Transanal Irrigation to Prevent Major Low Anterior Resection Syndrome
Low Anterior Resection SyndromeBowel dysfunction after rectal cancer resection comprises a vast array of bowel symptoms and associated quality-of-life impairment, collectively termed as low anterior resection syndrome (LARS). There are 40%-60% patients who suffer from major LARS after sphincter-preserving surgery. No consensus exists for LARS treatment or prevention. Transanal irrigation (TAI) was reported to play a helpful role in the management of major LARS and fecal incontinence. However, the preventive effect and daily accessibility need further confirmation. In this randomized trial, TAI compared with best support treatment, is used in patients who received curative low anterior resection for rectal cancer with diverting stoma, after completion of the stoma reversal. The primary outcome is the occurrence of major LARS after 6 months of the treatment.
A Controlled Trial of Growth Hormone in Phelan-McDermid Syndrome and Idiopathic Autism
Phelan-McDermid SyndromeAutism Spectrum Disorder (ASD)This clinical trial will use growth hormone as a novel treatment for Phelan-McDermid syndrome (PMS) and idiopathic autism. A double-blind, placebo-controlled crossover trial design will be used in 30 children with idiopathic autism and 15 children with PMS to evaluate the the effects of growth hormone on visual evoked potentials (VEPs), socialization, language, and repetitive behaviors. The researchers expect to provide evidence for the feasibility of using VEPs in PMS, and to show support for growth hormone in ameliorating clinical symptoms of ASD.
The Interaction and Regulation Mechanism of Different Exercise Patterns With Metabolic Syndrome...
ObesityMetabolic SyndromeIn 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.
Research on Risk Factors and Interventions of Polycystic Ovary Syndrome Complicated With Depression...
PCOS (Polycystic Ovary Syndrome) of Bilateral OvariesDepression1 moreThe 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.
DEXamethasone EARLY Administration in Hospitalized Patients With Covid-19 Pneumonia
COVID-19Acute Respiratory Distress Syndrome2 moreThe aim of this study is to evaluate the efficacy of dexamethasone in hospitalized adults with COVID-19 pneumonia who do not require supplementary oxygen on admission, but have high risk of developing acute respiratory distress syndrome (ARDS). This is a prospective, multicenter, phase 4, parallel-group, randomized and controlled trial that is open-label to investigators, participants and clinical outcome assessors. Eligible participants include adults (age 18 years or older), diagnosed with SARS-CoV-2 infection, evidence of infiltrates on chest radiography or computerized tomography, peripheral capillary oxygen saturation ≥94% and 22 breaths per minute breathing room air, and high risk of developing ARDS defined by a lactate dehydrogenase higher than 245 U/L, C-Reactive Protein higher than 100 mg/L, and absolute lymphocytes lower than 800 cells/µL. Eligible participants will meet two of the three before analytical criteria associated with severe COVID-19. Patients will provide written informed consent. Exclusion criteria include patients with a history of allergy to dexamethasone, pregnant or lactating women, oral or inhaled corticosteroids treatment within 15 days before randomization, immunosuppressive agent or cytotoxic drug therapy within 30 days before randomization, neutropenia <1000 cells/µL, human immunodeficiency virus infection with CD4 cell counts <500 cells within 90 days after randomization, dementia, chronic liver disease defined by ALT or AST ≥5 times the upper limit of normal, chronic kidney injury defined by a glomerular filtration rate ≤30 ml/min, hemodialysis or peritoneal dialysis, uncontrolled infection, and patients who are already enrolled in another clinical trial. Study participants will be randomized in a 1:1 ratio to receive dexamethasone base 6 mg once daily for seven days or standard of care. The primary endpoint is to prevent of development of moderate ARDS. Based on the Berlin criteria, moderate ARDS is defined by a PaO2/FiO2 ratio >100 mmHg and ≤200 mmHg. Study participants will be randomized in a 1:1 ratio to receive dexamethasone versus standard of care using a randomization platform. Included participants will be hospitalized at the time of randomization. The study will be undertaken at Infanta Leonor-Virgen de la Torre University Hospital, Enfermera Isabel Zendal Emergency Hospital, and Infanta Cristina Hospital, Madrid, Spain.
Classification, Functional Stratification and Biomarkers in Ciliopathy (CILLICORIRCM)
CiliopathiesNephronophthisis4 moreThe purpose of the C'IL-LICO RICM study is to develop innovative and transformative diagnostic and prognostic for patients suffering from ciliopathies leading to renal failure. The objectives is to decipher disease mechanisms and highlight signaling pathways altered in at-risk to develop renal failure patient groups and to produce a prognostic biomarker-based kit to predict the evolution of ciliopathy patients towards renal impairment.
Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases
Severe Combined Immunodeficiency (SCID)Immunodeficiency With Predominant T-cell Defect8 moreThe purpose of this study is to determine whether bilateral orthotopic lung transplantation (BOLT) followed by cadaveric partially-matched hematopoietic stem cell transplantation (HSCT) is safe and effective for patients aged 5-45 years with primary immunodeficiency (PID) and end-stage lung disease.
Exercise Dose and Metformin for Vascular Health in Adults With Metabolic Syndrome
Metabolic SyndromeInsulin Sensitivity1 moreArterial disease is the leading cause of morbidity/mortality in Metabolic syndrome (MetS). This occurs early as evidenced by arterial dysfunction that, in turn, raises blood pressure and glucose. Health organizations recommend exercise in an intensity based manner to promote cardiovascular adaptation and prevent disease. Metformin is a common anti-diabetes medication that reduces future type 2 diabetes and cardiovascular risk. However, the optimal exercise dose to be combined with metformin for additive effects on vascular function is unknown. Based on the investigator's preliminary work, the overall hypothesis is that metformin blunts adaptation following high intensity exercise training (HiEx) by lowering mitochondrial derived oxidative stress signaling. The investigators further hypothesize that low intensity exercise (LoEx) training combined with metformin will promote additive effects on vascular function compared to LoEx or HiEx+metformin, and maintain/improve non-exercise physical activity patterns. In this double-blind trial, obese 30-60y MetS participants will be randomized to: 1) LoEx+placebo; 2) LoEx+metformin, 3) HiEx+placebo; or 4) HiEx+metformin for 16 weeks.
Novel Form of Acquired Long QT Syndrome
Long QT SyndromeConnective Tissue Diseases1 moreThe goal of this study is to determine whether anti-Ro/SSA antibodies are associated with acquired QT prolongation in subjects with connective tissue disease. The investigators will investigate whether gender or race influence correlation between anti-Ro antibody status and prolonged QT interval and the role of inflammatory cytokines in association with anti-Ro antibodies and QT prolongation. The investigators propose to add an additional objective to test whether QT prolongation is reversible with moderate doses of prednisone in patients with QT interval greater than 500 msec.
CODY: A Study Evaluating The Safety And Efficacy Of QTORIN 3.9% Sirolimus Topical Gel For The Prevention...
BCCs in Gorlin Syndrome PatientsA Phase 2b study looks at the safety and efficacy of a treatment that is being investigated for people with certain diseases. This study will be conducted at multiple centers in the United States where participants with Gorlin Syndrome, also known as basal cell nevus syndrome, will be randomly placed into two groups; one group will receive the active topical gel, the other a topical vehicle gel, also know as placebo. Participants will apply this topical product to their face once a day for 6 months. The study will be looking at the number of new BCCs that develop on the faces of all the participants during this time.