Clinical Research Directory

Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM

This is a first-in-human, non-randomized, open-label study designed to evaluate the safety, tolerability, and pharmacodynamics (PD) of TN-201 in adult patients with symptomatic hypertrophic cardiomyopathy (HCM) caused by mutations in the MYBPC3 gene.

A Study of Artificial Intelligence ECG With ECG Devices to Detect Hypertrophic Cardiomyopathy Distinct From Athlete's

The purpose of this study is to evaluate the AI-ECG algorithm for HCM in detecting HCM and in differentiating it from athlete's using not only the standard 12-lead ECG, but also ECGs obtained with the Apple Watch and Alivecor KardiaMobile devices.

A Study to Assess the Real-World Effectiveness of Mavacamten in Adult Patients With Obstructive Hypertrophic Cardiomyopathy in China

The purpose of this study is to assess the effectiveness of mavacamten treatment in Chinese adults with symptomatic obstructive hypertrophic cardiomyopathy (HCM) in real-world clinical practice

Empagliflozin in Hypertrophic Cardiomyopathy

The proposed intervention will be administration of empagliflozin at a standard dose of 10 mg daily for a period of 12 months. Patients with diagnosed diabetes will be excluded from the study. Patients (n = 250) will be randomized in a double-blind fashion to empagliflozin or placebo group. The primary endpoint of the study will be the change in peak oxygen uptake (VO2 max) measured in a cardiopulmonary exercise test. VO2max is an objective indicator of physical performance and will be evaluated before and after empagliflozin or placebo treatment.

A Study of EDG-7500 in Adults With Hypertrophic Cardiomyopathy (CIRRUS-HCM)

This study is being conducted in order to understand the safety and effects of different doses of EDG-7500 as a single dose in adults with obstructive hypertrophic cardiomyopathy (oHCM) and as multiple doses in adults with obstructive or nonobstructive hypertrophic cardiomyopathy (nHCM).

A Prospective Incident Study of Arrhythmias in Hypertrophic Cardiomyopathy

The purpose of this research study is to understand more about various heart rhythms (electrical problems) in persons with hypertrophic cardiomyopathy with and without sleep apnea.

Institutional Registry of Rare Diseases

The goal of this observational study is to create a single macro registry system with data collection on common clinical features, grouping the different rare diseases (RD). Moreover, the specific goals are to generate an alert system for possible cases of RD with data from the electronic medical record, to describe the occurrence of RD in the evaluated population, to characterize the population, to describe patterns of diagnosis and treatment of RD present at the time, and to explore patient-reported outcomes.

Austrian Hypertrophic Cardiomyopathy Registry

The Austrian Hypertrophic Cardiomyopathy (HCM) Registry is a prospective, multicenter registry enrolling patients at multiple outpatient clinics across Austria including academic and non-academic centers. Patients will undergo a structured examination process including assessment for symptoms of HCM, past medical history, concomitant medication, family history and the presence of HCM-specific red flags. Furthermore, clinical data derived from electrocardiogram, echocardiography, laboratory analysis, and genetic testing will be collected focusing on a lean variable dictionary and, in addition, specific hypothesis-driven research parameters. All data are entered into an electronic case report form (eCRF) (Phoenix Clinical Trial Management System). In order to perform multicenter analyses, data can be extracted from the eCRF after approval by the steering committee.

Exercise Intolerance in Non-obstructive Hypertrophic Cardiomyopathy

The EXCITE-HCM study is a randomized, controlled, blinded clinical trial designed to evaluate the effect of moderate intensity exercise training versus usual physicial activity on the improvement of HCM-related symptoms and cardiac function. About 70 participants will be recruited and randomized on a 1:1 ratio to either moderate intensity training or usual physicial activity interventions. Patients will be followed during a period of 24 weeks and assesesments as physical examination, questionnaires, 12 lead ecg's, biomarker levels, echocardiogram, Cardiac Magnetic resonance, PET and CPET will be performed to evaluate their response to the intervention.

Mavacamten Pregnancy Surveillance Program

The purpose of this observational pregnancy safety study is to assess maternal, fetal, and infant outcomes after exposure to mavacamten at any time during pregnancy and/or breastfeeding.

A Clinical Study to Evaluate the Long-term Safety of HRS-1893 in Hypertrophic Cardiomyopathy

This study mainly evaluates the long-term safety of HRS-1893 in subjects with hypertrophic cardiomyopathy.

