Active clinical trials for "Syndrome"

The investigators conducted a prospective un-blinded pilot study of Vitamin D plus Calcium (Ca) supplementation in overweight (BMI > 27) premenopausal women diagnosed with Polycystic Ovarian Syndrome (PCOS), as defined by the Rotterdam Criteria, 2003, and who were deficient in vitamin D as reflected by serum 25-hydroxy (25-OH) vitamin D (serum levels < 20 ng/mL).

This study will evaluate the safety, tolerability and efficacy of multiple doses of AFQ056 in patients with Fragile X Syndrome. The dose range will be 50 to 150 mg b.i.d. The primary read-out of efficacy is reduction in Aberrant-Behavior Checklist score.

Pudendal neuralgia is a recent identified pathology, extremely invalidating, related to chronic pelvic entrapment. Nowadays, pudendal neuralgia can be treated with: neuropathic pains treatment specific kinesitherapy Alcock's canal and sacrospinal ligament infiltrations under scan with diagnostic block local steroids injections and surgical decompression of pudendal nerve with transrectal approach. Only surgery was validated after a randomised protocol studying surgery versus abstention, performed and published by the CHU de Nantes. Many techniques have been proposed for realization of pudendal nerve infiltrations. The results of these infiltrations have never been published, and no randomised study had ever evaluated those results, even at short-run. Very few randomized studies have validated steroids infiltrations techniques in canal syndrome neuropathies. The primary objective of the investigators phase IV trial is to evaluate the efficacy of three different types of pudendal nerve infiltrations in Alcock's canal and sacrospinal ligament: group A: only local anesthetic (control arm) group B: local anesthetics associated with local steroids group C: local anesthetics associated with local steroids and important volumes of physiological serum

This is a 24-month study of the use of laronidase administered into the spinal fluid to treat cognitive decline in mucopolysaccharidosis I (MPS I). MPS I is a rare genetic condition due to deficiency of the enzyme alpha-l-iduronidase. Laronidase is the manufactured form of the enzyme alpha-l-iduronidase. MPS I is a heterogeneous disease with several clinical phenotypes ranging from the most severe, Hurler syndrome, to the attenuated forms, Hurler-Scheie and Scheie. Although patients with milder forms of MPS I may not have grossly observable problems with cognition, these patients do have learning difficulties that are apparent in school and with neuropsychological testing. The goal of this study is to evaluate whether intrathecal recombinant human alpha-l-iduronidase (rhIDU) injections can stabilize or improve cognitive decline in individuals with MPS I.

Life for long-term bone marrow transplant patients is complicated by endocrine late effects including growth hormone (GH) deficiency, thyroid hormone deficiency and sex steroid deficiency. Recently, studies have also identified problems with metabolic syndrome in adult bone marrow transplant (BMT) survivors. Metabolic syndrome has been identified as a constellation of insulin resistance, truncal obesity and high lipid levels (dyslipidemia) and is associated with an increased risk of type 2 diabetes and cardiovascular disease. Thus the early identification of metabolic syndrome is important. To date, studies have not identified how young an age metabolic syndrome begins in BMT survivors. The investigators' study will consist of two aims: Evaluation of children who have survived BMT for growth hormone deficiency, abnormal lipid metabolism, hypothyroidism and gonadal dysgenesis. The investigators will utilize growth hormone stimulation testing, sex steroid levels, an oral glucose tolerance test (OGTT) and fasting lipid profile to evaluate for concomitant endocrinopathy, prediabetes and impaired glucose tolerance in a cohort of BMT survivors. Cross-sectional study of peripheral and hepatic insulin sensitivity in children surviving BMT using a hyperinsulinemic euglycemic clamp and the stable isotope 6,6 [2H2] glucose. These aims will provide pilot data to power the first definitive study of insulin resistance in childhood BMT survivors.

The primary objective of this study is to evaluate the efficacy and safety of ON 01910.Na Concentrate when it is administered as an intravenous continuous infusion (IVCI) over 72 hours once every 2 weeks in a broad population of MDS patients. Rationale for this study is based on the activity observed in another study with ON 01910.Na in patients with refractory anemia with excess blasts (RAEB) 1 and 2 MDS. This study will examine ON 01910.Na in a broader population of MDS and AML patients. This phase I/II study will establish the Maximum Tolerated Dose (MTD) starting with a dose of 800 mg/m2 per day administered over 24 hours for 2 consecutive days as a continuous intravenous infusion, once a week for 3 weeks of a 4-week cycle and examine the efficacy and safety profile at the MTD.

An enteral diet supplemented with ginger extract in acute respiratory distress syndrome (ARDS) patients may be beneficial for gas exchange and could decrease duration of mechanical ventilation and length of stay in intensive care unit (ICU).

Primary objective: To assess the effect of rimonabant on visceral fat area over a period of 12 months when prescribed with a mild hypocaloric diet in abdominally obese patients with metabolic syndrome Secondary objectives: To assess the effect of rimonabant over a period of 12 months on: Liver fat content using CT scan (Computed Tomography scan) Anthropometric measures (weight, waist circumference, body composition using Dual Energy X-ray Absorptiometry (DEXA)) Lipid, lipoprotein profile Glycemia, insulinemia and HbA1c Adipokines, inflammatory and hemostatic markers To evaluate the percentage of patients with metabolic syndrome at 12 months To evaluate the safety and tolerability of rimonabant in these patients In four selected US sites the effect of rimonabant at 12 months will be also assessed on: Basal lipolysis and insulin suppressed lipolysis (euglycemic hyperinsulinemic clamp). Resting metabolic rate and substrate oxidation at rest using indirect calorimetry. Adipose tissue histology and expression of genes involved in glucose and lipid metabolism (superficial adipose tissue biopsy).

This phase I trial is studying the side effects and best dose of 7-hydroxystaurosporine when given together with perifosine in treating patients with relapsed or refractory acute leukemia, chronic myelogenous leukemia, or myelodysplastic syndromes. 7-Hydroxystaurosporine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as perifosine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving 7-hydroxystaurosporine together with perifosine may kill more cancer cells.

Summary of the proposed research: The intravenous application of prostacyclin (PGE1) or its stable analogue, iloprost, has been used to cause a decrease not only of the pulmonary but also of the systemic vascular tone. Aerosolized prostacyclin, on the other hand, can result in a selective pulmonary vasodilatation without affecting the systemic blood pressure as shown in preliminary studies/case reports. No large trials exist for this type of use of the drug so far. Furthermore, aerosolized PGI2 can improve gas exchange and pulmonary shunt in clinical settings of impaired ventilation/perfusion ratio as it occurs in adult respiratory distress syndrome (ARDS) due to the redistribution of pulmonary blood flow from non-ventilated to ventilated, aerosol accessible lung regions. Therefore, the investigators propose to carry out a prospective, double blinded, randomized trial to show that the nebulized iloprost decreases pulmonary hypertension selectively and improves oxygenation in ARDS.