45 Clinical Trials for Various Conditions
The goal of this Phase I Open-Label study is to demonstrate the safety and feasibility of VentriGel injection in children with Hypoplastic Left Heart Syndrome (HLHS). The main questions it aims to answer are: * Whether VentriGel is safe in treating patients with HLHS * Whether there are any preliminary improvements in measures of cardiac function following Ventrigel injection
The purpose of this research is to investigate the best way to manage evolving hypoplastic left heart syndrome (HLHS).
The purpose of this study is to test whether Lomecel-B™ works in treating patients with hypoplastic left heart syndrome (HLHS) and to gather additional information about the safety of Lomecel-B. Lomecel-B contains human mesenchymal stem cells (MSCs) as the active ingredient. MSCs are special cells in the body that are able to change into other types of cells, such as heart, blood, and muscle cells. MSCs are found in various tissues of the body, such as the bone marrow, which is the spongy tissue inside of your bones. Lomecel-B uses MSCs from bone marrow of unrelated young healthy donors. These are called "allogeneic", and do not require donor matching to the patient.
Researchers want to better understand what happens to the heart when the stem cells are injected directly into the muscle of the right side of the heart during the Stage II palliative surgery for single ventricle patients with hypoplastic left heart syndrome (HLHS) or HLHS variant. Researchers want to see if there are changes in the heart's structure/function following this stem cell-based therapy and compared to children that have not had cell-based therapy.
This study is designed to assess the safety, tolerability, and efficacy of Lomecel-B as an adjunct therapy to the standard stage II (BDCPA) surgical intervention for HLHS. Lomecel-B will be delivered via intramyocardial injections
The objectives of this pilot study are to evaluate the feasibility and safety of intramyocardial injection of autologous c-kit+ cells during the Stage II BDCPA operation and to observe effects on clinical outcome including right ventricular myocardial function, severity of tricuspid regurgitation, incidence of serious adverse events, re-hospitalizations, changes in health status, the need for transplantation, or mortality.
This study is intended to evaluate the safety and feasibility of intramyocardial injection of allogeneic mesenchymal cells during the Bi-Directional Cavopulmonary Anastomosis (BDCPA) surgery for hypoplastic left heart syndrome (HLHS) pediatric patients.
This is a Phase I study to determine the safety and feasibility of injections of autologous umbilical cord blood (UCB) cells into the right ventricle of Hypoplastic Left Heart Syndrome (HLHS) children undergoing a scheduled Glenn surgical procedure. The investigators are doing this research study to find out if autologous stem cells from the individual's own umbilical cord blood can be used to strengthen the muscle of the right side of their heart. This will help determine the safety and feasibility of using cell-based regenerative therapy as an additional treatment for the management of HLHS.
Cell-based cardiac regeneration has been the focus of acquired, adult heart disease for many years. However, congenital heart disease with severe structural abnormalities may also be reasonable targets for cell-based therapies. Interestingly, the pediatric heart is naturally growing and may be the most amendable to regenerative strategies. Therefore, identifying autologous cells (cells from the patient's own body) would be important to initiate these studies. This study aims to validate the use of umbilical cord blood as a source of autologous cells for the purpose of cardiac repair of congenital heart disease. Cells will be isolated from the cord blood to help us determine the feasibility of collection, processing, and storage of these samples at the time of birth of infants with prenatal diagnosis of hypoplastic left heart syndrome. This study may be useful for the development of pre-clinical and clinical studies aimed at the long-term goal of repairing damaged heart muscle.
For fetuses with severe aortic stenosis, in utero balloon aortic valvuloplasty may improve fetal growth of left heart structures and thus improve potential for biventricular repair strategies after birth.
Hypoplastic left heart syndrome (HLHS) is a severe form of congenital heart disease that consists of multiple obstructions to flow through the left heart and aorta, as well as hypoplasia of the left ventricle. Most patients require a three-stage surgical protocol starting within days of birth. Stage I of this process is the Norwood reconstruction (within the first few days of life), Stage II (usually required within 3-8 months) involves creation of a direct connection between the patient's superior vena cava and the pulmonary arterial confluence (bidirectional Glenn anastomosis), and the last stage is creation of a Fontan circulation (typically within the first 2-4 years). This "single ventricle" approach requires the right ventricle to perform as the only circulatory pump for the entire body. Our long-term goal is to develop regenerative strategies to strengthen and augment the right ventricular muscle of the single-ventricle heart following surgical palliation in HLHS patients. To determine the safety and feasibility of a cell-based therapeutic intervention at the Stage II surgery, we aim to document the natural history of post-surgical care in HLHS patients having undergone standard of care with protocol specific follow-up over the course of a 6-month period. This prospective study will document the natural history in patients with HLHS after planned Stage II surgical palliation with a focus on cardiovascular parameters within 6 months following surgery in 10 patients.
