Active clinical trials for "Syndrome"

This study is a controlled trial of metformin in individuals with fragile X syndrome between the ages of 6 and 25 years. Participants will be randomized in a double-blind design to either drug or placebo and will attend three visits to the study site in a 4-month period for a series of tests. The primary objectives are to assess safety, tolerability, and efficacy of metformin in the treatment of language deficits, behavior problems, and obesity/excessive appetite in individuals with fragile X syndrome.

The aim of this study is to elucidate whether night-time dosing of mirebegron can improve nocturia, compared with daytime dosing of mirabegron.

This phase I trial studies the safety of transplantation with a haploidentical donor peripheral blood stem cell graft depleted of TCRαβ+ cells and CD19+ cells in conjunction with the immunomodulating drug, Zoledronate, given in the post-transplant period to treat pediatric patients with relapsed or refractory hematologic malignancies or high risk solid tumors.

This research study is assessing a new drug, duvelisib, in combination with a drug that is already FDA approved, venetoclax, as a possible treatment for participants with CLL or those with Richter's Syndrome

Children with inadequate intestinal absorption due to loss of large amounts of small bowel require intravenous nutrition (feeding through the vein) to sustain hydration and nutrition to avoid starvation and dehydration; however, intravenous (IV) nutrition can lead to complications including liver failure. Tube feeding directly to the small intestine avoids the complications of IV nutrition, but fats are not fully digestible due to inadequate bowel function. We propose to predigest the fat using a small cartridge attached to the feeding tube to allow for rapid absorption with the possibility of reducing or eliminating the need for intravenous nutrition

Background: Bronchiolitis obliterans syndrome (BOS) is a complication people can experience after hematopoietic stem cell transplant. It usually affects people with chronic graft versus host disease (cGVHD). This occurs when donor stem cells attack the cells of the person who received them. BOS reduces airflow and oxygen levels in the body. It may be caused by neutrophil elastase in the body. Researchers believe the new drug alvelestat (MPH966) may help. Objectives: To test the safety of alvelestat (MPH966) and see what dose best inhibits neutrophil elastase in people with BOS after a stem cell transplant. To study how well the best dose improves lung function in those people. Eligibility: Adults 18 and older who have had a hematopoietic stem cell transplant and have cGVHD and BOS. Design: Participants will be screened with a medical history, physical exam, and blood and urine tests. They will have lung function and heart function tests. They will have computed tomography scans of the chest. Study part 1: Participants will take the starting dose of the study drug by mouth twice a day for 14 days. This is 1 cycle. They will get different doses, for up to 4 cycles. Study part 2: Participants will take the study drug twice a day by mouth at the dose set in part 1, for up to 12 months. Participants will keep medicine diaries. Participants will have several study visits. These may include: Repeats of the screening tests. Bronchoscopy with bronchoalveolar lavage. Sputum samples taken. 6-minute walking test. cGVHD assessment and answer questions. Participants will be contacted after the study for up to 24 months.

The purpose of this study is to evaluate the efficacy as well as safety of rifaximin combined fecal microbiota transplantation(Gut microbiota reconstruction) in the treatment of IBS-D.

The hypothesis is that acupuncture is equally effective as metformin (both treatments combined with lifestyle management) in improving whole body glucose homeostasis in insulin resistant women with polycystic ovary syndrome (PCOS), and that both are superior to lifestyle management alone. The investigators hypothesize that acupuncture and metformin induce ovulation and improve hyperandrogenism, as well as health related quality of life (HRQoL) and symptoms of anxiety and depression. Although equally effective (acupuncture and metformin), the investigators hypothesize that acupuncture is associated with less negative side-effects. The investigators also hypothesize that these treatments have the potential to restore epigenetic and molecular alterations in target tissues (endometrial-, adipose-, and skeletal muscle tissue) and thus have the potential to prevent the development of type 2 diabetes (T2D).

The purpose of study is to evaluate the efficacy of the electrical muscle stimulation (EMS) device in patients with patellofemoral pain known as anterior knee pain. Usual treatment for it is physical therapy (PT). We want to evaluate and see if adding the electrical muscle stimulation will fasten the recovery and improved outcome in patellofemoral pain syndrome. Half of participants will receive EMS and PT while other half will receive PT only.

This is a minimal risk case-controlled single arm intervention study, including 10 patients with a prior history of takotsubo and 10-age and sex matched healthy controls. Subjects will undergo in laboratory testing to measure autonomic function. They will then undergo a 15-week program of device-guided breathing with remote measures of autonomic function obtained at home. Analysis will determine the reproducibility of home autonomic measures and the provide preliminary data to determine the efficacy of device-guided breathing on autonomic measures and quality of life in patients with takotsubo.