Active clinical trials for "Syndrome"

This is a non-interventional, multinational, multi-centre study with primary data collection, to further document the safety and efficacy of osilodrostat administered in routine clinical practice in patients treated with osilodrostat for endogenous Cushing's Syndrome

This is a prospective, multi-centre, double blinded, randomized controlled trial, which will be conducted under a common protocol. The aim of this study is to evaluate the efficacy of vaginal laser for treatment of GSM (Genitourinary Syndrome of Menopause) compared to the sham procedure in postmenopausal women. The study population is female subjects > 18 years old with symptoms of genitourinary syndrome of menopause (GSM) who have not menstruated for at least 5 years.

Alpha-1 kinase (ALPK1) has been reported as a potential causative gene for ROSAH Syndrome. Genetic variants including T237M have been found in ROSAH Syndrome patients. Our in-house study has found that T237M mutation leads to hyperactivity of ALPK1, which may be the cause of the inflammatory syndromes found in ROSAH Syndrome patients. We hypothesize that T237M mutation ALPK1 cause ROSAH Syndrome and an ALPK1 inhibitor can be a potential therapy for treating this disease. To test our hypothesis, we designed an experiment in which ex vivo peripheral blood mononuclear cells (PBMCs) from ROSAH Syndrome patients will be exposed to a potent ALPK1 inhibitor (DF-003) or placebo. We expect to see downregulation of activated inflammatory genes, chemokine/cytokines and acute phase proteins in the ROSAH Syndrome patient samples that are exposed DF-003.

This is a multi-centre, double-blind, randomized phase III trial comparing metformin to placebo in patients with advanced prostate cancer starting (or have recently started) androgen deprivation therapy (ADT).

Posterior Reversible Encephalopathy Syndrome (PRES) is a clinical-radiological entity, which is associated with a variety of clinical conditions. The imaging and clinical findings are typically reversible, but many patients still have permanent neurological sequelae with a fatal outcome. Up to now there are no consistent conclusions about the factors that affecting its prognosis. The objective of our study is to discuss the effects of different causes, different imaging findings and laboratory parameters on the prognosis of PRES and deepen the understanding of the nature of PRES. This will contribute to predict the prognosis of patients with PRES and to further investigate the pathogenesis of PRES so as to guide the clinical treatment and follow-up evaluation. A multi-center retrospective case study will be performed from January 2016 to July 2017 and a total of 400 patients who meet the criteria for PRES diagnosis from 10 sub-centers are anticipated included in this research. Patients will be divided into several subgroups according to the etiology. Date will be collected from the clinical records，imaging and laboratory data of the patients, including demographic data, clinical data, imaging findings, laboratory parameters and follow-up data. During the follow-up, MRI and blood biochemical examination will be performed once more. The imaging findings of the patients will be assessed by two neuroimaging physicians, any difference between them will be agreed upon by consensus. Prognosis of the patients will be assessed by using the modified Rankin scale (mRS) scores. All of the above data is saved into the database. In this study we will review etiologies and imaging findings, laboratory and follow-up data of the patients we selected. Univariate and multivariate analysis will be performed within the group and between groups according to the subgroups. Then we will discuss the connection between different causes, different imaging findings and laboratory parameters and the prognosis of posterior reversible encephalopathy syndrome.

Rare diseases frequently affect women of childbearing age. Pregnancy in these women has become less rare, but remains associated with high levels of complications. One obstacle to their optimal management during pregnancy is that there are no prospective studies of pregnancy during rare diseases and several connective tissue diseases. As a consequence, the management of these pregnancies is non-standardised in terms of treatment, monitoring (frequency of consultations, laboratory tests and ultrasound), and organisation of care. Moreover, although these women (all diseases combined) are frequently exposed to medications potentially incompatible with pregnancy, little is known about the frequency of these exposures and especially their consequences to mother and child. For these reasons, researchers and clinicians from different specialties created an interdisciplinary research group on pregnancy and rare diseases (GR2), intended to improve the management of these patients' pregnancies. Using a single computer server, the investigators plan to set up a large prospective study of pregnancies in patients with rare diseases: various forms of myositis, lupus, antiphospholipid syndrome, Sjogren syndrome, scleroderma, and inflammatory rheumatic diseases. The investigators objective is to analyse the complications of pregnancies in women with rare diseases and then to improve their management and their quality of life.

