Heart failure clinical trials in Boston

Researchers are looking for a better way to treat children and young adults who have heart failure with left ventricular systolic dysfunction (LVSD). Heart failure with left ventricular systolic dysfunction (LVSD) is a condition where the left side of the heart is weak and struggles to pump blood effectively, leading to symptoms like shortness of breath, fatigue, and poor growth. The study treatment, finerenone (also called BAY94-8862), is under development to treat newborns, children, and young adults with heart failure and LVSD. It works by blocking a protein that contributes to inflammation, scarring, and thickening in the heart and blood vessels, which may help the heart pump more blood effectively. The main purpose of this study is to learn about how safe finerenone is and how well it works in the long-term treatment of heart failure and LVSD. To understand how safe the treatment is, the study team will gather information on the number of patients who experience medical problems after taking finerenone, also known as "treatment emergent adverse events" (TEAEs). Additionally, they will collect blood samples to measure levels of an electrolyte called potassium and monitor blood pressure. They will also assess kidneys function using the estimated glomerular filtration rate (eGFR). In this study, which is an extension of the earlier done FIORE study, finerenone will also be studied in newly enrolled newborns under 6 months with heart failure and LVSD and children and young adults from the FIORE study. The participants will be aged from newborns up to 18 years. All the participants will continue to receive their standard treatment as routine care for heart failure, along with finerenone during the study. The participants will be in the study for around 10 to 11 months, depending on whether they rolled-over from the FIORE study or are newly enrolled newborns and infants \<6 months of age. They will take study treatment for up to 9 months. During this period, at least 6 visits are planned for participants. During these visits, the study team will: * have their blood pressure, heart rate, temperature, respiratory rate, height and weight measured * have blood samples taken * have physical examinations * have their heart examined by an electrocardiogram and echocardiography * answer questions about their medication and whether they have any adverse events, or have their parents or guardians' answer * for newborns and infants, evaluate the acceptability of the study drug formulation through parents or guardians' feedback. An adverse event is any medical problem that a participant has during a study. Doctors keep track of all adverse events that happen in studies, even if they do not think the adverse events might be related to the study treatments. The doctors will check the participants' health a month after the participants take their last treatment.

The goal of this clinical trial is to learn if therapy with activin ligand-trap biological therapy (an investigational drug) combined with exercise training can improve exercise capacity and cognitive function in heart failure with preserved ejection fraction (HFpEF). The main questions it aims to answer are: * Does activin-ligand trap biological therapy improve exercise capacity as measured by change in peak oxygen uptake (peak VO2) from baseline to week 12? * Does activin-ligand trap biological therapy improve cognitive function as assessed by the NIH-Toolbox Cognition Battery (NIHTB-CB) composite score and Rey Auditory Verbal Learning Test (RAVLT) from baseline to week 12? Researchers will compare activin-ligand trap biological therapy to a placebo (a look-alike substance that contains no drug) to see if activin-ligand trap therapy works to improve exercise capacity and cognitive function in patients with HFpEF.

This multicenter pilot study will enroll adult participants with a confirmed clinical diagnosis of transthyretin amyloid cardiomyopathy (ATTR-CM) and heart failure (HF) controls with documentation within the past 2 years that either excludes ATTR-CM or indicates a low probability of ATTR-CM. Ocular imaging and other study data will be collected to assess the feasibility of developing and preliminarily evaluating a machine learning model to discriminate ATTR-CM cases from HF controls without ATTR-CM.

Severe skeletal wasting and catabolic weight loss are highly common among patients with heart failure with reduced ejection fraction (HFrEF). This prospective randomized controlled trial will compare changes in the muscle mass in the arms and the legs (appendicular lean mass) in patients with HFrEF randomized between 3 groups of no, low- or high-dose protein supplementation. The dietary protein supplementation will be Ensure(R) products manufactured by Abbott Nutrition. The Investigators hypothesize that skeletal muscle wasting in HFrEF is promoted by neurohumoral activation of catabolic metabolism (such as GDF-15 and ActRII pathways) and can be at least partially reversed by increased dietary protein intake. It is anticipated that this study will determine whether dietary protein supplementation helps to prevent muscle wasting and will advance understanding of the GDF-15 and ActRII muscle wasting pathways.

