25 Clinical Trials for Various Conditions
PumpKIN is a multicenter, prospective, single-arm feasibility study; Evaluating the investigational Jarvik 2015 VAD in pediatric patients with heart failure. This feasibility trial will enroll 10 subjects at up to 7 sites in the US. The primary objectives of this investigational device exemption (IDE) clinical investigation are to assess the feasibility of using the Jarvik 2015 in pediatric patients with severe heart failure who require mechanical circulatory support. Feasibility will be assessed by evaluating the safety profile of the Jarvik 2015 device in eligible subjects.
This study consists of two parts (Part 1 and Part 2). The purpose of Part 1 is to evaluate the way the body absorbs, distributes, metabolizes and removes the drug LCZ696. This will help determine the proper dose of LCZ696 for Part 2 of the study. The purpose for Part 2 is to compare the effectiveness and safety of LCZ696 with enalapril in a double-blind manner, in pediatric heart failure patients over 52 weeks of treatment.
To collect information on pediatric patients receiving Cardiac Resynchronization Therapy (CRT) with permanent biventricular pacing (BVP) as a method of treating chronic heart failure.
The primary objective of this study is to monitor the safety and tolerability of vericiguat.
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 is a single-center, non-randomized study.The study staff will use the InSpectraTM tissue oxygen saturation (StO2) monitor manufactured by Hutchinson Technology to measure baseline StO2 levels after applying the noninvasive probe to the thenar eminence. After a stable reading is obtained, a blood pressure cuff will be inflated 40 mmHg above the obtained systolic pressure and the rate of desaturation (Rdes; % × sec-1) will be recorded. After 3 minutes or once the StO2 level comes to zero (whichever is earlier), the cuff pressure will be released and the rate of reperfusion (Rres; % × sec-1) will be measured. The investigators hypothesize that heart failure in children causes a baseline lower thenar tissue oxygen saturation (StO2), a faster rate of desaturation (Rdes) and a prolonged rate of reperfusion (Rres). The investigators also hypothesize that these changes will correlate with the severity of heart failure. The results of this study will provide groundwork for studies looking at correlation of therapy modification based on the combination thenar StO2 and clinical presentation.
The purpose of the study was to evaluate the safety, tolerability and pharmacokinetics of an intravenous infusion of serelaxin on top of standard of care therapy, in pediatric patients with acute heart failure (AHF)
Stress hyperglycemia, a state of abnormal metabolism with supra-normal blood glucose levels, is often seen in critically ill patients. Tight glycemic control (TGC) was originally shown to reduce morbidity and mortality in a landmark randomized clinical trial (RCT) of adult critically ill surgical patients but has since come under intense scrutiny due to conflicting results in recent adult trials. One pediatric RCT has been published to date that demonstrated survival benefit but was complicated by an unacceptably high rate of severe hypoglycemia. The Heart And Lung Failure - Pediatric INsulin Titration (HALF-PINT) trial is a multi-center, randomized clinical treatment trial comparing two ranges of glucose control in hyperglycemic critically ill children with heart and/or lung failure. Both target ranges of glucose control fall within the range of "usual care" for critically ill children managed in pediatric intensive care units. The purpose of the study is to determine the comparative effectiveness of tight glycemic control to a target range of 80-110 mg/dL (TGC-1, 4.4-6.1 mmol/L) vs. a target range of 150-180 mg/dL (TGC-2, 8.3-10.0 mmol/L) on hospital mortality and intensive care unit (ICU) length of stay (LOS) in hyperglycemic critically ill children with cardiovascular and/or respiratory failure. This will be accomplished using an explicit insulin titration algorithm and continuous glucose monitoring to safely achieve these glucose targets. Both groups will receive identical standardized intravenous glucose at an age-appropriate rate in order to provide basal calories and mitigate hypoglycemia. Insulin infusions will be titrated with an explicit algorithm combined with continuous glucose monitoring using a protocol that has been safely implemented in 490 critically ill infants and children.
The goal of this clinical trial is to explore the impact that an at-home cardio-oncology rehabilitation (CORE) may have on short-term cardiovascular fitness and psychosocial wellness in pediatric cancer survivors. The main question it aims to answer are * To evaluate the efficacy of an at-home CORE model on short-term cardiovascular fitness and psychosocial wellbeing in adolescent cancer survivors. * To evaluate the exercise adherence rate among adolescents at risk for heart failure and assess barriers to compliance. * To explore which specific CORE resources are of most value to patients in creating sustainable healthy lifestyle modifications. * Hypothesis: Pediatric cancer survivors who implement exercise and dietary recommendations will demonstrate improvement in cardiovascular fitness and general wellness. A multidisciplinary team approach can facilitate adherence to a moderately rigorous exercise prescription, and thus enhance the health benefits of a CORE program at CHLA. Participants will undergo cardiovascular studies and a quality-of-life survey prior to exercise intervention, and at the end of the 6-month study period.
This study aims to discover circulating microRNAs (associated with drug doses and levels) that can be used to characterize the overall immune state in pediatric 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.
The purpose of this study is to evaluate the device performance and monitor the safety and effectiveness of the Berlin Heart EXCOR Active Driving System while being used with the approved EXCOR Pediatric Ventricular Assist Device. EXCOR Active Driving System is intended for use with the approved EXCOR Pediatric VAD. The EXCOR Pediatric VAD is intended to provide mechanical circulatory support as a bridge to cardiac transplantation for pediatric patients. Pediatric candidates with severe isolated left ventricular or biventricular dysfunction who are candidates for cardiac transplant and require circulatory support may be treated using the EXCOR Pediatric. EXCOR Active is intended for use in a clinical setting. EXCOR Active can be used in any kind of hospital unit (e.g. OR, ICU, intermediate care unit or general care unit). The driving unit may be moved between clinical units using the caddy or baby buggy; however, a patient must always be accompanied by a person trained in the use of the manual pump and emergency procedures during transport in the event of an emergency. The driving unit can be transported during operation.
