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The purpose of this study is to evaluate the safety and tolerability of HQK-1001 administered daily for 8 weeks in subjects with beta thalassemia intermedia

The investigators propose a pilot trial to obtain preliminary information regarding the safety and response rate of patients with symptomatic lymphatic malformations treated with oral Selenium. Information obtained in this pilot trial will be used to plan future phase 2 clinical trials. Hypotheses: - Selenium will be safe and efficacious in the treatment of adolescents and young adults with symptomatic lymphatic malformations - Disease response will correlate with serum levels of selenium and blood levels of antioxidants essential to selenium metabolism.

The Membranous nephropathy (GEM)idiopatic is the most frequent cause of syndrome néphrotique at the adult and represent approximately 2 % of the causes of terminal chronic renal insufficiency. A etiology balance assessment must be systematically realized to eliminate a secondary cause. Antibodies managed against the receiver of phospholipases A2 secreted by type M (PLA2R1) was recently detected in a population of GEM idiopatic, but not secondary GEM. PLA2R1 is expressed by the podocytes of the glomerules of healthy subjects, and this receiver co-is located with the deposits of extramembraneous IgG of the expanding subjects of idiopathique GEM. The good IgG the extramembraneous depositsof GEM idiopathic recognize PLA2R1. At some patients, the activity anti-PLA2R1 seems to decrease or to disappear during the stake in forgiveness of the disease. Some cases of second offense of GEM idiopathique after renal transplantation presenting antibodies anti-PLA2R1 have also described. The appearance of antibody anti-PLA2R1 seems parallel to the increase of a proteinurie in touch with a second offense of GEM, and antibodies sometimes disappeared after a therapeutic strengthening by Rituximab allowing to obtain a forgiveness. A GEM can also appear of novo on the renal transplant, it is to say without notion of GEM on the native loins. The physiopathology of this affection remains unknown. Working hypothesis and objectives We shall look in which proportion the presence of antibody anti-PLA2R1 is associated with the second offense of GEM idiopathic in renal transplantation. We anticipate that the GEM of novo of the renal transplant answers a different physiopathology, and will not be associated with the presence of antibody anti-PLA2R1. We hope to demonstrate that at the expanding patients of antibody anti-PLA2R1, the title of these antibodies is correlated in the activity of the disease and in the renal survival, and that the longitudinal follow-up of the title of these antibodies has an interest forecast and therapeutics.

Insulin resistance is defined as the decreased ability of insulin to perform its biological function in the muscle, liver and fat. Genetic and environmental factors are known to influence insulin sensitivity. It is not known how this is mediated. This study looks at the role of epigenetics (modifications of proteins associated with DNA and methylation of DNA) in alterations in insulin resistance. We will study lean healthy people, obese non-diabetic people and people with type 2 diabetes to characterize the DNA methylation patterns in muscle in each group. The second aim of the study is to see how a single bout of exercise affects the DNA methylation in the muscle. The third aim looks at the effect of 8 weeks of supervised exercise on the DNA methylation. The investigators are trying to understand the role of DNA (deoxyribonucleic acid) methylation in insulin resistance in skeletal muscle and blood tissues. DNA methylation is a normal chemical process in the body that modifies DNA. By studying this, the investigators hope to better understand the causes of insulin resistance.

- The researcher will interview you for about 3 hours in a private office. - Study location: All these procedures will be done at Dr. Dunn's research offices at UCSF. If you prefer, we can conduct the interview at your home in or near the San Francisco bay area. - You will be asked questions about your general background, and about how providing care for the person with Alzheimer's Disease has affected you. You will also be asked some basic questions about the person with Alzheimer's Disease (such as how well they are functioning; personal information will not be collected, however). - Then, you will be given a consent form for an imaginary study related to Alzheimer Disease. An imaginary study means that it is not a real study, but it is a made-up one. You or your relative will not really participate in the study that is described in the consent form, because it is only imaginary and created just for this research. - Then, you will be asked questions to measure how well you understood the information presented to you. For example, you will be asked about the procedures, risks and benefits. - You will also be interviewed regarding your opinions and attitudes about research participation. If you give your permission, we will audiotape this interview to help us more accurately record this information. You may refuse to answer any of the questions that make you uncomfortable. You do not have to agree to be audio-taped to participate in this study. - You will be given some tests designed to measure memory, attention and other thinking abilities. These tests are for research purposes only, and are not being used for any type of diagnostic purposes. The research purpose is to help us understand proxies' abilities to make decisions as caregivers for seriously ill patients. - You will be asked questions about your mood and about caring for someone with dementia. You may refuse to answer and questions that make you uncomfortable. This is a research study about how family members make research participation decisions for a relative with Alzheimer's or a related dementia. The purpose of this study is to learn more about how proxies (the family member who makes decisions for that relative), would think through decisions about whether to enroll their relative in studies for not-yet-approved medications for Alzheimer's Disease. The long-term goal of the study is to improve the way researchers inform families about clinical research studies and opportunities for participation. This study is being sponsored by the National Institute on Aging. Study participants will be interviewed by a research staff member about research enrollment decisions for their relative. A number of standardized questionnaires will be administered. There is only one visit required. The interview lasts about three hours. The investigators will try to make it as convenient as possible for your schedule. This is a non-intervention study. UCSF CHR # H58055-31168-02 v.1 Date: 012709

