Active clinical trials for "Syndrome"

Allogeneic blood stem cell transplant remains the only potential curative treatment for myelodysplastic syndromes (MDS) to date. Pre-transplant induction chemotherapy with leukemia-type regimens is associated with significant toxicity and even death. The hypomethylating agents decitabine and 5-azacytidine have been shown in studies to cause improved hematologic parameters and partial or complete responses in patients with high risk MDS compared to standard therapy. In contrast to leukemia-type chemotherapy, decitabine is associated with a relatively low risk of toxicity. We therefore propose to treat transplant-eligible MDS patients with Decitabine as induction therapy and a bridge to transplant. Hypothesis: Decitabine is able to reduce disease burden as measured by blood and marrow blast counts prior to allogeneic hematopoietic stem cell transplant to below 5%. Decitabine is well-tolerated by patients with high-risk MDS and will be a safe induction agent and bridge prior to allogeneic transplant in transplant-eligible patients.

Background: Sjögren's syndrome is an autoimmune disease (where the immune system attacks normal body tissues) that affects the salivary glands. Many people with Sjögren's syndrome are not able to make enough saliva because their salivary glands are inflamed. The dry mouth that results can interfere with daily activities and can lead to dental cavities, mouth sores, and infections. Injections of corticosteroids into the parotid glands can improve saliva production in people with Sjögren's syndrome, but current treatment practices may provide only temporary relief. Researchers are interested in studying the effectiveness of stronger corticosteroid injections (using dexamethasone) to determine how the corticosteroid treatment actually works. Objectives: - To evaluate the effectiveness and mechanics of dexamethasone injections to improve saliva production in individuals with primary Sjögren's syndrome. Eligibility: - Women between 18 and greater of age who have been diagnosed with primary Sjögren's syndrome, and have had a biopsy of the minor salivary glands in the past 5 years that shows a moderate level of inflammation. Design: Participants will be screened with a full medical history and physical examination, blood and urine tests, and salivary gland biopsies. Participants will also be screened with tests of saliva flow production and evaluation of the salivary ducts and glands, and will complete questionnaires about dry mouth symptoms. At the first treatment visit, participants will receive an injection of dexamethasone into one parotid gland and an injection of saline into the other gland. After the injections, participants will provide a blood sample to test the level of dexamethasone in the blood. Two weeks after the first treatment, participants will return for an evaluation visit to have saliva flow rate measurements taken, and will complete a questionnaire about dry mouth symptoms. Four weeks after the first treatment, participants will have a second treatment for each parotid gland, with the same tests and questionnaires as before. Participants will have additional evaluation visits 6 and 8 weeks after the first treatment visit, with a followup telephone call approximately 6 weeks after the last dexamethasone treatment visit.

Term and near term newborns can present acute respiratory distress syndrome (RDS). Surfactant treatment has been shown effective in reducing mechanical ventilation and oxygen treatment durations in the preterm newborn. Whether surfactant treatment is beneficial for term and near term newborns is unknown. The purpose of this study is to compare surfactant treatment vs. nasal continuous positive airways pressure in the newborn between 35 and 41 weeks of gestation with RDS within the first 24 hours of life. The study's primary endpoint is "survival with no oxygen treatment at 72 hours of life". The secondary endpoints are: death, surfactant treatment, pneumothorax, secondary infections, pulmonary hypertension, inhaled nitric oxide treatment, fluid loading treatment, vasopressive amines treatment, mechanical ventilation duration, nCPAP treatment duration, Oxygen treatment duration, Oxygen treatment at 28 days of life, hospitalization duration and treatment strategy cost.

Phase 1, single-center, open-label, sequential cohort dose escalation study. This is a 3 + 3 design study involving at least 3 subjects in ascending dose cohorts, with subjects participating up to 10 weeks. The overall study objectives are to evaluate the safety and tolerability of TXA127 in thrombocytopenic subjects with low or intermediate-1 risk MDS. Evaluation of the platelet response and the erythroid and granulocytic responses to TXA127 will provide preliminary efficacy data.

Progestin-induced endometrial shedding (PIES) followed by clomiphene citrate is fertility treatment of choice in anovulatory women with polycystic ovary syndrome (PCOS). However, some preliminary data suggest that skipping PIES could result in a higher live birth rate. The investigators are performing the first randomized controlled trial to find out if skipping the use of progestin during fertility treatment of anovulatory PCOS women is associated with improved pregnancy and live birth rates compared to the traditional approach of using progestin prior to use of clomiphene citrate.

The study will determine the benefit of Colchicine versus placebo for cardiac surgery patients on the post-operative development of atrial fibrillation and post-pericardiotomy syndrome. Primary Objective. Colchicine will reduce the composite endpoint of incidence of post-operative atrial fibrillation and post-pericardiotomy syndrome at 3 months following cardiac surgery. Secondary Objectives. Colchicine will reduce the incidence of constrictive physiology on echocardiography at 3 months following cardiac surgery. Reduction in the burden of symptomatic and asymptomatic atrial fibrillation in the 3 months following cardiac surgery with the use of colchicine.

This is a first in human, non-randomized, open-label, dose escalation study to investigate the safety, pharmacokinetics, immunogenicity and pharmacodynamics of repeat doses of KHK2823.

To investigate the long-term safety and tolerability of clobazam when administered for 1 year as adjunctive therapy in paediatric patients aged ≥1 to ≤16 years with Dravet Syndrome.

In this study, newborn babies with respiratory distress syndrome (RDS), receiving oxygen via nasal continuous airway pressure (CPAP) modalities, and needing surfactant treatment will be randomized to standard delivery of surfactant via and endotracheal tube airway (inserted after pre-medication for pain with a short-acting narcotic), or to surfactant delivery via laryngeal mask airway (LMA). The intent is to remove the airways and return babies to non-invasive CPAP support, after surfactant is given. The primary outcome measure is the rate of failure of initial surfactant therapy. Standardized failure criteria are reached: a) early, if the baby is unable to be placed back on non-invasive CPAP (i.e., needs tracheal intubation and mechanical ventilation) or, b) late, if the baby requires ventilation, retreatment with surfactant within 8 hours or more than 2 doses of surfactant. The objective of this protocol is to reduce the need for endotracheal intubation and mechanical ventilation in preterm neonates with RDS needing rescue surfactant therapy by instilling surfactant though an LMA, while achieving comparable efficacy of surfactant treatment. The hypothesis is that surfactant treatment through an LMA will decrease the proportion of babies with RDS who require mechanical ventilation or subsequent intubation, when compared to standard surfactant treatment following endotracheal intubation with sedation.

The purpose of this study is to determine if Botulinum Toxin-A (Botox) injection will improve symptoms of constipation in obstructed defecation syndrome (ODS).