Active clinical trials for "Syndrome"

Alport syndrome is a rare, inherited condition characterized by a combination of glomerular nephropathy progressing to kidney failure, deafness, and eye involvement. This disease is associated with mutations in the genes encoding one of the three IV collagen chains expressed in the glomerular basement membrane. Significant progress has been made in understanding the molecular mechanisms responsible for the disease, but relatively little in understanding the progression of renal failure and in the area of therapeutics. We have shown in a retrospective European study that blockers of the renin angiotensin system may slow disease progression, but no controlled studies have been performed. Finally, innovative therapies (anti-micro-RNA, stem cells) have recently shown their effectiveness in animal models of the disease, and industrials are planning to quickly carry out phase 1 trials to test molecules. Carrying out therapeutic trials in humans will require full knowledge of the natural history of the disease (isolated hematuria, microalbuminuria, macroalbuminuria, renal failure and its progression) and gathering a sufficient number of patients, especially in the early stages. These trials and the indications for treatments would be greatly facilitated by the discovery of biomarkers that make it possible to predict the progression to renal failure earlier than the onset of proteinuria. The study aims to: Establish a European database on Alport syndrome to assess the natural history of the disease. To investigate the impact of the disease on the educational and professional life of patients and their families, and on the adherence and tolerance to renin-angiotensin system blockers prescribed to proteinuric patients. Investigate access to molecular diagnostics and genetic counseling, as well as identify biomarkers that can predict progression of kidney disease. This project will be carried out at a French level with the support and participation of the very active renal rare disease sector, in collaboration with various countries wishing to participate.

The study, identified as VIX001-PACS-01, is a Phase 1, open-label, dose-escalation trial evaluating the safety, tolerability, preliminary efficacy, and dose effect of VIX001, an amniotic fluid product, in patients with Post-Acute COVID-19 Syndrome (PACS) and cognitive impairment. Conducted at the University of Miami Hospital and Clinics, the trial aims to enroll up to nine participants, or up to 18 using a 3+3 dose escalation design. Intravenous injections of VIX001 will be administered at three ascending doses (1 ml, 3 ml, or 10 ml), and participants will be assessed for safety, cognitive impairment, pain, activity, and quality of life at baseline and various timepoints. The primary objective is to evaluate the safety of VIX001, while secondary objectives include assessing its potential efficacy and patient-reported outcomes. The study duration is expected to last approximately 18 months, including enrollment, evaluation, and post-study observation periods. The findings will contribute to understanding VIX001's safety and efficacy in treating PACS-related cognitive impairment.

According to World Health Organization (WHO), in 2010, Polycystic Ovarian Syndrome (PCOS) affected approximately 116 million women worldwide (3.4% of the population). It has been considered one of the most common causes of female infertility and the most common endocrine disorder. The standard diagnosis for the syndrome dates back to international conferences organized by the National Institutes of Health (NIH) in 1990 and the Rotterdam European Society of Human Reproduction and Embryology/ American Society for Reproductive Medicine (ESHRE/ASRM) sponsored PCOS consensus workshop group in 2003 and 2004. Clinical manifestations of the disease may include menstrual irregularities, amenorrhea, ovulation-related infertility, polycystic ovaries, and signs of androgen excess such as acne and hirsutism. This condition may also lead to chronic diseases such as obesity, type 2 diabetes (T2D), dyslipidaemia, and cardiovascular events. Despite the increasing knowledge concerning PCOS, the global picture of the disorder is deficient in a number of geographic regions. Understanding the global prevalence will help to better assess the public health and economic implications of PCOS in Trinidad, allow for improved screening methods, help elucidate the underlying factors and foster improved understanding of the molecular mechanisms in improving the evolutionary process.

Polycystic Ovary Syndrome (PCOS) is a complex endocrine disorder characterized by hyperandrogenism, insulin resistance, and ovulatory dysfunction. One of the diagnostic criteria for PCOS is the presence of polycystic ovaries, which are enlarged ovaries with multiple follicles on ultrasound. However, the mechanisms that lead to the development of polycystic ovaries in PCOS are not completely understood. One potential area of investigation is the role of ovarian innervation in the pathophysiology of PCOS. Therefore, the aim of this study is to evaluate the ovarian innervation in PCOS patients through ovarian biopsy sampling.

Dapagliflozin is the first SGLT2 inhibitor to be approved for CKD treatment regardless of diabetes status. Since many etiologies of non-diabetic nephropathy are characterized by intraglomerular hypertension, it was hypothesized that dapagliflozin acutely decreases GFR and proteinuria in patients without diabetes at risk of progressive kidney function loss such as nephrotic patients via a glucose independent hemodynamic mechanism. The aim of the study is to assess the effect of Dapagliflozin on proteinuria and estimated glomerular filtration rate in non-diabetic patients with nephrotic syndrome in order to slow the decline in kidney function and the progression to ESRD and to prevent the complications of nephrotic syndrome like thrombotic diseases, peritonitis, hyperuricemia, and recurrent infections.

This is an observational, multi-center, longitudinal cohort study to characterize adults with DS ages 25 years and above enrolled at specialized care centers. The aim is to assess changes in cognition, behavior, function and health over approximately 32 months. Blood will be collected for the development of plasma AD biomarkers useful in the DS population.

The primary objective is to quantify the degree of pain relief in patients undergoing gonadal vein embolization with coils as well as identify clinical or imaging factors that are predictive of a positive response to treatment, or poor response to treatment.

This is a phase 3, multicenter, double-blind, randomized, placebo-controlled, parallel-group study to evaluate the efficacy and safety of CSL112 on reducing the risk of major adverse CV events [MACE - cardiovascular (CV) death, myocardial infarction (MI), and stroke] in subjects with acute coronary syndrome (ACS) diagnosed with either ST-segment elevation myocardial infarction (STEMI) or non-ST-segment elevation myocardial infarction (NSTEMI), including those managed with percutaneous coronary intervention (PCI) or medically managed.

The objective of this protocol is to develop an institution-wide liquid biopsy protocol that will establish a common process for collecting blood and corresponding archived tumor specimens for future research studies at the University Health Network's Princess Margaret Cancer Centre. Circulating cell-free nucleic acids (cfNA), including cell-free DNA (cfDNA) and cell-free RNA (cfRNA), are non-invasive, real-time biomarkers that can provide diagnostic and prognostic information before cancer diagnosis, during cancer treatment, and at disease progression. Cancer research scientists and clinicians at the Princess Margaret are interested in incorporating the collection of peripheral blood samples ("liquid biopsies") into research protocols as a means of non-invasively assessing tumor progression and response to treatment at multiple time points during a patient's course of disease.

This phase II trial studies how well nivolumab works in preventing colon adenomas in participants with Lynch syndrome and a history of surgery to remove part of the colon. Monoclonal antibodies, such as nivolumab, may interfere with the ability of tumor cells to grow and spread.