTITUDE-HCM is a global, multicenter, double-blind, parallel-group, placebo-controlled Phase 2b study that will assess the efficacy and safety of ninerafaxst…
A Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicenter Study to Evaluate the Efficacy and Safety of SOtaglifloziN in symptomATic Obstructive And Non-obstructive Hypertrophic CardioMyopathy (SONATA-HCM)
The main purpose of the study is to determine the changes in symptoms and functional limitations in participants with symptomatic hypertrophic cardiomyopathy (…
Personalized Exercise Training to Improve Functional Capacity in Transthyretin Cardiac Amyloidosis
Transthyretin cardiac amyloidosis causes debilitating heart failure in older adults. The proposed research will develop a personalized exercise training progra…
Genetic Studies of Patients and Their Families With Inherited Cardiovascular and Neuromuscular Diseases.
We are studying the genetics of human cardiovascular and neuromuscular disease. There are many different genetic regions that have been associated with the dev…