This is a multicenter, non-interventional study to observe the natural progression of the disease and to study the prevalence of pre-existing antibodies to AAV9 used for gene therapy in a population of patients with PKP2 gene-associated ARVC. Participation from all patients is encouraged regardless of interest in or eligibility for gene therapy.

The overall goal of this PET-MR imaging trial is to evaluate 11C-Martinostat, a histone deacetylase targeted radioligand, in patients with aortic stenosis, individuals with diabetes, and healthy volunteers.

This first-in-human study is designed to evaluate the safety, and preliminary efficacy (PD) of TN-401 gene therapy in adult patients with symptomatic PKP2 mutation-associated ARVC.

This registry evaluates patient characteristics, real-world treatment patterns, and short- and long-term outcomes in a population of patients in the United States and Europe with symptomatic obstructive hypertrophic cardiomyopathy (HCM) who are receiving mavacamten, receiving other treatment for obstructive HCM, or not receiving treatment for obstructive HCM due to intolerance or failure of prior treatment. United States Sub-Study: The purpose of this study is to evaluate the safety of mavacamten in patients with symptomatic obstructive HCM in the real-world setting. Europe Sub-Study: The purpose of this study is to evaluate the effectiveness and safety of mavacamten in patients with symptomatic obstructive HCM in the real-world setting.

The focus of this study is to test the efficacy of a 12-week, phone-delivered Positive Psychology-Motivational Interviewing (PP-MI) intervention, with additional twice weekly PP and health behavior text messages for a total of 24 weeks (with interactive, algorithm-driven, goal-focused text messages in the final 12 weeks), compared to an attention-matched MI-based educational condition, in a randomized trial (NIH Stage II) of 280 patients with New York Heart Association class I-III Heart Failure (HF).

The goal of this observational study is to measure shear wave velocity (SWV) in patients with non-reduced left ventricular ejection fraction (LVEF) heart failure and left ventricular hypertrophy, with or without transthyretin amyloidosis with cardiomyopathy (ATTR-CM), and in control subjects without heart failure or cardiomyopathy. The main question it aims to answer is: • Can SWV be used to measure myocardial and liver stiffness in the study's target populations? Researchers will compare patients with ATTR-CM, patients without ATTR-CM, and a control group to determine the distributions of SWV in each population. Participants will: * Have a standard cardiology assessment, including a physical exam, blood work, and an echocardiogram. * Undergo an investigational assessment with the eMyosound LYRA device to measure SWV in their myocardium and liver. * Have the investigational assessment repeated by a second observer to assess measurement reliability.

In the United States, the current standard of prehospital (i.e. outside of hospitals) emergency care for children with life-threatening illnesses in the community includes remote physician support for paramedics providing life-saving therapy while transporting the child to the hospital. Most prehospital emergency medical services (EMS) agencies use radio-based (audio only) communication between paramedics and physicians to augment this care. However, this communication strategy is inherently limited as the remote physician cannot visualize the patient for accurate assessment and to direct treatment. The purpose of this pilot randomized controlled trial (RCT) is to evaluate whether use of a 2-way audiovisual connection with a pediatric emergency medicine expert (intervention = "telemedical support") will improve the quality of care provided by paramedics to infant simulator mannequins with life threatening illness (respiratory failure). Paramedics receiving real-time telemedical support by a pediatric expert may provide better care due to decreased cognitive burden, critical action checking, protocol verification, and error correction. Because real pediatric life-threatening illnesses are rare, high stakes events and involve a vulnerable population (children), this RCT will test the effect of the intervention on paramedic performance in simulated cases of pediatric medical emergencies. The two specific aims for this research are: * Aim 1: To test the intervention efficacy by determining if there is a measurable difference in the frequency of serious safety events between study groups * Aim 2: To compare two safety event detection methods, medical record review, and video review

