Active clinical trials for "Syndrome"

"The purpose of this study is to assess the safety and efficacy of oral administration of itacitinib for the prevention of cytokine release syndrome (CRS) in male or female participants aged 12 years or older and who are planning to receive an approved immune effector cell (IEC) therapy for hematologic malignancies.

This phase Ib trial investigates the side effects of the combination of nivolumab and ipilimumab, and to see how well they work in treating patients with cancers that have come back (relapsed) or does not respond to treatment (refractory) and have an increased number of genetic changes. Immunotherapy with monoclonal antibodies, such as nivolumab and ipilimumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Tumor mutational burden (TMB) is the total amount of genetic changes or "mutations" found in tumor cells. Some studies in adults with cancer have shown that patients with a higher TMB (an increased number of genetic changes) are more likely to respond to immunotherapy drugs. There is also evidence that nivolumab and ipilimumab can shrink or stabilize cancer in adult patients with cancer. This study is being done to help doctors learn if the combination of nivolumab and ipilimumab can help children, adolescents, and young adults patients live longer.

Obstructive Sleep Apnea Syndrome (OSA) is one of the most common chronic diseases and is associated with prognostic cardiovascular and metabolic co-morbidities. OSAS is the recurrence of complete (apnea) or partial (hypopnea) collapse of the upper airway during sleep resulting in sleep fragmentation and chronic intermittent hypoxia (ICH) which are the major determinants of cardiovascular and metabolic complications including type 2 diabetes, obesity and non-alcoholic fatty liver. These comorbidities are associated with a change in intestinal microbial ecology. In most cases, there is a reduction in bacterial genetic diversity and more or less specific signatures of cardiovascular and metabolic diseases, making it possible to envisage personalized and innovative therapeutic treatments. In animals exposed to intermittent hypoxia, there is local hypoxia that increases intestinal permeability, produces a reduction in microbiota diversity and favors microbial species that are at the origin of pro-inflammatory factors. Continuous Positive Airway Pressure (CPAP) is the standard treatment for OSA. Its effect on the intestinal microbiota has not yet been evaluated.

This uncontrolled feasibility pilot study explores the acceptability and potential effect of a 14 week, 11-module, therapist-assisted, internet-delivered treatment program, "One step at the time", for patients moderately affected by Bodily Distress Syndrome. The study includes 25 participants aged 18-60 with multiple functional somatic symptoms for a duration of minimum 6 months. The focus of the feasibility trial is evaluation of treatment response, treatment satisfaction, program utility, recruitment and retention rates, data completion rates, and time requirement. The primary feasibility criterion is a +2.0 points change in patient-rated physical health measured by the SF-36 aggregate score physical health from before to after treatment.

The DO OAM study is a mono-center, interventional, prospective study carried out in CH VALENCE. It will estimate the prevalence of dental movements in patients treated by oral Appliance for Sleep Apnea syndrome. The investigators will used fingerprinting technique by taking multiple photographs of the teeth that allow for the three-dimensional reconstruction of the teeth this study will provide a better understanding of dental movement caused by oral appliance

The therapeutic management of Obstructive Sleep Apnoea Syndrome in children remains a debated subject, only otorhinolaryngology surgery (adenoidectomy) has been studied on a large scale. Pathophysiologically, increased pharyngeal collapsibility is a major endotype of the disease and the investigators have shown that this surgery can improve pharyngeal compliance. The development of approaches to treat pharyngeal hypotonia by maxillofacial rehabilitation supports the treatment of this endotype. A study in adults showed a benefit from playing the didgeridoo, a wind instrument, for 3 months, without pathophysiological explanation. The investigators hypothesise that playing this instrument improves pharyngeal compliance (re-education effect) in a similar way to the effect observed after otorhinolaryngology surgery. This proof-of-concept study aims to demonstrate the effect of didgeridoo in children without syndromic pathology with a formal otorhinolaryngology surgical indication resulting from tonsillar hypertrophy (Brodsky grades III and IV) and symptomatology suggestive of Obstructive Sleep Apnoea Syndrome (Pediatric Sleep Questionnaire score ≥ 0.33). The investigators will take advantage of the long delay in performing the adenoidectomy (~6 months) in their university hospital to evaluate, before the scheduled surgery, the effect of the didgeridoo used for three months.

Congenital hyperinsulinism (HI) is a rare disorder of pancreatic beta cell insulin secretion that causes persistent and severe hypoglycemia starting at birth. Hyperinsulinism/hyperammonemia (HI/HA) syndrome is the second most common type of congenital HI and is caused by activating mutations in glutamate dehydrogenase (GDH). Patients with HI/HA exhibit fasting hyperinsulinemic hypoglycemia, protein-induced hypoglycemia, hyperammonemia, seizures, and intellectual disability independent of hypoglycemia. These effects result from abnormal GDH activity in the beta cells, liver and kidney cells, neurons, and astrocytes. The only available treatment for HI/HA syndrome is diazoxide, which acts on the beta cells to decrease insulin secretion but has no effect on GDH activity itself or on other cell types. Thus, there remains a significant unmet need for improved therapies for this disorder. Pre-clinical data show that vitamin E inhibits GDH activity in human cell lines and improves fasting hypoglycemia in a GDH HI mouse model. Pilot study data show that vitamin E supplementation with a moderate dose is well-tolerated in children and adults with HI/HA syndrome, while continuing diazoxide treatment. However, most subjects continued to exhibit protein-induced hyperinsulinemic hypoglycemia. We hypothesize that a higher vitamin E dose will inhibit GDH over-activity in subjects with HI/HA syndrome, resulting in improved hyperinsulinemic hypoglycemia, reduced blood ammonia concentration, and decreased seizure activity.

At the beginning COVID-associated lung injury was considered as typical ARDS, hence respiratory and nonrespiratory treatments were delivered according to general principles for this kind of illness. There is hypothesis that in predisposed individuals, alveolar viral damage is followed by an inflammatory reaction and by microvascular pulmonary thrombosis. The investigators suggest that thrombolytic therapy may be beneficial when compared to standard care in patients with SARS-CoV-2 and severe respiratory failure.

Since subjects with Down Syndrome (DS) have different and variable levels of intellectual deficit, in approaching the patient with DS and before evaluating the different therapeutic strategies and carrying out a dental and orthodontic treatment, an initial analysis would be useful the patient's level of cooperation. The hypothesis underlying the study is the possibility of predicting the degree of success of dental and orthodontic treatment in the patient with Down's Syndrome by completing an evaluation form by the clinician. The general objective of the study is to test the effectiveness of the module in assessing and quantifying the degree of patient collaboration. This form is filled in entirely during the first visit and updated in subsequent sessions in conjunction with the different operational phases. Based on the score obtained by the patient, the clinician can hypothesize the degree of cooperation during the operative sessions and compliance with the indications provided. Therefore, the evaluation of the score obtained will guide the clinician in choosing the type of therapy that will have the least risk of failure because this will be chosen having evaluated not only the dental and/or orthodontic therapeutic needs, but also the real ability of the patient to collaborate.

This study will evaluate whether the anti-inflammatory effect of colchicine is beneficial in ACS patients