Endocrinology

The study compares 2 medicines for children who do not have enough hormone to grow: somapacitan given once a week (a new medicine) and Norditropin® given once a day (the medicine doctors can already prescribe). Researchers will test to see how well somapacitan works. The study will also test if somapacitan is safe. Participants will either get somapacitan or Norditropin® - which treatment participants get, is decided by chance. Both participants and the study doctor will know which treatment participants get. The study will last for 4 years. Participants will attend 19 clinic visits and have 1 phone call with the study doctor.

The study is a 2-arm, multicenter, 1:1 randomized, placebo controlled clinical trial. All subjects will receive close monitoring for development of AGT or T1DM. Subjects will receive Abatacept or placebo and close monitoring for development of AGT or T1DM. To assess the safety, efficacy, and mode of action of Abatacept to prevent AGT and T1DM. The primary objective is to determine whether intervention with Abatacept will prevent or delay the development of AGT in at-risk autoantibody positive non-diabetic relatives of patients with T1DM. Secondary outcomes include: the effect of Abatacept on the incidence of T1DM; analyses of C-peptide and other measures from the OGTT; safety and tolerability; and mechanistic outcomes.

The study is a 2-arm, double blinded, multicenter, 2:1 randomized, placebo controlled clinical trial. Subjects will receive hydroxychloroquine or placebo and close monitoring for progression of T1D.

Cystic Fibrosis Related Diabetes has been identified by the CF community as one of the top ten priorities for CF research. In CF clinical decline due to dysglycemia begins early, prior to diagnosis of diabetes and increases mortality from pulmonary disease. There is presently no way to determine who, of those with dysglycemia, will experience clinical compromise. However, the CF Center in Milan has found that measurable age- and sex-dependent variables on oral glucose tolerance testing (OGTT) predict β-cell failure-the primary driver of decline in CF. the investigators propose a multi-center trial to develop nomograms of age and sex dependent reference values for OGTT-derived measures including glucose, insulin, c-peptide, and the resultant OGTT-derived estimates of β-cell function, β cell sensitivity to glucose, and oral glucose insulin sensitivity (OGIS) and to determine correlation of these with clinical status (FEV-1, BMI z score, number of pulmonary exacerbations over the past 12 months). In a subset of the cohort the investigators will perform additional studies to determine possible mechanisms driving abnormal β cell function, including the role of lean body mass (as measured by DXA), impact of incretin (GLP-1, GIP) and islet hormones (glucagon, pancreatic polypeptide) on β cell function and the relationship of reactive hypoglycemia and catecholamine responses to β cell function, as well as the relationship of β cell sensitivity to glucose as determined by our model to abnormalities in blood glucose found in a period of free living after the study (determined by continuous glucose monitoring measures (Peak glucose, time spent >200 mg/dl, standard deviation). the investigators will also develop a biobank of stored samples to allow expansion to the full cohort if warranted and to enable future studies of dysglycemia and diabetes in CF. the investigator's eventual goal is utilization of the nomograms to determine the minimum number of measures to accurately predict risk for clinical decline from dysglycemia in CF.

This randomized phase III trial studies how well combination chemotherapy (vincristine sulfate, dactinomycin, cyclophosphamide alternated with vincristine sulfate and irinotecan hydrochloride or vinorelbine) works compared to combination chemotherapy plus temsirolimus in treating patients with rhabdomyosarcoma (cancer that forms in the soft tissues, such as muscle), and has an intermediate chance of coming back after treatment (intermediate risk). Drugs used work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Combination chemotherapy and temsirolimus may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known whether chemotherapy plus temsirolimus is more effective than chemotherapy alone in treating patients with intermediate-risk rhabdomyosarcoma.