To evaluate the role of routine staging laparoscopy in patients with potentially resectable pancreatobiliary cancer. All analyses are performed using SAS version 9.1.3 for Windows (SAS institute, Cary, NC). Clinical and pathological variables will be analyzed using the χ2 test (or Fisher`s exact test) and the Student`s t test, depending on the normality of the distribution. P-values of < 0.05 are considering statistically significant. The purpose of this study is to evaluate the role of routine Staging Laparoscopy in patients with potentially resectable pancreatobiliary cancer.

Cirrhosis of liver is a leading cause of morbidity and mortality worldwide . Complications including ascites, spontaneous bacterial peritonitis (SBP), variceal bleed, hepatic encephalopathy, hepatorenal syndrome (HRS) and development of hepatocellular carcinoma (HCC) have poor prognosis and further decreases the survival in these patients. It has been believed that cirrhosis is irreversible and that treatment should focus on preventing the progression of liver fibrosis/dysfunction and its complications. Currently the only effective treatment is liver transplantation, an increasingly limited and expensive resource especially in developing countries. Furthermore, transplantation comes with a requirement for lifelong immunosuppression, and considerable long-term side effects that include chronic renal failure, post-transplant lymphoproliferative disease, and cardiovascular complications. Short of liver transplant, recently, reports of unexpected plasticity in adult bone marrow have raised hopes that stem cell therapy may offer exciting therapeutic possibilities for patients with end stage liver diseases. It has been shown that in response to acute or chronic liver damage, bone marrow derived stem cells can spontaneously populate the liver and differentiate into hepatic cells. Animal and human studies suggested that such cells might contribute to the regeneration after different kinds of liver injuries . In animal models, after liver injury, bone marrow-derived circulating pluripotent cells have been reported to participate in liver repopulation with both non-parenchymal cells and hepatocytes. This repopulation process, however, seems to be highly dependent on the type of liver injury and experimental conditions. Fusion with hematopoietic cells has been substantiated as a mechanism by which hepatocytes can regenerate, and studies have demonstrated improved liver histology and survival in patients with cirrhosis following mobilization of bone marrow stem cells by granulocyte-colony stimulating factor (G-CSF) . Three recent studies have demonstrated G-CSF induced mobilization of bone marrow stem cells (CD34 cells) in peripheral blood and their subsequent increase in liver tissue and improved survival in patients with alcoholic hepatitis and acute on chronic liver failure (ACLF) . However there is insufficient data on whether G-CSF improves survival and prognosis in patients with cirrhosis. Also, Malnutrition is commonly seen (60-70%) in cirrhotics and have adverse prognosis on its outcome . The protein catabolic state of cirrhosis is associated with severe growth hormone (GH) resistance, with low levels of insulin-like growth factor (IGF)-I and its major binding protein (IGFBP)-3 . GH therapy in cirrhosis have been shown to improve nitrogen economy and to improve the GH resistance in a small pilot study. Also, GH therapy of short duration has shown to increase IGF1 levels, IGFBP-3 levels in patients of cirrhosis . GH therapy has also shown to improve liver regeneration and protein synthesis after hepatectomy in patients of HCC with cirrhosis . However there is scarcity of data on clinical impact of long term administration of GH therapy in patients of cirrhosis.

