View clinical trials related to Syndrome.
Filter by:This phase I trial studies the side effects and best dose of CD4+ and CD8+ HA-1 T cell receptor (TCR) (HA-1 T TCR) T cells in treating patients with acute leukemia that persists, has come back (recurrent) or does not respond to treatment (refractory) following donor stem cell transplant. T cell receptor is a special protein on T cells that helps them recognize proteins on other cells including leukemia. HA-1 is a protein that is present on the surface of some peoples' blood cells, including leukemia. HA-1 T cell immunotherapy enables genes to be added to the donor cells to make them recognize HA-1 markers on leukemia cells.
Nephrotic syndrome (NS) represents one of the most common diagnoses in pediatric and adult nephrology, with a prevalence of 16 per 100,000 children and 3 per 100,000 adults in Western countries. In most cases, the pathogenesis of NS remains elusive, and the clinical phenotype of patients does not allow discrimination among different causes. Thus, children with NS are usually treated with corticosteroids before a biopsy is taken, and approximately 80% of them respond to such a treatment. According to this observation, pediatric NS has been separated into two broad categories; Steroid-Sensitive Nephrotic Syndrome (SSNS) and Steroid-Resistant Nephrotic Syndrome (SRNS). In both these categories the biopsy result is usually Minimal Change Disease (MCD) while a few may show Focal and Segmental Glomerulosclerosis (FSGS). Although children affected by SSNS have good long-term prognosis, most patients with SRNS progress to End Stage Renal Disease (ESRD) within 2-10 years of diagnosis . In adults a biopsy diagnosis of FSGS is more common than in children and more patients will not respond to corticosteroids alone and will need additional immunosuppressant medication. About 40% will progress to ESRD within 10 years . Currently, at least 19 genes have been clearly identified with association to SRNS harboring ~300 independent mutations, conferring a considerable genetic heterogeneity to the disorder. Genetic testing is emerging as a useful diagnostic tool in SRNS as it has implications for clinical course, treatment response, risk for posttransplant proteinuria and prenatal diagnosis. An approach for genetic testing based on the current evidence seems cost-effective and may help in the best possible management of SRNS . The NPHS2 gene, is located on chromosome 1 and is also known as the Podocin gene. It encodes the podocin protein. Podocin is a 383-amino acid lipid-raft-associated protein localized at the slit diaphragm, where it is required for the structural organization and regulation of the glomerular filtration barrier. Its interaction with other slit diaphragm proteins eg. nephrin, NEPH1, CD2AP and TRPC6 is important in mechanosensation signaling, podocyte survival, cell polarity, and cytoskeletal organization . It has been reported that variants in the NPHS2 gene are associated with NS . The commonly studied rs61747728 NPHS2 gene polymorphism also known as p.R229Q has been reported to be associated with NS and SRNS . However others have failed to report an association , which might be due to population differences. The rs61747728 is a non-synonymous variant found on exon 5 which is suggested to be involved in in altering the functional properties of podocin in vitro and possibly in vivo . The investigators will therefore investigate the frequency of the p.R229Q variant in Middle East patients with NS. Genetic analysis will have important implications in several aspects:- 1. Understanding the biology of the disease in this part of the world. 2. Counselling patients about their clinical course and what medication they will respond to. 3. Counselling patients about the possibility of a kidney transplant sooner in their disease course
Long term assessment of ovarian reserve after more than two years of laparoscopic ovarian drilling in Polycystic Ovary Syndrome
This is a Phase 2b, randomized, double-blind, placebo-controlled, dose-optimization study to evaluate the efficacy, safety, and tolerability of NBI-98854 titrated to the subject's optimal dose administered once daily (qd) for a total of 12 weeks of treatment in pediatric subjects with TS.
Prader-Willi Syndrome (PWS) is a multisystemic genetic disease characterized by hypotonia, mental retardation, hyperphagia, and uncontrollable hunger due to hypothalamic dysfunction, caused by dysregulation of genes located in chromosome 15q11-q13. The goal of this study is to evaluate the effects of Transcranial Direct Current Stimulation (tDCS) on hyperphagia and behavior in PWS. Forty children and adolescents (11-24 years) with clinical and cytogenetic-molecular diagnosis of Prader-Willi syndrome will be assessed before and after 10 tDCS session with: Food Craving Questionnaire (FCQ), Aberrant Behavior Checklist (ABC), Dykens hyperphagia questionnaire. Caregivers self-reported the participant's behaviors at home and, lately, they will be categorized and quantified. tDCS will be applied for 20 minutes with electrodes of 25cm2 wrapped in cotton material soaked in saline solution. The anode at the left dorsolateral prefrontal cortex (F3) and the cathode at the contralateral area (F4). Children from 11-12 years will receive a current of 1mA; above 13 years, 2mA.
This is a minimal risk case-controlled single arm intervention study, including 10 patients with a prior history of takotsubo and 10-age and sex matched healthy controls. Subjects will undergo in laboratory testing to measure autonomic function. They will then undergo a 15-week program of device-guided breathing with remote measures of autonomic function obtained at home. Analysis will determine the reproducibility of home autonomic measures and the provide preliminary data to determine the efficacy of device-guided breathing on autonomic measures and quality of life in patients with takotsubo.
This study aim to investigate the effects of physium therapy for improving pressure pain thresholds (PPTs), range of motion,Neck Disability Index, the multidimensional health related quality of life (SF-12) and the multidimensional health related quality of life and pain in patients with mechanical neck pain (NP).
This study is aimed to characterize Russian population of Usher patients.
Dry eye complaints occur in 5.5 to 33.7% of the population, and are ranked as the most frequent symptoms of patients visiting ophthalmologists. Dry eye syndrome is caused by the reduced production and/or improper quality of the tear film. One of the causes of reduced tear production is Sjögren's syndrome. Sjögren's is estimated to affect up to 4 million patients in the US alone. It affects mostly middle aged women (40-50 years of age) with a female to male prevalence ratio of 9:1. The current study seeks to evaluate the safety and efficacy of LO2A ophthalmic solution in the symptomatic treatment of dry eye in patients with Sjögren's syndrome. This study will be conducted in compliance with the protocol, GCP,and applicable regulatory requirements.
A 3-month study was conducted in flood victims from affected villages in the Tumpat district, Kelantan. Participants were given either probiotic, Bifidobacterium infantis M63 (M-63 group) or no probiotics (control group) for three months. At baseline and 3-month, participants were assessed for thewater, sanitation and hygiene (WaSH) practices, abdominal symptoms, breath testing for hydrogen and methane to detect the presence of SIBO and also fecal samples for gut microbiota profiling.