34 Clinical Trials for Various Conditions
The purpose of this study is to find out more about the effectiveness of Tadalafil in improving hemodynamic capabilities, endothelial function, and end-organ function in patients who have previously undergone a Fontan Palliation.
This is a prospective international multi-center registry and biorepository trial of children and adolescents/young adults (AYA) undergoing hematopoietic cell transplantation (HCT) to assess the impact of endotheliopathies in the HCT setting as a contributor of significant morbidity and mortality.
This multi-center prospective intervention study is designed to develop coenzyme Q10 (CoQ10) supplementation as a cost-effective adjunctive therapy for burn injury. The long-term goals of this project are to establish the beneficial effects of CoQ10 on multiple organ dysfunction and on the clinical and functional outcomes of burn victims.
Hematopoietic stem cell transplantation (HCT)-associated thrombotic microangiopathy (TMA) is an understudied complication of HCT that significantly affects transplant related morbidity and mortality. The investigators hypothesize that early intervention with complement blocker eculizumab will double survival in HCT recipients with high risk TMA, as compared to historical untreated controls. An optimal eculizumab dosing schedule can be determined for this population through eculizumab pharmacokinetic/pharmacodynamic (PK/PD) testing.
The TRIPS study is a prospective, multi-center, double-blind, adaptively randomized, placebo-controlled clinical trial of the drug anakinra for reversal of moderate to severe hyperinflammation in children with sepsis-induced multiple organ dysfunction syndrome (MODS).
The GRACE-2 study is a prospective, multi-center, double-blind, randomized controlled trial of the drug GM-CSF vs placebo in children with sepsis-induced multiple organ dysfunction syndrome (MODS) who have immunoparalysis with mild to moderate inflammation.
This study will try to determine what causes toxic side effects of stem cell transplantation, such as increased blood pressure, increased heart rate, decreased kidney function and abnormal heart rhythms. Stem cells are used to treat various diseases, including cancer, aplastic anemia and sickle cell disease. The cells may be given fresh to the patient or they may be preserved first with a chemical called DMSO and frozen for later use. Some stem cell transplant procedures include infusion of red blood cells along with the stem cells. This study will examine whether side effects of stem cell transplants are associated with the DMSO preservative in frozen cells or with hemoglobin (a protein released from defrosted red blood cells) or neither of these factors. Healthy volunteers and patients scheduled to receive a stem cell transplant may be eligible for this study. Candidates must be between 10 and 80 years of age. Transplant patients will undergo a stem cell transplant. The cells are infused through a catheter placed in a vein for the procedure. Depending on the patient s requirements, the infusion may or may not include red blood cells and may or may not contain DMSO. Healthy volunteers undergo a 4-hour saline infusion. The saline (water mixed with salt) is infused through a catheter (plastic tube) placed in a vein in the arm. In addition, all participants have the following tests and procedures: * Heart monitoring: Healthy volunteers wear a portable heart monitor, attached to the chest using four stickers, for 24 hours starting the morning of the infusion. Transplant patients wear the same device for 48 hours, starting the morning before the infusion. * Blood draws and urine collections before, during, just after and the morning after the infusion of saline or stem cells. * Heart ultrasound before, during or just after and the morning after the infusion. * Peripheral artery tonometry: A small cup is placed on one finger of each hand to measure blood flow in the finger. A blood pressure cuff is inflated around the lower arm and tight pressure is maintained for about 5 minutes.
The goal of this observational study is to learn about how transposable element levels affect the patient outcomes in sepsis. The main questions it aims to answer is: 1. Do transposable elements prematurely age the immune systems of patients with sepsis? 2. Do transposable elements correlate with increased mortality in patients with sepsis in the ICU? 3. Do transposable elements correlate with increased amount of secondary infections in patients with sepsis in the ICU? Participants will have blood drawn from them but will not be subjected to additional interventions.
Severe acute respiratory syndrome coronavirus 2-mediated coronavirus disease (COVID-19) is an evolutionarily unprecedented natural experiment that causes major changes to the host immune system. We propose to develop a test that accurately predicts short- and long-term (within one-year) outcomes in hospitalized COVID-19 patients broadly reflecting US demographics who are at increased risk of adverse outcomes from COVID-19 using both clinical and molecular data. We will enroll patients from a hospitalized civilian population in one of the country's largest metropolitan areas and a representative National Veteran's population.
The purpose of this prospective, non-interventional, multi-centre clinical study is to assess the clinical validity of the Heparin Binding Protein (HBP) assay for indicating the presence, or outcome, of severe sepsis (including septic shock), over 72 hours, in patients with suspected infection following emergency department admission.
Pharmacology of Exenatide in Pediatric Sepsis, PEPS is a phase 1-2 research study that will examine drug safety, drug metabolism, drug action and preliminary drug clinical effects of four does of exenatide injected every 12 hours to children with shock from infection (septic shock). The investigators hypothesize that exenatide can be safely dosed to children with sepsis to achieve blood levels of drug similar to that achieved in teenagers with type 2 diabetes. The investigators further hypothesize that injection of exenatide to children with septic shock will normalize blood glucose levels and decrease levels of inflammation proteins in the blood during the early course of sepsis.