Immunosuppressive therapy is required to prevent organ rejection, however, dosing of immunosuppressive agents is complicated by patient-specific differences impacting the body's absorption and elimination of these agents. The goal of this research proposal is to clinically validate an innovative precision medicine strategy for dosing the immunosuppressant tacrolimus in pediatric heart transplant, which will in turn lead to improvements in long-term transplant survival outcomes. The strategy and techniques used in this project can be extended to improve drug therapy across multiple pediatric diseases requiring chronic therapy.
The purpose of this study was to evaluate long-term safety and tolerability and to provide post-trial access to sacubitril/valsartan to eligible participants who successfully completed CLCZ696B2319 (PANORAMA-HF) core study Part 2 as per protocol.
The TEAMMATE Trial will enroll 210 pediatric heart transplant patients from 25 centers at 6 months post-transplant and follow each patient for 2.5 years. Half of the participants will receive everolimus and low-dose tacrolimus and the other half will receive tacrolimus and mycophenolate mofetil. The trial will determine which treatment is better at reducing the cumulative risk of coronary artery vasculopathy, chronic kidney disease and biopsy proven-acute cellular rejection without an increase in graft loss due to all causes (e.g. infection, PTLD, antibody mediated rejection).
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.
Project Summary We propose an ancillary study to The Heart and Lung Failure Pediatric Insulin Titration trial (HALF PINT), which is investigating the impact of normalizing blood glucose using insulin infusions on clinical outcomes among children with hyperglycemia and heart and lung failure. In this ancillary study, we will measure plasma levels of inflammatory, coagulation, and fibrinolysis proteins and genotype DNA for polymorphisms among patients enrolled in the HALF PINT trial. The results from this ancillary study will help us to understand potential mechanisms through which normalizing blood glucose provides benefit, which may lead to development of new therapeutic strategies in critically ill children
It is routine practice for a cardiologist to perform a battery of tests to assess the degree of heart failure. The purpose of this study is to establish a non-invasive method to reliably predict cardiac output state in real-time in children and adolescents with heart failure in an outpatient setting. This study will rely on the use of near infrared spectroscopy monitors to measure cardiac output in the outpatient setting.
The purpose of the study is to determine whether use of the Berlin Heart EXCOR® Pediatric Ventricular Assist Device for bridge-to-transplant is associated with a reasonable assurance of safety and probable benefit such that the EXCOR® Pediatric merits approval by the Food and Drug Administration under a Humanitarian Device Exception (HDE).
Palliative care in pediatric oncology and neurology is well described in the literature. There is a lack of information on the care of children terminally ill due to heart failure. A significant number of children are diagnosed with terminal heart failure for which palliative care is required. Objective: To describe palliative care of children over one year of age with end-stage heart failure, including need for and types of pain control, hospice use, need for and use of home nursing and quality of life issues.
This study will evaluate the efficacy and safety of administering an angiotensin converting enzyme inhibitor (ACE-I) (enalapril) to infants with a functional single ventricle. The study will also compare the effect of ACE-I therapy to placebo on somatic growth and compare the effect of ACE-I therapy to placebo on signs and symptoms of heart failure, neurodevelopmental and functional status, ventricular geometry, function, and atrioventricular (AV) valve regurgitation. In addition, the study will determine the relationship between genetic polymorphisms linked to ventricular hypertrophy (enlarged heart) and the response to ACE-I therapy and compare the incidence of adverse events in subjects treated with ACE-I with those in subjects treated with placebo.
Anthracycline chemotherapies (e.g. doxorubicin, daunorubicin) are commonly given to treat pediatric cancer, and carry a risk of cardiotoxicity. Over the long term, children who receive these therapies have an increased risk of heart failure and early cardiovascular death. However, current strategies for identifying patients who are at risk prior to the development of significant changes in heart function are limited. This study will focus on imaging markers of cardiac injury and dysfunction with the goal of developing improved diagnostic tests and treatment strategies.
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDLC).
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to \<12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDLC).
Inotropic therapy is a well-established practice for children with advanced congestive heart failure (CHF). Inotropes are intravenous medications to help the heart pump stronger and prolong life while awaiting heart transplantation. Traditionally children have been maintained on inotropic therapy in the hospital under close, monitored supervision. The United Network for Organ Sharing (UNOS), which governs organ distribution, has now changed listing criteria allowing patients awaiting heart transplantation to be discharged to home, yet maintaining a higher status on the waiting list. In adults, home inotropic therapy has been shown to be a safe and cost-effective bridge to transplantation. To date, there are no data on the use of home inotropic therapy in children. Hypothesis: We request to do a retrospective chart review of patients receiving this therapy to determine safety and efficacy of continuous ambulatory home inotropic therapy in children.
One of the risks associated with heart transplantation is failure of the graft. A graft is where the new heart is attached to the original vessels in the body. Approximately 10% of children suffer graft failure which, leads to heart failure and possible death. The problem is that we do not know some of the causes of graft failure thus, it is difficult to diagnose early and treat. Due to graft failure, lots of children are placed back on the transplant list and receive another new heart. In this study, we plan to perform a retrospective chart review looking to see if we can correlate graft failure with a problem with the vessels called coronary allograft vasculopathy or rejection. In order to do this, we will collect data from patient's charts that have been diagnosed with graft failure and compare their clinical presentation/data to pathology reports of the explanted hearts from these children. Explanted hearts are the old transplanted hearts that are removed in order to put a new heart into the body. Explanted hearts at our institution are always sent to pathology for analysis thus it will be quite easy to perform this review.