Active clinical trials for "Cystic Fibrosis"

People with CF have a high incidence of diabetes, called CFRD. CFRD is an important cause of worsened morbidity and mortality, thus understanding the pathophysiology underlying its development is imperative. Insulin deficiency has been well recognized as one cause of CFRD; however the clinical presentation and studies of pathogenesis indicate that the etiology is more complex. There is strong evidence that normal metabolism of carbohydrate, protein and fat is altered in CF. We believe that the inflammatory response to chronic underlying lung disease is responsible for insulin resistance and alters substrate metabolism, and that these changes, in addition to insulin deficiency cause CFRD. Our global hypothesis is that hyperglycemia is caused, in part, by high rates of gluconeogenesis resulting from excessive amino acid substrate availability caused by cytokine-mediated protein catabolism. We further hypothesize that inflammation alters normal fatty acid metabolism leading to lipogenesis, an energy wasteful pathway. We will recruit 24 adult CF subjects and 10 controls (similar in distribution in lean tissue mass, age and gender) and will categorize them according to glucose tolerance (OGTT), as well as insulin secretion and insulin sensitivity using the Tolbutamide-stimulated IVGTT and the Minimal Model. Clinical status will be characterized by measuring pulmonary function and modified NIH scores, in addition to measuring levels of circulating cytokines. Gluconeogenesis (GNG) will be quantified by measuring the incorporation 2H into the 2nd, 5th and 6th carbons of glucose. Amino acid turnover rates will be measured using stable isotopes of lactate and alanine and whole body protein turnover (WBPT) will be measured using [1-13C]leucine and [15N2]urea. Fat metabolism will be evaluated by measuring ketone body turnover using stable isotopes, and by quantifying lipogenesis using the isotopomer equilibration method. Key enzymes of fatty acid metabolism will also be measured. We will utilize indirect calorimetry to measure resting energy expenditure. Subjects will be recruited from the CF centers at the University of Texas- Southwestern and the South Central CF Consortium.

Exocrine pancreatic insufficiency (EPI) is a condition where the pancreas does not have enough pancreatic enzymes to break down food. Some symptoms of EPI are frequent gas/bloating, unexplained stomach pains, frequent diarrhea, and foul-smelling, greasy stools. The purpose of this study is to see how effective CREON is for treating symptoms of EPI due to causes other than cystic fibrosis (CF), chronic pancreatitis (CP), pancreatectomy (PY), or pancreatic cancer (PC). CREON (Pancrelipase) is an approved drug used to treat people who cannot digest food normally due to their pancreas not making enough enzymes. Adult participants with a diagnosis of EPI due to causes other than CF, CP, PY, or PC will be enrolled. Around 50 participants will be enrolled in approximately 20 sites in the United States. Participants will receive oral CREON capsules with each meal and snack beginning at Day 1 for 27 weeks. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the course of the study at a hospital or a clinic. Participants will need to be confined for 2 separate periods of 6 to 8 days each to measure Co-efficient of Fat Absorption (during screening and after enrollment). The effect of the treatment will be checked by medical assessments, blood and stool tests, checking for side effects and completing questionnaires.

This is a prospective, descriptive, observational research study designed to observe and document the clinical practice by domain experts, and how the knowledge of new findings that are published in the medical literature affect clinical decision making. The study will evaluate risk factors and co-variants, including genetic variants that are associated with disease progression such as pain, inflammation, organ dysfunction, disability and quality of life.

The Sponsor is developing KB407, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) to the airways of people with cystic fibrosis via nebulization. This study is designed to evaluate safety and tolerability of KB407 in people with cystic fibrosis. This study will enroll 4 participants into each of the first two cohorts and will enroll five subjects into the last cohort. Cohort 1 will receive a single dose of KB407 and be followed for 60 days. Subjects in Cohort 1 may rollover into Cohort 2 at the Day 28 Visit. A Data Safety Monitoring Board (DSMB) will meet to determine study progress from Cohort 2 into Cohort 3. In Cohort 2, subjects will be dosed bi-weekly at Day 0 and Day 14. In Cohort 3 subjects will be dosed weekly at Day 0, Day 7, Day 14 and Day 21. All subjects will be followed for a year after the last dose of KB407.

The investigators hypothesize that measurement of beta adrenergic induced sweat rate using an evaporimeter can accurately and reliably determine different levels of CFTR dysfunction within a spectrum of patients expressing various degrees of cystic fibrosis disease. The investigators overall goal is to determine whether the evaporimeter technique of measuring beta-adrenergic induced sweat rate is capable of accurately and reliably identifying different levels of CFTR dysfunction, as a prerequisite before advancing this technique as biomarker assay into clinical trials.

The goal of this study is to perform a pilot study to evaluate the utilization of oxygen as an inhaled contrast agent to image the airway spaces in normal and diseased human lungs to allow an effect size estimate to power future studies.

The hypothesis is that the intranasal use of Pulmozyme will decrease the severity of sinusitis in Cystic Fibrosis and lead to an improved quality of life.

Hypothesis:DCI 1020 capsules are safe and effective in treating exocrine pancreatic insufficiency in CF patients <= 2 years of age. The results of this study are intended to be submitted to the FDA as part of the NDA package for marketing approval of PANCRECARB (DCI 1020).

The current study examines the efficacy of two treatments to help children with cystic fibrosis (CF) meet their dietary calorie requirements of 120% to 150% of the recommended daily allowance of energy and the effect of these treatments on weight gain and maintenance. One treatment provides children with CF and their parents nutrition education about the best foods for meeting their dietary needs. The second treatment gives children with CF and their families similar nutritional information plus behavioral parenting methods for motivating children to eat the recommended foods. Children with CF and their families are seen weekly for 7 treatment sessions across 9 weeks for the active phase of treatment. Families are then followed for 2 years after treatment in order to better understand how long the treatments are effective and to determine the health benefits of better nutrition status and weight gain.

The study is a prospective, double blinded, placebo controlled, randomized study to evaluate the effects of daily oral estrogen supplements on bone health, sexual and reproductive health, quality of life and markers of inflammation and lung function when given to hypogonadal women with Cystic Fibrosis related Bone Disease (CFBD).