10 Clinical Trials for Various Conditions
Congenital heart disease (CHD) includes a wide variety of types of disease, including congenital abnormalities of the heart valves. This can range from bicuspid aortic valve and other aortic valve deformities to more complex disease such as tetralogy of Fallot. For many kinds of CHD, the optimal timing of interventions remains unclear. For instance, in tetralogy of Fallot, there is still equipoise about when to offer pulmonary valve replacement (PVR), while in aortic regurgitation, some patients can remain stable for many years. The primary focus of this study is to use continuous physiologic data (CPD), obtained using wearable biosensors (a type of biometric monitoring technology), to develop improved biomarkers of disease progression and prognosis from patients with congenital heart disease (CHD) who are pregnant while they are at home as well as looking at patients' experience and interaction with wearable biosensor technology at home.
The objectives of this study and registry are to offer the best management possible for patients with brain arteriovenous malformations (AVMs) (ruptured or unruptured) in terms of long-term outcomes, despite the presence of uncertainty. Management may include interventional therapy (with endovascular procedures, neurosurgery, or radiotherapy, alone or in combination) or conservative management. The trial has been designed to test a) whether medical management or interventional therapy will reduce the risk of death or debilitating stroke (due to hemorrhage or infarction) by an absolute magnitude of about 15% (over 10 years) for unruptured AVMs (from 30% to 15%); and, b) to test if endovascular treatment can improve the safety and efficacy of surgery or radiation therapy by at least 10% (80% to 90%). As for the nested trial on the role of embolization in the treatment of Brain AVMs by other means: the pre-surgical or pre-radiosurgery embolization of cerebral AVMs can decrease the number of treatment failures from 20% to 10%. In addition,embolization of cerebral AVMs can be accomplished with an acceptable risk, defined as permanent disabling neurological complications of 8%.
The study will use blood (serum and plasma) and tissue obtained from participants undergoing prescribed surgical resection of vascular anomalies of interest proposed in this study. The study will also use blood (serum and plasma) and tissue collected and stored in a tissue bank maintained by the Department of Hematology/Oncology.
Congenital Portosystemic Shunt (CPSS) is a rare condition important by the multiplicity and severity of associated complications. CPSS is venous anomaly in which blood coming from the intestines only partially passes through the liver. This leads to the accumulation of potentially toxic factors that cause systemic effects. Complications vary among the individuals, and currently, it is challenging to predict which individuals will develop severe complications. The IRCPSS registry is established with the aim of centralizing detailed clinical follow-up and biological information from participants around the world who suffer from Congenital Portosystemic Shunt (CPSS). A multidisciplinary consortium of experts is collaborating to enhance our understanding of the prevalence, natural history, individual risks, and physiopathology of the disease through the IRCPSS registry.
A single arm clinical trial evaluating the safety and efficacy of the second generation TEVG as vascular conduits for extracardiac total cavopulmonary connection.
Aim #1: Define and determine the prevalence of pulmonary vascular disease and diastolic dysfunction as assessed by the gold standard of invasive hemodynamic cardiopulmonary exercise testing. Aim #2: Determine the role of rest-exercise echocardiography for the assessment hemodynamics in Fontan physiology. Aim #3: Evaluate the clinical impact of pulmonary vascular disease and ventricular diastolic dysfunction.
The main purpose of this study is to define the complex genetic and pathogenic basis of thoracic aortic aneurysm (TAA) and other forms of aortopathy and/or aortic valve disease by identifying novel disease-causing genes and by identifying important genetic modifiers for aortic and aortic valve disease severity.
Neonates and infants that have cardiac surgery with cardiopulmonary bypass (CPB) for congenital heart disease are at great risk for experiencing life-threatening low cardiac output syndrome (LCOS) in the first 24 hours after surgery. The poor perfusion and inadequate oxygen delivery that occurs may result in multiple organ failure and death. It is LCOS that is responsible for the majority of early postoperative deaths in this population of neonates and infants. Improved pediatric probes placed in peripheral locations using near infrared spectroscopy (NIRS) may permit continuous monitoring of venous saturations reflecting overall perfusion and oxygen balance in the tissues. Following parental or guardian consent, 30 neonates and infants scheduled to undergo surgery for congenital heart disease will be enrolled. At the end of surgery, four EQUANOX Advance 8004CB probes will be placed on the flank, lower extremities and the forehead. Continuous NIRS saturations will be monitored and stored for analysis. Point of care (POC) lactates will be obtained after admission to the intensive care unit (ICU) every 2 hours for the first 24 hours postoperatively, then once at 48 hours. The association between NIRS oxygen saturation and POC lactate values will be assessed using mixed linear models taking into account the repeated measures design. Exploratory analyses will be performed to assess whether NIRS oxygen saturation is associated with outcomes such as days in ICU, adverse events and mortality. The ability to use noninvasive, continuous monitoring for overall perfusion and cardiac output will allow better and earlier therapy for neonates and infants following cardiac surgery.
Clinical evidence reveals that some patients who undergo cardiac transplantation exhibit problems with pulmonary vascular resistance as well. In some studies, an increase in pulmonary vascular resistance has been used as an indicator for increased 3-day and 3-month mortality after heart transplantation2. In this study, the investigators would like to look at pulmonary vascular resistance in patients with a surgical history of the Fontan procedure followed by heart transplantation.
SELVA: A Multicenter, Phase 3 Baseline-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations The main purpose of this study is to assess the change in microcystic lymphatic malformations IGA after 24 weeks of treatment with QTORIN 3.9% Rapamycin Anhydrous Gel in approximately 40 participants with microcystic lymphatic malformations. Efficacy will be evaluated at 24 weeks and patients have the option of continuing on treatment for \>24 weeks.