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PAtient Similarity for Decision-Making in Prevention of Cardiovascular Toxicity (PACT): A Feasibility Study
This is a single-center, double-arm, open-label, randomized feasibility study that will determine whether a novel clinical decision aid accessed via the electr…
Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
The purpose of this study is to test the safety and preliminary efficacy of AAVrh.10hFXN to treat the cardiomyopathy associated with Friedreich's ataxia (FA). …
Genetic Markers of Cardiovascular Diseases and the Potential Role in Sudden Unexpected Death in Epilepsy.
Epilepsy is a common condition which affects over 3 million people in the US. Patients with uncontrolled epilepsy have a lifetime risk of sudden unexpected dea…
WiTNNess: An International Natural History Study of Autosomal Recessive TNNT1 Myopathy
WiTNNess is designed to accurately document the natural course and variation of muscle disease caused by pathogenic changes of the TNNT1 gene. The primary aim …
Prospective Observational Study of the ICD in Sudden Cardiac Death Prevention (PROSe-ICD)
The overall hypothesis of this study is that subtle interactions between structural (substrate) and functional (trigger) abnormalities of the heart, some of wh…
Risk Stratification of Ischemic and Non-ischemic Cardiomyopathies in Racial and Ethnic Minority Groups in the Bronx - Cardiovascular Multimodality Imaging Study
Determining the etiology of cardiomyopathy is of high clinical importance for optimal treatment strategy and prediction of prognosis. There is increased risk f…
The Utility of pGz in Primary Mitochondrial Disorders
This is a multi-aim study, studying the effects of conventional exercise (measured through Cardiopulomary Exercises Testing or an in-bed pedal exercise) and pa…
Multicenter Trial to Evaluate the Safety and Effectiveness of the Cardiohelp System for up to 30 Days of Support in Children With Severe Cardiac Failure
There are two primary goals of this multicenter clinical trial that combines an FDA device trial and a phase II drug trial in the same study cohort. These two …
An Open-label Phase 2a Study to Evaluate the Safety and Efficacy of AlloNK®, an Allogeneic Cord Blood-derived NK Cell Therapy, in Combination With Rituximab in Relapsing Forms of B-cell Dependent Rheumatologic Diseases
A Basket Trial of Refractory Rheumatoid Arthritis (RA), Sjögren's Disease (SjD), Idiopathic Inflammatory Myopathies (IIMs) and Systemic Sclerosis (SSc) subject…
A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque- like sub…
Photoplethysmography (PPG) to Predict Ejection Fraction and Other Echographic Data in the General Population
The investigators are aiming to investigate the association between ejection fraction (EF) determined by echocardiography and signals obtained from Photoplethy…
The Development of Minimally Invasive Nanosensor Technology to Quantify Mitochondrial Function in Human Muscle
Past mitochondrial disease treatment studies have been unsuccessful in determining treatment efficacy, and a major factor has been the lack of validated biomar…