View clinical trials related to Syndrome.
Filter by:The aim of this study is to determine if treatment with Medical Air (21% oxygen in room air) compared to supplemental oxygen (100% oxygen) will lead to similar improvements in the central apnea-hypopnea index (CAHI) for infants with Prader-Willi Syndrome. Despite the vast amount of research investigating the cause of central sleep apnea, there remain gaps in knowledge, lending to further research efforts. The decision to compare oxygen to medical air is based on several theorized mechanisms. The first of which is the supposition that provision of medical air may act as an arousal stimulus for the hypothalamus, thereby preventing sleep disordered breathing. Secondly, the hypercapnic challenge performed by Livingston et al demonstrated a delayed hypercapneic arousal response in PWS subjects despite simultaneous hyperoxia, leading us to question if therapeutic oxygen really plays a significant role in treating CSA. Lastly, the delivery of medical air via nasal prongs may provide sufficient arousal to terminate the cycle of events leading to central apnea, as described by Urquhart et al. A deeper understanding of central sleep apnea is essential to ameliorating its adverse sequelae, which include symptoms of ADHD, impaired attention, behavioral problems, and academic difficulties.
A single-center, self-control, open-label study of efficacy and safety of Iguratimod in patients with Sjögren's Syndrome
Treatment Study to assess of safety and efficiency of conditioning with Plerixafor and G-CSF as additional agents for prevention of graft failure after transplantation with TCR alpha/beta grafts depletion in patient with Wiskott-Aldrich syndrome.
This phase I trial studies the best dose and side effects of recombinant vesicular stomatitis virus carrying the human NIS and IFN beta genes (VSV-hIFNbeta-sodium iodide symporter [NIS]) with or without cyclophosphamide or ipilimumab and nivolumab or cemiplimab in treating patients with multiple myeloma, acute myeloid leukemia (AML) or lymphoma that has come back or does not respond to treatment. A virus, called VSV-hIFNbeta-NIS, which has been changed in a certain way, may be able to kill cancer cells without damaging normal cells. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's DNA and may kill cancer cells. It may also lower the body's immune response. Immunotherapy with ipilmumab and nivolumab or cemiplimab may induce changes in body's immune system and may interfere with the ability of tumor cells to grow and spread. Giving VSV-hIFNbeta-NIS and ruxolitinib phosphate may work better at treating multiple myeloma, acute myeloid leukemia and T-cell lymphoma.
This is a prospective, randomized trial comparing different doses of rosuvastatin in patients with acute coronary syndrome post drug-eluting stents implantation. Through the study, the investigators aim to evaluate the effects of high dose rosuvastatin calcium on "target vessel" endothelialization and "non-target vessel" plaque stability. Moreover, the investigators may provide mechanically evidence of clinical application of high dose rosuvastatin in patients with acute coronary syndrome.
Primary Sjögren's syndrome (pSS) can affect various organs, sometimes leads to life-threatening conditions and is always responsible for a decreased quality of life. Its evolution is chronic, with flares and relapses, and the need for reliable biomarkers to be carried out routinely is major in patients' follow-up. Because of the existence of autoreactive immunoglobulins E (IgE) in autoimmune diseases, the recently described role for anti-Ro/SSA antibodies in inducing interferon alpha (IFNα) signaling and the specific pharmacologic properties of IgE, anti-Ro/SSA IgE should be an interesting biomarker to determine pSS's activity. The aim of the study is to evaluate whether the proportion of anti-Ro/SSA IgE positive patients is higher in patients with active disease (i.e. Eular Sjögren Syndrome Disease Activity Index≥ 5). All consecutive patients with pSS (new or already known diagnosis) will be included, Anti-Ro/SSA IgE titers will be determined, the disease's features will be collected (including Eular Sjögren Syndrome Disease Activity Index/Eular Sjogren's Syndrome Patient Reported Index).
The investigators are registering all nephrotic syndrome (NS) patients regardless of the primary causes and developing a NS database in China. Patients will be followed-up and both baseline and follow-up information will be recorded in the registration system. The treatment response, longitudinal changes of renal function, renal survival, patient survival, infection events and acute kidney injury etc, will be analyzed using the NS database.