A Phase 2, multi-center, randomized, controlled, open-label study to evaluate the effects of the intraperitoneal, liposomal formulation VS-01 in patients with an acute episode of hepatic and/or extrahepatic organ dysfunctions and failures in the presence of liver cirrhosis (Acute-on-Chronic Liver Failure, ACLF) and accumulation of fluid in the abdominal cavity (ascites)
Mitochondria are organelles (a specialized subunit of a cell) responsible for providing cells with energy. For reasons not yet understood, mitochondria will release their DNA into blood in response to cellular injury or cell death. With a simple blood draw, investigators can measure the amount of mitochondrial DNA in a patient's blood. The investigators' hypothesis, is that mitochondrial DNA can be used as a surrogate marker of cellular injury to predict patient outcomes. The investigators intend to test their hypothesis by measuring mitochondrial DNA in adult patients presenting to the Emergency Department with sepsis (a life-threatening condition due to an infection) and observing their hospital course.
Approximately 40,000 infants are born each year in the United States with congenital heart defects (CHD), and heart defects are the leading cause of birth defect-related deaths in the United States. While advances in surgical treatment, cardiac bypass, and post-operative management have improved mortality for children born with heart defects, these children continue to have significant morbidity related to post-operative malnutrition, multiple organ dysfunction (MODS), and sepsis. Proposed mechanisms for post-operative sepsis and MODS is via loss of intestinal epithelial barrier function (EBF) or intestinal micro biome diversity. The purpose of this multi-center observational cohort study is to understand the extent to which practice variation for routine post-operative care might worsen intestinal barrier dysfunction and reduce diversity of the intestinal microbiome for infants undergoing surgical correction of left sided cardiac obstructive defects. We will enroll 80 children with left sided obstructive congenital cardiac lesions across several US congenital cardiac centers to obtain clinical data and biological specimens. We will leverage existing differences in nutritional and antibiotic strategies at these centers to better understand how intestinal barrier function and the intestinal microbiome may contribute to post-operative multiple organ dysfunction syndrome.
Predict the development of multiple organ failure in patients with trauma and severe burns.
Ventilated ICU patients frequently have sepsis and the majority have delirium, a form of brain dysfunction that is an independent predictor of increased risk of dying, length of stay, costs, and prolonged cognitive impairment in survivors. Universally prescribed sedative medications-the GABA-ergic benzodiazepines-worsen this brain organ dysfunction. The available alternative sedation regimens, the shorter acting GABA-ergic propofol, and the alpha2 agonist, dexmedetomidine, have both been shown to be superior to benzodiazepines, and yet are different with regard to their effects on innate immunity, bacterial clearance, apoptosis, cognition and delirium. The MENDS2 study will compare propofol and dexmedetomidine, and determine the best sedative medication to reduce delirium and improve survival and long-term brain function in our most vulnerable patients- the ventilated septic patient.
Septic shock is in critically ill patients is a condition associated with a high rate of organ failure and hereto attributable mortality \~45-55% Hypothesis: Mild Induced Hypothermia reduces the mortality of critically ill patients with septic shock by reducing organ metabolism, counteracting on microcirculatory thrombosis, genetically downregulating tissue apoptosis and by reducing bacterial growth rate and toxin production.
Sepsis represents the leading cause of childhood mortality worldwide. However, as distinct from adult medicine, there exists a large knowledge gap regarding long term health related quality of life (HRQL) and functional status (FS) following pediatric sepsis. This lack of sepsis outcomes data is critical because failure to identify children at risk for sepsis associated HRQL/FS deterioration may delay delivery of crucial rehabilitation medicine efforts to facilitate recovery. Moreover, failure to identify mechanisms of sepsis associated HRQL/FS deterioration may impede development of novel, effective interventions for these children. For the first time the LAPSE investigation will quantify deterioration of HRQL/FS among children surviving sepsis. We will measure the incidence, magnitude and duration of HRQL/FS alterations associated with pediatric septic shock, and examine clinical, sociodemographic, and parent/family factors potentially associated with such adverse outcomes. Because sepsis affects a heterogeneous group of children, long term morbidity associated with sepsis likely depends on premorbid health status and parent, family and home characteristics, as well as children's clinical course during sepsis critical illness. Mechanisms underlying adverse sepsis outcomes among children are poorly understood at this time. Clinically multiple organ dysfunction syndrome (MODS) has been clearly linked to sepsis mortality. To begin to understand pathophysiology underlying pediatric sepsis morbidity, this investigation will seek to identify evidence for association of HRQL/FS alterations following sepsis with intensity and duration of sepsis mediated organ dysfunction as well as with pre-existing comorbidities and parent, family, and home characteristics. The long-term goal of this research program is to timely identify children at high risk of sepsis mediated HRQL/FS deterioration and ultimately to design effective interventions to minimize such risk. The primary objectives of this investigation are to comprehensively characterize HRQL/FS trajectory and to critically examine the potential role of sepsis mediated organ dysfunction as well as pre-existing comorbidities and parent, family, and home characteristics as risk factors for the adverse outcomes. The central hypothesis is that intensity of sepsis organ dysfunction will predict magnitude of HRQL/FS deterioration. We also hypothesize that the trajectory towards baseline HRQL/FS following the sepsis event will also depend on pre-existing co-morbidities and parent, family, and home, and characteristics. Knowledge of these potential mechanisms will ultimately facilitate development of targeted interventions to maximize HRQL/FS among children surviving sepsis.