Further study details as provided by Duke University: Purpose: To evaluate the feasibility and safety of collecting and infusing autologous umbilical cord blood (UCB) in newborn infants with hypoplastic left heart syndrome (HLHS). Study Rationale and Hypotheses: The major goal of this study is to determine whether infusion of autologous UCB cells in neonates with hypoplastic left heart syndrome is feasible and safe. The rationale for the study and for the potential benefit of UCB is based upon the following hypotheses: 1. Infants with HLHS have significant neural injury evidenced from both prenatal and early antenatal brain MRI findings and infusion of UCB cells may lessen neural injury. Although the exact mechanism is unknown, UCB cell infusion may ameliorate neural injury via paracrine and anti-inflammatory effects that enhance post injury repair and may promote endogenous functional compensation of other cortical areas resulting in significant clinical improvements. 2. UCB cells may also enhance cardiac function, minimize scar formation, and reverse detrimental remodeling after cardiac injury.
The purpose of this study is to determine whether children and adolescents 8-18 years of age with HLHS and related lesions who have undergone stage I palliation during infancy using an allograft patch demonstrate continued evidence of HLA antibody formation.
Survival for one of the most complex forms of congenital heart disease, hypoplastic left heart syndrome (HLHS), has improved dramatically. However, little is known about family stress, coping and outcomes following the diagnosis of HLHS. It is expected that families face emotional, social and financial stressors. Health care professionals have a unique opportunity to positively influence how families interpret and adapt to these stressors. The specific aims of the study are to describe perceived stress, and coping skills utilized, in parents of children with HLHS and their impact on family outcomes measured as well-being, adaptation and caregiver/family quality of life, and to describe changes in stress, coping, and adaptation and differences in perceptions of mothers versus fathers of children with HLHS over the first 14 months of life. The Resiliency Model of Family Adjustment and Adaptation (McCubbin, Thompson, \& McCubbin, 1996) is the theoretical framework that guides this research. Hypotheses: 1. Family perception of stress, and coping skills utilized, will have an impact on family outcomes measured as well-being, adaptation, and caregiver/family quality of life. 2. Variables influencing perception of stress and variables influencing family coping will be significant predictors of family adaptation outcomes. 3. Perceptions of stress, coping skills utilized, and family adaptation outcomes will improve during the first 14 months of life with an infant with HLHS. 4. Mothers and fathers will report different perceptions of stress, coping skills utilized, and family adaptation outcomes during the first 14 months of life with an infant with HLHS.
There have been many improvements in the care of children with hypoplastic left heart syndrome (HLHS). This has helped these children survive longer. Because these children now live longer, researchers are recognizing developmental disabilities (the children are behind in their thinking or physical activity) in children with hypoplastic left heart syndrome. The purpose of this research study is to help the investigators learn more about developmental disabilities in children with hypoplastic left heart syndrome. During the time of your child's first surgery for hypoplastic left heart syndrome, a monitor was placed on your child's forehead to measure the oxygen levels in the brain. This monitor is called Near Infrared Spectrometry (NIRS). The researchers involved in this study want to know if oxygen levels in the blood vessels of the brain have any effect on developmental disabilities later in life in children with hypoplastic left heart syndrome. The researchers plan to enroll 60 research subjects.
A single arm clinical trial evaluating the safety and efficacy of the second generation TEVG as vascular conduits for extracardiac total cavopulmonary connection.
This clinical trial is studying the use of different levels of oxygen exposure during and after cardiopulmonary bypass in eligible infants to learn about its safety during heart surgery. In addition to having the various doses of oxygen, participants will also have blood samples, ultrasounds of the head, and brain wave patterns monitored. The hypotheses of this trial are: * that there will be no difference with regards to adverse events between the infants in the normoxia group compared to the infants in the standard of care group * there will be a significant difference in the measured partial pressure of oxygen (PaO2) values between the two treatment groups. * the use of normoxia during cardiopulmonary bypass and in the immediate post-operative period will result in clinically significant decrease in oxidative stress as measured by thiobarbituric acid reactive substances (TBARS) after cardiac surgery
The primary aim of this study is to determine if clinically relevant doses of buccally administered oxandrolone are safe and tolerable in neonates with hypoplastic left heart syndrome (HLHS) or other single right ventricular anomalies who have undergone a Norwood procedure. The secondary aim is to evaluate the efficacy of buccally administered oxandrolone in improving objective indices of growth and nutrition in neonates who have undergone a Norwood procedure.
All neonates with congenital heart disease undergoing stage one palliation (Norwood procedure, Damus-Kaye-Stansel procedure) at Texas Children's Hospital will be regularly monitored for B-type natriuretic peptide (BNP) and Troponin level before the surgical procedure, on arrival to the cardiac intensive care unit after their surgical procedure, every 6 hours during the first 24 hours of the post-operative period, followed by daily levels for the first week, and then weekly during patient's regular laboratory work up schedule. These cardiac biomarkers are linked to demographic, hemodynamic, respiratory, pharmacological data available via Sickbay.