The NATIENS study is a phase III randomized study to examine the optimal treatment and mechanisms of each of two treatments (cyclosporine 5 mg/kg bid for 14 days versus etanercept 50 mg subcutaneously at day 0 and day 3) versus the current standard of care which is harmonized supportive care for the treatment of Stevens-Johnson Syndrome and toxic epidermal necrolysis (SJS/TEN). SJS/TEN is typically a drug-induced disease in adults with a mortality of up to 50% or higher in elderly adults. Although progress has been made in elucidating strong genetic risk factors that have led to pre-prescription screening and prevention the risk factors for most drugs and ethnicities represented in the United States are currently unknown. Currently there are a number of small observational studies and a non-blinded small randomized study however there is no strong or definitive evidence base to support any one treatment intervention over supportive care alone and this remains considered a standard of care for SJS/TEN. The primary objective of the study is to conduct a randomized double-blind double dummy stratified multicenter phase III study across 24 sites across the Unites States to determine whether two therapeutic interventions (etanercept versus cyclosporine) will improve short-term outcomes associated with SJS/TEN. The primary hypothesis of this study is that both etanercept and cyclosporine will show benefit over supportive care alone and that single dose etanercept 50 mg sc at days 0 and repeated 72 hours following initial dosing will show significant benefit over cyclosporine 5 mg/kg bid and supportive care alone. Our secondary outcomes are to determine the clinical outcomes at 3 and 12 months following initial presentation and to determine the molecular and cellular mechanisms of SJS/TEN through collection of timed samples to include DNA, RNA, PBMCs, blister cells and supernatant and skin. We hypothesize that patients will have significant sequelae identified at 3 and 12 months that will differ between treatment arms and that treatment interventions will significantly impact cytotoxic and cytokine signals with these biomarkers correlating with primary and secondary outcome. We also hypothesize that significant genetic associations will be found in association with drug-induced SJS/TEN. Eligible patients are >/= 18 who meet evidence for SJS/TEN clinical criteria as evidence by erythematous/dusky macules coalescing or denuded skin and blistering with positive Nikolsky sign which is mandatory criteria associated with mucous membrane involvement, prodromal symptoms including fever, myalgia and headache, increasing number of lesions and history of a medication. To continue with the study patients must meet pathological criteria. Randomization will occur by a secure central online computer-generated random number system through REDCap. Subjects will be allocated 1:1:1 to cyclosporine plus best supportive care, etanercept plus best supportive care or best supportive care alone. Patients, treating physician and outcome assessors will be blinded to the allocated treatment. The primary outcome of the study is time to complete re-epithelialization as defined by complete absence of erosion and compromised skin. Time to expected re-epithelialization of 21 days is the maximum healing time with supportive care in SJS/TEN patients which reflects the healing time of adult skin. The primary outcome will be independently assessed by the treating team to include any of a burn surgeon, dermatologist or wound specialist. Disagreement will be solved by independent adjudication by a minimum of two reviewers. Patients who have to discontinue a study medication will be analyzed by intent-to-treat analysis and followed for complications of SJS/TEN as per study protocol. Secondary outcomes of the study include: 1)time to halting of progression of SJS/TEN skin disease. Progression will be considered significant if there are any new blisters or erosions and halting of progression is defined as absence of these criteria with any new lesions; 2) all-cause mortality at 30 days, 3 months and 1 year following symptoms onset; 3) composite cause-specific mortality - outcome including death from sepsis, multi-organ failure and acute respiratory distress syndrome; 4) actual mortality versus expected mortality (as calculated by SCORTEN); 5) Time to cessation of acute ocular involvement (this will be tracked by the same serial photography evaluated by two independent Ophthalmology experts in SJS/TEN eye disease; 6) incidence of infections; 7) hospital length of stay; 8) adverse events due to therapy; 9) serial plasma granulysin, IL-15 concentrations (and other relevant biomarkers);10) Follow-up 3 months and 1 year from initial presentation for physical and mental health complications. For aims 2 and 3 a number of mechanistic studies will be performed on paired samples (DNA, RNA, PBMCs, plasma, blister fluid and skin).
Obstructive sleep apnea (OSA) and Obesity-Hypoventilation Syndrome (OHS) are common conditions in obesity, which may influence the prognosis in patients undergoing surgery. There is a need for simple screening tools to identify such patients at high risk. The current multicenter observational study aims to investigate occurrence of OSA and OHS in obese individuals undergoing elective abdominal surgery and further address its impact on perioperative and postoperative complications.
Horner's syndrome (HS) is a result of interruption of the sympathetic innervation to the eye and ocular adnexa, which can occur due to carotid artery dissection (CAD), along which the sympathetic nerve fibers travel to reach the eye. Dissection of the carotid artery is one of the etiologies for ischemic stroke in young patients. In our current study, we will explore the diagnostic accuracy of automated binocular pupillometry in the diagnosis or HS, and its potential in identifying dissection of the carotid artery in patients of young age who suffer ischemic stroke.