This study aims to determine, via skeletal muscle ultrasound (US), the extent, timing and relationship between skeletal muscle mass loss and outcomes after orthotropic heart transplantation (OHT) and left ventricular assist device (LVAD) implantation amongst patients with cardiogenic shock. Advanced therapies such as OHT and VADs in the heart failure (HF) population may promote skeletal muscle mass and subsequent quality of life, but there is a lack of literature assessing muscle mass changes in HF patients before and after advanced therapies using US imaging. Therefore this observational study will provide further insight into the 1) changes in lean body mass during critical illness and 2) the feasibility of using bedside US to assess lean body mass in the inpatient setting.

This is a Phase 2 dose-finding study in adult participants with symptomatic HFpEF.

Participants include men and women ≥ 40 years of age with T2DM, established CV disease, a history of HTN with an SBP of at least 130 mmHg at screening, who meet the predefined serum potassium level, and with at least one additional risk factor for HF. The study will include an optional pre-screening period to facilitate sites' identification of potentially eligible participants to enter the full screening assessments. Participants will not be required to visit the site and no informed consent is required for the optional pre-screening period. The pre-screening assessments do not replace the full screening tests at Visit 1. Upon entering the screening period, all consented participants (after signature of screening ICF) will be screened during an up to 14-day screening period. Participants who meet all screening inclusion/exclusion criteria but are not treated with SGLT2i or are treated for less than 4 weeks will enter a run-in period with dapagliflozin 10 mg once daily for at least 4 weeks (and not more than 6 weeks) before randomisation. Site visits will take place at approximately 2-, 4-, 8-, 16-, and 34-weeks following randomisation. Thereafter visits will occur approximately every 4 months. The study closure procedures will be initiated when the predetermined number of the first secondary endpoint events (ie, the composite of hospitalisation for HF or CV death) is predicted to have occurred i.e., the PACD. In case of premature discontinuation of the blinded study intervention, participants will remain in the study. Unless a participant meets the dapagliflozin specific discontinuation criteria, they will continue to receive open label dapagliflozin 10 mg. It is important that the scheduled study visits and data collection continue according to the study protocol.

The purpose of this study is to demonstrate the efficacy and evaluate the safety and tolerability of mavorixafor in participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are experiencing recurrent and/or serious infections as assessed by demonstrating its clinical benefit and increasing levels of circulating neutrophils.

The purpose of this study is to assess the efficacy and safety of the study drug, levosimendan (given orally), compared to placebo in participants with pulmonary hypertension with heart failure with preserved left ventricular ejection fraction (PH-HFpEF) as measured by the change in 6-Minute Walk Distance.

This study aims to compare the efficacy of vericiguat versus placebo on change in n-terminal pro-brain natriuretic peptide (NTproBNP) from baseline to Week 16 of the Base Period. The primary hypothesis is that vericiguat is superior to placebo in reducing NT-proBNP at Week 16 of the Base Period.

This study aims to develop and refine a microRNA (miR) biomarker panel that can be used to phenotype net immune state after heart transplantation using circulating miRs (associated with drug doses and levels). These miRs will be used to characterize the overall immune state in adult heart transplant patients and predict patients that will go on to develop infection and rejection. MicroRNAs (miRs) are small, non-coding RNA molecules that regulate gene expression and serve as molecular biomarkers found in the circulation.

ELEVATE-HFpEF is a prospective, randomized, controlled, double-blinded, multi-center, global, interventional pivotal study evaluating the safety and efficacy of dual chamber personalized pacing compared to minimal or no pacing for the treatment of patients with heart failure with preserved ejection fraction (HFpEF).

This study will find out if a new medicine called NNC6019-0001 can help reduce the risk of heart-related death and illness in participants with a condition called transthyretin amyloid cardiomyopathy (ATTR-CM), which affects the heart. Participants will either receive NNC6019-0001 or a placebo (a treatment with no active medicine), and which one they get is decided by chance. Everyone in the study will continue receiving their usual heart treatments as recommended by their doctor.