Patients under the age of 5, with a diagnosis of hypoplastic left heart syndrome (HLHS), unbalanced atrioventricular canal (uAVC), or borderline left heart who are undergoing staged LV recruitment following bidirectional Glenn (BDG) or undergoing BDG with plans for LV recruitment will be considered for enrollment in this study. Those patients enrolled in the study will be randomized to either the experimental arm or control arm of the study. Those patients randomized to the experimental arm will receive mesenchymal precursor cells (MPCs) injected directly into the LV endocardium during their LV recruitment or BDG procedure. Those patients randomized to the control arm will receive normal standard of care during their procedure with no injection of MPCs. It is believed that injection of MPCs will help improve the chances of those patients with single ventricle or borderline left ventricle being converted to biventricular circulation which could improve quality of life and longevity over palliation.
Single ventricle lesions are the leading cause of illness and death from congenital heart disease. The modified Fontan Operation is the corrective surgery for these lesions. The operation is done in stages over a few years and children who complete the operation are known to have greater neurodevelopmental (ND) deficits than the general population. The purpose of this study is to understand how blood flow to the brain (CBF) and brain lesions relate to ND outcome, as well as how CMRO2 relates to anatomic brain lesions. These relationships will be studied through Magnetic Resonance Imaging (MRI) and ND Testing.
The purpose of this initiative is to improve care and outcomes for infants with HLHS by expanding the NPC-QIC national registry to gather clinical care process, outcome, and developmental data on infants with HLHS between diagnosis and 12 months of age, by improving the use of standards into everyday practice across pediatric cardiology centers, and by engaging parents as partners in the process.
Purpose: To evaluate the pharmacokinetics, bioavailability and hemodynamic efficacy of ambrisentan after Fontan surgical palliation of single ventricle heart defects. Study activities and population group: Children undergoing Fontan surgical palliation for single ventricle defects will be eligible for the study. Up to 20 subjects will be enrolled (16 ambrisentan, 4 placebo) and will receive 3 days (3 doses) of ambrisentan starting on post-operative day #1 upon returning from the operating room. Ambrisentan plasma levels will be obtained at specified time points during treatment. Post-operative monitoring lines will be used to measure effects of ambrisentan on hemodynamics and pulmonary / systemic endothelial function.
The purpose of this study is to learn about the relationship between family factors and developmental and psychosocial outcomes in children with congenital heart disease at 6 years of age. A secondary purpose is to learn more about psychosocial outcomes in children with congenital heart disease and their families over time. About 250 mothers and fathers at 15 medical centers will take part in this study; 35 will be from Children's Hospital of Wisconsin.
Children who are born with heart defects undergo surgery when they are infants to correct the defects. However, many treatments that are used in pediatric heart patients were originally developed for adults and may not be the best option for children. This study will analyze tissue samples from newborns and infants undergoing surgery for heart defects to learn more about how a child's heart develops during the first year of life. This information may help to identify possible treatments geared specifically for the pediatric heart patient.
We believe that how a baby with Hypoplastic Left Heart Syndrome (HLHS)does after a major open heart operation, measured by things like blood pressure, oxygen saturation, heart rate and others, may have an impact on development. Studying how post-operative condition impacts outcomes may help us to protect babies better when they undergo surgery. This study will look at some of the long-term outcomes of children with HLHS, including both mental development and quality of life. We will use information from your child's medical record to see if early oxygen delivery has an impact on later development.
Due to the small size of infants and the use of bypass machines, physicians have difficulty assessing whether the infant's brain and body is getting enough oxygen during heart surgery. This study compares continuous monitoring via the NIRS (Near Infrared Spectroscopy) to the traditional methods of determining oxygen saturation.
This is an exploratory study designed to evaluate the incidence of, and to quantify sleep disordered breathing following stage I Norwood reconstructive surgery. Sleep disordered breathing will be correlated with: 1. Elevations in pulmonary vasculature resistance at the time of Stage II surgery. 2. Risks of death
In one of the most severe congenital heart defects, hypoplastic left heart syndrome (HLHS), the left ventricle is underdeveloped and the prognosis is worse than in most other heart defects. The underdevelopment can occur gradually during fetal growth caused by a narrowing of the aortic valve. At some international centers, such fetuses are treated with a balloon dilation of the narrowed valve, but there is no scientifically sound evidence that this treatment is effective. The aim of this study is: 1/ to evaluate whether balloon dilation during the fetal period of a narrowed aortic valve can reduce the risk of the left ventricle becoming underdeveloped and the baby being born with a so-called univentricular heart (HLHS); 2/ to investigate whether such treatment improves the prognosis for this group of children with a very complex and severe heart defect and 3/ to also describe side effects and risks in fetuses and mothers of the fetal procedure.
This clinical trial is being done to see if giving surfactant (Curosurf®) will decrease the number of days that infants will need a breathing tube, decrease the days in the critical care unit and decrease the number of days needed in the hospital. The primary hypothesis for this study is that there will be fewer days needed on mechanical ventilation and improved lung compliance and pulmonary gas exchange.