Cardiomyopathies and Heart Muscle Diseases: Cardiac Imaging in the Evaluation of Myocardial Fibrosis Transition

Heart scarring, also known as fibrosis, plays a major role in a lot of heart muscle abnormalities. These abnormalities of the heart muscle can lead to major issues such as symptoms of heart failure, dangerous heart rhythm disturbances and even death. However, a lot of these conditions are still not fully understood and treatment options are limited. We here aim to use a new radioactive dye called 68Ga-FAPI to identify patterns and the activity of heart muscle scarring. This radioactive dye is being used in humans particularly in identifying and monitoring cancers and has shown promise in identifying scarring in the heart as well. This will help us not only understand the underlying disease process and risk stratify these patients but also potentially help us develop new targeted therapies that can affect heart muscle scarring. Participants will undergo a baseline MRI scan using this new dye and a plain MRI scan will repeated 12-18 months after to see if there are any changes in the process.

VIZ ACCESS HCM - Multi-Site Registry

To describe the clinical, economic, and population characteristics of newly diagnosed, previously diagnosed, and suspected patients evaluated by Viz HCM. HCM is underdiagnosed in the community and AI algorithms have been developed as screening tools. However, it is not well understood how to best integrate AI screening tools and their potential impact.

Pediatric Cardiomyopathy Mutation Analysis

The goal of this protocol is to obtain information from individuals with cardiomyopathy and from their families in order to elucidate the molecular genetics of this disorder. This will provide the basis for future genetic counseling as well as contribute to elucidating the biology of normal and abnormal cardiac function.

Comparison of the Effects of Inspiratory Muscle Training and Baduanjin Exercises in Hypertrophic Cardiomyopathy Patients

The study aims to compare the effects of inspiratory muscle training and Baduanjin exercises on pulmonary function, exercise capacity, and quality of life in patients with hypertrophic cardiomyopathy. By investigating these interventions, the investigators seek to introduce novel approaches that can enhance pulmonary function, exercise capacity, and overall quality of life for these patients. In the study, which will involve three groups-the control group, the inspiratory muscle training (IMT) group, and the Baduanjin group-it was calculated that a total of 51 patients, with 17 in each group (n = 17), should be included. The IMT group will use an inspiratory muscle training device twice a day, every day of the week, for 15 minutes. This training will continue for 8 weeks, with supervision once a week and unsupervised sessions on the other days. The Baduanjin group will participate in a 50-minute exercise program, which includes a 10-minute warm-up, a 30-minute routine of eight separate movements, and a 10-minute cool-down. This will occur three times a week (twice in person and once online) for 8 weeks.

PRecIsion Medicine in CardiomyopathY

This is a retrospective cohort study of pediatric hypertrophic cardiomyopathy (HCM) patients using chart and registry review methodology. The studies objective is to develop and validate a sudden cardiac death (SCD) risk calculator that is age-appropriate for children with HCM that includes clinical and genetic factors.

Electromechanically Optimised Right Ventricular Pacing In Hypertrophic Cardiomyopathy (EMORI-HCM)

Hypertrophic Obstructive Cardiomyopathy (HOCM) is an inherited cardiac condition which causes the heart muscle to become abnormally thick causing obstruction of blood flow in the heart. This causes debilitating symptoms including shortness of breath, blackouts and chest pain. Current treatments are not ideal as the medication is often poorly tolerated or ineffective. People with HOCM can often have an Implantable Cardioverter Defibrillator (ICD) to shock them out of dangerous arrhythmias. ICD's can also be used as pacemakers and are a promising treatment option, since they can alter the sequence of the heart muscle contraction thereby relieving the obstruction to the blood flow, making it easier for the heart to pump. The study will recruit patients who already have an ICD/pacemaker or who are scheduled to have an ICD / pacemaker implanted. For patients who are due to have a device implanted high precision haemodynamic, echocardiographic and electrical measurement techniques will be used to assess whether adjusting the position of the pacing lead (at the time of implant) can bring about changes in LVOT gradient and blood pressure. These patients with a new device and also patients who already have a device in situ will then go on to have atrioventricular delay (AV Delay) optimisation so we can assess what the optimum AV delay should be programmed at in order to bring about the most improvement in LVOT gradient and blood pressure. Patients will then be recruited into a medium term double blinded randomised crossover study. They will have optimum RV pacing settings turned on for 3 months. They will then return and be crossed over and have optimum RV pacing turned off for a further 3 months. The primary outcome will be to see if optimum RV pacing being turned on is effective in improving symptoms and quality of life.

Prospective Evaluation Of Exercise-Induced Cardiac Conduction Instability In Predicting Ventricular Fibrillation Events In Hypertrophic Cardiomyopathy

Hypertrophic Cardiomyopathy (HCM) is an inherited heart condition. Most people who have it are unaware of any problems relating to it. Unfortunately, a small number of people with the condition can suddenly develop a dangerous fast heart beat that can lead to death. There is no cure, but implanting a cardioverter-defibrillator (ICD), which is like a pacemaker can save the life of affected individuals. However, ICD implantation has its own problems, so choosing who gets an ICD is a very important decision. The current approach for recommending people for an ICD has limitations and a better method is needed. Investigators have developed a new technique called the 'Ventricular Conduction Stability' (V-CoS). This involves wearing a special vest which records electrical signals from the heart, and then running on a treadmill. Investigators have used it to identify abnormalities in the hearts of people with HCM who have also survived a life-threatening event. This project aims to test new tool against current methods to ascertain which is better at identifying patients who should have an ICD.

