View clinical trials related to Syndrome.
Filter by:This phase II trial studies methylprednisolone, horse anti-thymocyte globulin, cyclosporine, filgrastim, and/or pegfilgrastim or pegfilgrastim biosimilar in treating patients with aplastic anemia or low or intermediate-risk myelodysplastic syndrome. Horse anti-thymocyte globulin is made from horse blood and targets immune cells known as T-lymphocytes. Since T-lymphocytes are believed to be involved in causing low blood counts in aplastic anemia and in some cases of myelodysplastic syndromes, killing these cells may help treat the disease. Methylprednisolone and cyclosporine work to suppress immune cells called lymphocytes. This may help to improve low blood counts in aplastic anemia and myelodysplastic syndromes. Filgrastim and pegfilgrastim are designed to cause white blood cells to grow. This may help to fight infections and help improve the white blood cell count. Giving methylprednisolone and horse anti-thymocyte globulin together with cyclosporine, filgrastim, and/or pegfilgrastim may be an effective treatment for patients with aplastic anemia or myelodysplastic syndrome.
This is a double-blind randomized controlled trial to evaluate the efficacy of the sympathetic block via thoracic vertebra T3 for the treatment of CRPS I upper limb. Patients with CRPS I refractory to medical treatment will be subjected to four physical therapy sessions and then the randomized for experimental or control block and then more four physiotherapy sessions. Patients will be evaluated after one month of the blockade (primary outcome) and then up to 12 months. Will be evaluated by analgesic scale (Mcgill, brief pain inventory, dn4 questionnaire, NPSI, VAS), functional (ADM) and quality of life (HAD and WHOQOL-brief).
INSIGHT is a longitudinal study of childhood nephrotic syndrome to determine genetic, serologic and environmental factors contributing to nephrotic syndrome and disease progression.
The purpose of this study is to explore the genetic basis of reproductive disorders and cleft lip and/or palate.
Obstructive Sleep Apnea Syndrome (OSAS) is associated with stroke as a risk factor but little is known about the consequences of OSAS on the outcome and the survival after stroke. The aim of the investigators study is first to evaluate the outcome and the survival of patients with stroke depending of OSAS (presence and severity of OSAS) and second to compare the outcome and survival of patients with severe OSAS depending on the treatment of the syndrome with nocturnal continuous positive airway pressure. The investigators hypothesis is that OSAS is associated with worst survival and outcome and needs to be treated at the subacute phase of stroke.
The proposed phase II trial is a multicenter, randomized, open-label study that will evaluate the efficacy and safety of azacitidine alone or in combination with lenalidomide in high-risk Myelodysplastic Syndrome (MDS) or Acute Myeloid Leukemia (AML) with a karyotype including del(5q). The primary objective will be to evaluate the efficacy in terms of response according to International Working Group (IWG) criteria for MDS and AML after 6 cycles of azacitidine or azacitidine + lenalidomide treatment, or at end of study if this occurs at an earlier time point.
Netherton Syndrome is a serious skin disorder caused by damage in a gene called SPINK5. This gene controls the formation of a protein called LEKTI, which important for skin barrier function. LEKTI inhibits certain enzymes (serine proteinases) in the outermost layer of the skin (epidermis). The function of the serine proteinases is to break down the intracellular cement that holds together the horny cells in the epidermis, in order for the skin to be able to shed cells (known as cell desquamation). LEKTI deficiency leads to an uninhibited desquamation of horny cells, and as a result the skin becomes red and scaly. The barrier function of the skin is also affected. The permeability of the skin increases, and its capacity to bind water decreases, which causes dryness. The thinness of the barrier also results in over absorption of chemicals, for example topical medical treatments. Historically one in ten infants dies before their first birthday. Currently there are no proven treatments to cure this condition. The investigators have been developing a gene therapy approach to treat this disorder. The investigators have used a disabled virus (vector) to carry a functional copy of the SPINK5 gene into skin stem cells. Proof-of-principle experiments have shown the investigators can restore almost normal shape and size of the upper layer of the skin in skin grafts grown in the lab. Even if only a small number of cells are genetically modified to carry the corrected SPINK5 gene, there seems to be a correction over a wide area of the graft. In this trial the investigators propose grafting of autologous epidermal sheets generated from genetically modified skin stem cells for the treatment of patients with Netherton Syndrome. The investigators anticipate production and release of LEKTI protein from even a small patch of skin will be beneficial.
The purpose of this study is determine the incidence of metabolic syndrome (MetS) in people of high risk, of 40 or more years and attended in the Primary Care. Evaluating the association between anxiety, depression, quality of life and the vital stressful events, and the development of the metabolic syndrome in general population. Our hypothesis is that population of the cohort with bigger degree of stress will develop earlier the metabolic syndrome. If our hypothesis about the metabolic syndrome are demonstrated, it would allow establishing in a future interventions on these factors of risk to prevent or to decrease the incidence of this syndrome in the Primary Care.
In this open prospective study 40 patients who received Mandibular Replacement Appliance (MRA) Therapy as treatment of Obstructive Sleep Apnea Hypopnea Syndrome (OSAHS) will be included. The patient's sleep will be registered during 2 periods of 2 days: one while the patient does not wear the MRA and, +/- 1 week later, one while the patient wears the MRA. Furthermore, 2 low dose CT scans (one with and one without the MRA device in the mouth) will be taken. These data must allow the investigators to validate the use of functional imaging (segmentation and Computational Fluid Dynamics) as a predictor of the outcome of the MRA therapy in OSAHS patients.
Post-marketing safety data on patients treated and untreated with eculizumab or ravulizumab.