The ProCESS study is large, 5-year, multicenter study of alternative resuscitation strategies for septic shock. The study hypothesizes that there are "golden hours" in the initial management of septic shock where prompt, rigorous, standardized care can improve clinical outcomes.
Cancer patients with liver or renal dysfunction will be treated with rubitecan capsules to define the maximum tolerated dose and the dose-limiting toxicity in this patient population, and to perform pharmacokinetic studies of rubitecan in this patient population.
The goal of this mechanistic clinical trial is to learn about the effects of medications called soluble guanylyl cyclase stimulators on vascular function and markers of kidney and brain injury in patients having heart surgery. The main questions it aims to answer are: 1. Does soluble guanylyl cyclase stimulation improve blood vessel function compared to placebo? 2. Does soluble guanylyl cyclase stimulation decrease markers of kidney injury and brain injury compared to placebo? Participants will be randomized to a soluble guanylyl cyclase stimulator called vericiguat or placebo, and researchers will compare vascular function and markers of brain and kidney injury to see if vericiguat improves vascular function and reduces markers of injury. This will provide important information to determine the underlying reasons that patients have some kidney and brain function problems after having heart surgery.
The purpose of the study is to continue to follow subjects who were enrolled in the CTOT-20 CLAD Phenotypes study. Subjects will provide clinical data and complete quality of life questionnaires that will be used to determine the clinical factors associated with the development of Chronic Lung Allograft Dysfunction (CLAD) after lung transplant.
Potential therapy with MACITENTAN in the treatment of Chronic Lung Allograft Dysfunction (CLAD) after Lung Transplantation. Pilot Study, Double-blind, "ADD-ON Therapy" with MACITENTAN to "usual standard of care immunosuppressive therapies" after lung transplantation for established BOS Stages I or II versus a "matched control group" who receive "usual standard of care immunosuppressive therapies" alone, results in a decrease in the Primary Endpoint: "rate of decline" in "Forced Expiratory Volume-1 sec (FEV1) versus time" while Secondary Endpoints including: differences in Six minute walk distance (6MWD), BORG Score, corrected single-breath diffusing capacity (DCO corrected) at time intervals of 1, 3, 6 months on therapy. Specific biomarkers for BOS, including inflammatory chemokines, which are routinely collected in the context of post-transplant "surveillance" will be analyzed. Chemokines which our group has previously described in the pathogenesis of the continuum of "acute-to-chronic lung allograft rejection", have included both C-C (CCL2, CCL5) and CXC (CXCL9, CXCL10, CXCL11) chemokines as determined in bronchial-alveolar lavage (BAL).
Our primary aim in the current study is to determine important tissue differences, including muscle and connective tissue changes, between postmenopausal women and reproductive age women with pelvic floor dysfunction to help develop targeted and noninvasive treatments.
The study aims to determine if defecation posture, when using a defecation posture modification device (DPMD), will improve ease of bowel movements in patients with pelvic organ prolapse, specifically rectoceles.
Urinary retention can lead to bladder over-distention, ischemia, and long-term voiding dysfunction, and early identification of urinary retention can help prevent these adverse events
This study evaluates the addition of a laparoscopic Burch colposuspension procedure at the time of laparoscopic sacrocolpopexy to prevent stress urinary incontinence. Half of the participants will undergo Burch colposuspension procedure at the time of sacrocolpopexy, and half of the participants will undergo sacrocolpopexy alone.
The purpose of this study is to compare safety and effectiveness of the Exair Prolapse Repair System for treatment of pelvic organ prolapse to traditional native tissue repair through 36 months of follow-up.
The purpose of this study is to collect information on the safety and effectiveness of Restorelle Direct Fix mesh and the surgical procedure to implant Restorelle. These results will be compared to the safety and effectiveness results in patients who have native tissue repair (without mesh) as their pelvic organ prolapse treatment.
Compare changes in bowel function before, 6 months and one year after Robotic-assisted laparoscopic sacral colpopexy. Compare objective anatomic outcomes before, 6 months and one year after Robotic-assisted laparoscopic sacral colpopexy.