View clinical trials related to Syndrome.
Filter by:A Study of Vurolenatide in Adult Patients with Short Bowel Syndrome.
To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls with Rett syndrome
Current treatments for patients with drug reaction with eosinophilia and systemic symptoms (DRESS) include supportive care, steroids and cyclosporine. No randomized controlled trial (RCT) exists in comparing these treatments and all available literature comes in the form of case reports and case series. These two treatments are considered standard of care and this trial seeks only to compare outcomes of DRESS between these two therapies. No additional labs, therapies or procedures will be used apart from those that are routinely done for patients with this diagnosis. This will be a pilot study to determine efficacy of the two therapies with particular endpoints in mind so that the investigators can study the safety of these two therapies in patients with DRESS. Data suggests a potential benefit for adults with DRESS using either steroids or cyclosporine but the investigators are seeking a comparison of efficacy of these two therapies. The study population will include adults with a Registry of Severe Cutaneous Adverse Reaction (RegiSCAR) score of greater than 4 (i.e. a likely diagnosis of DRESS). The investigators will exclude patients with sepsis, active Hepatitis B or C, active tuberculosis, a documented allergy to steroids or cyclosporine, and patients with an estimated glomerular filtration rate (eGFR) < 30 (unless on dialysis in which case the participants will be included).
This study is a basket trial designed to establish safety, tolerability and efficacy of MHV370 in Sjögren's Syndrome (SjS) and Mixed Connective Tissue Disease (MCTD).
The COVID-19 pandemic is caused by the severe acute respiratory syndrome coronavirus 2 (SARS CoV-2), an emerging coronavirus, which has already infected 192 million people with a case fatality rate close to 2%. About 5% of patients infected with SARS CoV-2 have a critical form with organ failure. Among critical patients admitted to intensive care, about 70% of them will require ventilatory assistance by invasive mechanical ventilation (MV) with a mortality rate of 35% and a median MV duration of 12 days. The most severe lung damage resulting from SARS CoV-2 infection is the acute respiratory distress syndrome (ARDS). The virus infects alveolar epithelial cells and capillary endothelial cells leading to an activation of endothelium, hypercoagulability and thrombosis of pulmonary capillaries. This results in abnormal ventilation / perfusion ratios and profound hypoxemia. To date, the therapeutic management of severe SARS CoV-2 pneumonia lay on the early use of corticosteroids and Interleukin-6 (IL-6) receptor antagonist, which both reduce the need of MV and mortality. The risk factors of death in Intensive Care Unit (ICU) are: advanced age, severe obesity, coronary heart disease, active cancer, severe hypoxemia, and hepatic and renal failure on admission. Among MV patients, the death rate is doubled in those with both reduced thoracopulmonary compliance and elevated D-dimer levels. Patients with severe alveolar damage are at risk of progressing towards irreversible pulmonary fibrosis, the incidence of which still remain unknown. The diagnosis of pulmonary fibrosis is based on histology but there are some non-invasive alternative methods (serum or bronchoalveolar biomarkers, chest CT scan). We aim to assess the incidence of pulmonary fibrosis in patients with severe SARS CoV-2 related pneumonia. We will investigate the prognostic impact of fibrosis on mortality and the number of days alive free from MV at Day 90. Finally, we aim to identify risk factors of fibrosis.
The main aim of the study is to see if signs and symptoms of myelodysplastic syndromes disappear when treated with pevonedistat combined with decitabine and cedazuridine. Participants will receive an infusion of pevonedistat 3 times during a 28-day cycle. They will also take decitabine and cedazuridine tablets once a day for the first 5 days of the same cycle. A minimum of 6 28-day cycles is recommended, but participants can stop treatment at any time. A bone marrow biopsy, bone marrow aspirates, and blood samples will be collected during the study. Participants will attend a follow-up visit 30 days after their last dose of pevonedistat. Once treatment has ended, participants will be followed up with either monthly clinic visits or will be contacted every 3 months.
Congenital hyperinsulinism (HI) is a rare disorder of pancreatic beta cell insulin secretion that causes persistent and severe hypoglycemia starting at birth. Hyperinsulinism/hyperammonemia (HI/HA) syndrome is the second most common type of congenital HI and is caused by activating mutations in glutamate dehydrogenase (GDH). Patients with HI/HA exhibit fasting hyperinsulinemic hypoglycemia, protein-induced hypoglycemia, hyperammonemia, seizures, and intellectual disability independent of hypoglycemia. These effects result from abnormal GDH activity in the beta cells, liver and kidney cells, neurons, and astrocytes. The only available treatment for HI/HA syndrome is diazoxide, which acts on the beta cells to decrease insulin secretion but has no effect on GDH activity itself or on other cell types. Thus, there remains a significant unmet need for improved therapies for this disorder. Pre-clinical data show that vitamin E inhibits GDH activity in human cell lines and improves fasting hypoglycemia in a GDH HI mouse model. Pilot study data show that vitamin E supplementation with a moderate dose is well-tolerated in children and adults with HI/HA syndrome, while continuing diazoxide treatment. However, most subjects continued to exhibit protein-induced hyperinsulinemic hypoglycemia. We hypothesize that a higher vitamin E dose will inhibit GDH over-activity in subjects with HI/HA syndrome, resulting in improved hyperinsulinemic hypoglycemia, reduced blood ammonia concentration, and decreased seizure activity.
The current study intends to establish a prediction method and evaluation system for polycystic ovary syndrome (PCOS) complicated with affective disorder, such as depression and anxiety, through the epidemiological investigation. Randomized controlled studies on the efficacy of various intervention methods should be carried out to develop early intervention measures and methods in order to reduce the harm of psychological disorders, to facilitate the mental health of PCOS patients, and thus to improve the quality of life.
In preterm infants with neonatal respiratory distress syndrome (RDS), exogenous pulmonary surfactant(PS) replacement therapy is one of the most important therapeutic breakthrough to reduce neonatal incidences of bronchopulmonary dysplasia(BPD) and/or death. But not all preterm infants with RDS can be beneficial. Otherwise, the international neonatal acute RDS (NARDS) collaborative group provides the first consensus definition for NARDS in 2017. And whether or not PS being beneficial in preterm infants with NARDS remains unknown.
In order to obtain suitable exercise intervention model for children and adolescents with obese or metabolic syndrome in China, we conduct a RCT to examine the effects of different intensity exercise intervention on weight, and other cardiometabolic risk factors among children and adolescents, we also test the effects of exercise interventions on multi-omics such as metabolomics, gut microbiome, genetics, and explore the potential mechanisms by which exercise interventions modulate cardiometabolic risk. This is a randomized controlled study. Children and adolescents with obesity or metabolic syndrome were randomly divided into three groups, including low intensity exercise training group, moderate and intermittent high intensity exercise training group. The investigators will obtain organ hydrodynamics indexes by abdominal ultrasound and computational fluid dynamics technology, and obtain the metabolic regulation mechanism of the body in response to exercise through the detection of metabolism related small molecules in blood and urine and the analysis of intestinal flora, so as to explore the potential value and mechanism of different exercise modes in the treatment of obesity and metabolic syndrome. At the same time, the behavior and self-consciousness of children and adolescents will be investigated to obtain the suitable exercise intervention model for Chinese children and adolescents with obesity and metabolic syndrome. In conclusion, this study aims to demonstrate which exercise mode can alleviate obesity or reverse metabolic diseases, analyze which factors can sense exercise, and explore the potential mechanisms through mutil-omic approach, so as to improve metabolic syndrome.