The essential arterial hypotension and allostasis registry is a prospective, observational research that has the purpose of demonstrating that essential blood pressure (BP) disorders and the associated comorbidities are a result of the inappropriate allostatic response to daily life stress. This required a functioning brain orchestrating the evaluation of the threat and choosing the response, this is a mind-mediated phenomenon. If the response is excessive it contributes to high BP, if deficient to low BP, and the BP itself will identify the allostatic pattern, which in turn will play an important role in the development of the comorbidities. To do so, consecutive patients of any age and gender that visit a cardiologist's office in Medellin, Colombia, are recruited. Individuals are classified according to their arterial BP and allostasis and follow them in time to see what kind of diseases develops the most (including BP) in the follow up according to the categorization of the characteristic chosen and after adjustment for confounder's variables. In addition, stress events with their date are registered. HYPOTHESIS The causes of the diseases are multifactorial. Physical, biochemical, psychological, social, and cultural dimensions of development dynamically interact to shape the health development process. A person´s health depends on their: Biological and physiologic systems External and internal environment (a) physical, b) internal behavioural and arousal state as registered by the brain. Their interaction. The allostatic mechanisms to the internal and external stressors (allostatic load) involves a network composed by: Functional systems; mediated by: The Autonomic Nervous System The endocrine system The immune system Structural changes: whenever the internal and/or external stressors are long lasting and/or strength enough, they may induce changes in: Epigenetic, endophenotypes, polyphenism. Plasticity The interaction between a) and b). The network response do not affect exclusively the BP, propitiating the development of comorbidities, which may prompt strategies for prevention, recognition and ultimately, treatment. The allostatic model defines health as a state of responsiveness. The concept of psycho-biotype: The allostasis is the result of both: biological (allostasis) and psychological (psychostasis) abilities. It is proposed that both components behave in similar direction and magnitude. Immune disorders may be associated with the development of cancer. High BP population has a higher sympathetic and lower vagal tone, this has been associated with a decrease in the immune´s system function. Resources and energy depletion: Terms like weathering have been used to describe how exposures to different allostatic loads gradually scrape away at the protective coating that keeps people healthy. It is postulated that High BP individuals have more resources and energy.

Parkinson disease (PD), multiple system atrophy (MSA) and progressive supranuclear palsy (PSP) are neurodegenerative disorders. PD and MSA are alpha-synucleinopathies, which are characterized by the abnormal accumulation of alpha-synuclein, while tau protein accumulates in PSP. The development of biological markers for the diagnosis and prognosis in PD, MSA and PSP remains an unmet need. Such biological markers are crucial for future disease-modification and neuroprotection trials. Alpha-synuclein has a high potential for biomarker development since it constitutes the pathological hallmark feature in PD and MSA. The oligomeric alpha-synuclein seems to be particularly involved in abnormal protein aggregation in alpha-synucleinopathies. The main objective is to compare oligomeric alpha-synuclein CSF levels between PD, MSA and PSP patients. PD and MSA patients will receive Cerebrospinal Fluid (CSF) and blood sampling at two study visits (baseline and after 12 months). Major secondary objectives are (i) to assess potential associations between the biomarker and clinical measures of disease severity and progression in MSA and PSP, and (ii) to assess the variation of the biomarker and its correlation to disease severity and progression in PD, MSA and PSP.

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.

Lymphatic anomalies are a rare subset of vascular anomalies that are poorly understood. the understanding of the natural history, long-term outcomes, risk factors for morbidity and mortality, and the relative benefit of medical therapies and procedures is limited.The goal of this project is to better understand these diseases and improve the care of theses rare patients. To do this, the investigators are conducting an observational study of patients with lymphatic anomalies, including an annual follow-up questionnaire to gather prospective data on mortality, morbidity, treatments, and functionality as well as quality of life.