Investigators, Study Sites: Single-center trial at the Department of Pediatric Cardiology, Department of Cardiac Surgery and Department of Anesthesiology, University Medical Center, Heidelberg, Germany Exploratory proof of concept study (Investigator initiated trial) Indication: Postoperative pulmonary hypertension in infants and children undergoing cardiopulmonary bypass surgery for intracardiac repair of left-to-right shunt Objectives: To compare the efficacy of aerosolized iloprost with inhaled nitric oxide to prevent postoperative pulmonary hypertensive crises Design: Exploratory, open label, randomized study with parallel-group design; Duration of observation: 72 hours Population: Infants older than 4 weeks and children less than 18 months of age presenting with left-to-right shunt and increased pulmonary blood flow. Inclusion will be independent on the presence or absence of preoperative pulmonary hypertension Sample Size: - 20 patients: inhaled nitric oxide (iNO) - group; - 20 patients: aerosolized iloprost (ILO) -group Treatment: - Both groups: controlled ventilation, sedation, analgetics, inotropic substances as required, standardized intensive care treatment. - iNO - group: concentration of iNO at 10 ppm; administered by mechanical ventilation. - ILO - group: aerosolized Iloprost at a dose of 0,5 µg/kg body weight (12x / 24h), administered by ultrasound nebulizer. Efficacy Parameters: Occurrence of "minor" or "major" pulmonary hypertensive crises (PHTC) Safety Parameters: Arterial blood pressure, oxygen saturation, complete blood count Statistical Procedures: All analyses in this exploratory trial are descriptive, giving confidence intervals for differences between treatment groups. Primary analysis variable: Rate of occurrence of "major" or "minor" pulmonary hypertensive crises Secondary variables: Presence of hours of pulmonary hypertension, Duration of mechanical ventilation Inhaled nitrous oxide (iNO) will be compared to aerosolized iloprost (ILO) in pediatric patients after cardiac surgery with pulmonary hypertension. The hypothesis is that iloprost is more effective in preventing pulmonary hypertensive crises.

Addiction treatment is often characterized by long delays between first contact and treatment as well as high no-show and drop out rates leading to unused capacity in apparently full agencies. Patients do not get needed care and agency financial stability is threatened. The Network for Improvement of Addiction Treatment (NIATx) began as a high-intensity improvement collaborative of 39 addiction treatment agencies distributed across 25 states. NIATx substantially improved time to treatment and continuation in treatment by making improvements to organizational processes (such as first contact, intake and assessment, engagement, level of care transitions, paperwork, social support, outreach, and scheduling) in preliminary studies. While the results are very encouraging, they have, by intent, been obtained from a select group of agencies using a high-cost combination of services. A more practical diffusion model is needed to spread process improvements across the spectrum of treatment agencies. This study is a cluster-randomized trial to test the effectiveness and cost of less expensive combinations of the services that make up the NIATx collaborative (interest circles, coach calls, coach visits and learning sessions). This cluster-RCT randomly assign 201 treatment agencies in 5 states to four experimental arms. The agencies were randomized to an intervention for 18 months with a 9 month sustainability period. The study aimed to: 1) Determine whether a state-based strategy can (with NIATx support) can lead mainstream treatment agencies to implement and sustain process changes that improve the study's primary outcomes: time to treatment, annual clinic admissions, and continuation in treatment; and 2) Evaluate the effectiveness and cost of the services making up NIATx. This study aims to create a practical model for improving efficiency and effectiveness of addiction treatment.

This trial will evaluate subjects with Primary Immunodeficiency Disorder (PIDD) who are currently self-infusing Vivaglobin therapy for at least six months prior to changing to Hizentra® therapy. Following screening and enrollment, subjects will continue to self-infuse Vivaglobin® for seven infusions and then be converted to subcutaneous Hizentra® treatment which they will continue for the next 6 months. The study will determine if Hizentra® provides improved subject satisfaction by Treatment Satisfaction Questionnaire for Medication. Diaries related to the local site reactions, the number of infusion sites per subject/per dose, volume of Hizentra® per site, duration of infusions, systemic side effects of the new medication and dose on IgG levels and antibody titers, and local site reactions with Hizentra®. The purpose of this study is to measure the changes in the Treatment Satisfaction Questionnaire for Medication in the areas of effectiveness, side effects, and convenience of administration of each medication in Primary Immunodeficiency Disorder (PIDD) subjects transitioning from subcutaneous Vivaglobin® to Hizentra®.