Evaluation of the Efficacy and Safety of Wearable ECG (AT-Patch) in Patients With Hypertrophic Cardiomyopathy Requiring 48-Hour Holter MonitoringEvaluation of the Efficacy and Safety of Wearable ECG (AT-Patch) in Patients With Hypertrophic Cardiomyopathy Requiring 48-Hour Holter Monitoring

* Validation of validity, superiority, and safety of data analysis results using AT-Patch compared to 48-hour Holter test results * Number of subjects: 100 (including 10% dropout rate) * Performance and safety were verified by simultaneously attaching a 48-hour Holter and a wearable Holter device (ATP-C75 or ATP-C135).

A Retrospective Cohort Study of Mavacamten Patient Support Program in Canada

This real-world study will describe the demographic and clinical characteristics and mavacamten treatment of adult patients with obstructive hypertrophic cardiomyopathy who participated in the Bristol-Myers Squibb (BMS)-sponsored CAMZYOS Patient Support Program in Canada.

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 for cardiovascular disease and higher propensity for cardiovascular related mortality among Black and non-Hispanic White patients. Recently, advanced cardiac imaging has become a vital tool in diagnosis and risk stratification of cardiovascular disease. Very limited data is available on the prevalence and characteristics of different cardiovascular diseases in Hispanic and African American minority groups, therefore, studying different racial and ethnic minority groups in the Bronx population is an exceptionally valuable source to determine the prevalence of cardiomyopathies among minority groups along with study survival in this population. This study aims to determine the etiology of cardiovascular disease in a diverse patient population by utilizing various cardiovascular imaging modalities, with a focus on cardiac magnetic resonance (CMR) imaging and to develop risk stratification models by applying advanced cardiovascular imaging markers.

HCMR - Novel Markers of Prognosis in Hypertrophic Cardiomyopathy

Hypertrophic cardiomyopathy (HCM) is the most common monogenic heart disease and the most frequent cause of sudden cardiac death (SCD) in the young. It is characterized by unexplained left ventricular hypertrophy (LVH), diffuse and patchy fibrosis, and myofibrillar disarray. While the majority of patients remain asymptomatic, prognosis is poor in a subset who present with SCD or progress to heart failure (HF). Current methods to predict risk of these adverse events and to target therapy are limited. Current medical therapy does not protect against SCD, nor does it prevent development of HF. Therefore, the identification of novel risk markers would help develop therapeutic targets aimed at altering the phenotypic expression to impact the natural history, especially SCD and HF. Cardiovascular magnetic resonance (CMR) is emerging as a powerful tool for diagnosis and risk stratification in HCM including assessment of LV mass and pattern of hypertrophy. Late gadolinium enhancement by CMR is a marker of focal myocardial fibrosis which is thought to underlie the arrhythmogenic substrate as well as promote development of HF. The investigators hypothesize that HCM patients with a higher primary outcome event rate can be identified by novel CMR findings. The majority of cases of HCM are autosomal dominant and about 60% are caused by mutations in genes encoding cardiac sarcomeric proteins. However, the relationship between genetic mutation, disease phenotype, and clinical outcomes remains poorly understood. The investigators hypothesize that HCM patients with sarcomeric HCM mutations will have a higher primary outcome event rate and more marked myocardial pathology on CMR than those without. Furthermore, there may be a link between sarcomeric mutations and fibrosis, as mutation carriers with overt HCM as well as those without hypertrophy have elevated markers of collagen turnover. The investigators therefore hypothesize that serum biomarkers of collagen metabolism in HCM will predict outcomes. Thus, the Specific Aim is to develop a predictive model of cardiovascular outcomes in HCM by: 1) using exploratory data mining methods to identify demographic, clinical, and novel CMR, genetic and biomarker variables associated with the outcomes and 2) develop a score from the predictive model that can be used to assess risk given a patient's combination of risk factors, thus establishing the evidence base to enable clinical trial design to reduce morbidity and mortality in HCM in a cost-effective manner.

A 10-Minute Cardiovascular Magnetic Resonance Protocol for Cardiac Disease

This study aims to identify and assess new CMR techniques that can improve current CMR protocols.

Hypertrophic Cardiomyopathy Federated Learning Implementation Platform

HCM FLIP study is a two-phase protocol focusing on the detection of Hypertrophic Cardiomyopathy using Electrocardiograms and Echocardiograms through Federated Learning.