Active clinical trials for "Cystic Fibrosis"

Presently, effectiveness of treatments for CF lung disease is judged by improvement in lung function (FEV1). However, in CF patients, FEV1 can range from severely decreased to normal, and improvements may occur slowly. Thus, clinical trials require many patients over prolonged periods to evaluate medications. As the pace of drug development accelerates, it is no longer possible to test all of the promising candidate therapies using conventional study designs. A sensitive technique for assessing lung inflammation has been developed which uses the expression of genes located in circulating blood cells. Mononuclear cells pass repeatedly through the blood vessels of the lung, and are exposed to many of the inflammatory products that are present in the airways. Over the past 4 years the investigators have identified a small group of candidate genes that are unregulated or downregulated in response to antibiotic treatment. The investigators now propose to prospectively test this method of quantifying lung inflammation in a large group of CF patients undergoing treatment of pulmonary exacerbations. Blood will be sampled before and after antibiotic treatment for a pulmonary exacerbation, and the relative change in gene expression will be compared to improvement in FEV1 and other clinical responses, to determine the utility of this method for use in studies. If successful, this technique could allow for a rapid and noninvasive method to gauge immediate effects by new treatments, and assist caregivers in determining optimal treatment strategies for the individual.

The purpose of this study is to provide the necessary data and experience to design a larger, full scale clinical trial to determine if a certain medicine (repaglinide), which increases the amount of insulin secreted by the pancreas, can improve the nutritional status and pulmonary function of adolescents and young adults with cystic fibrosis and prediabetes by improving blood glucose control. The investigators are also trying to determine the relationship between systemic inflammatory factors and glucose impairment.

Clinical and translational research in cystic fibrosis (CF) is hampered by a lack of biomarkers that can be used to identify promising new therapies. There is an urgent need for development and validation of biomarkers that more quickly predict the usefulness of potential drugs in CF and might prognosticate clinical course. In particular, combinations of protein biomarkers that can be obtained non-invasively offer great promise. The goal of this project is to determine whether protein biomarkers in blood can demonstrate a beneficial effect of treatment over two weeks. We intend to initially target an acute pulmonary exacerbation in CF because we know that subjects being treated with intravenous antibiotics and enhanced mucus clearance display clinical improvements within two weeks. We propose to prospectively collect blood samples from a large cohort of well-characterized CF persons serially during inpatient admissions for a pulmonary exacerbation and longitudinally during annual visits. Through this proposal, we hope to identify a CF lung injury biomarker panel that increases in the setting of an acute pulmonary exacerbation and improves rapidly following intravenous antibiotic therapy. Additionally, we will begin to explore whether this CF lung injury biomarker panel might also prognosticate clinical course including decline in pulmonary function. Finally, this study will serve as an important source of blood samples that will be banked for future biomarker and therapeutic studies designed to benefit the entire CF community.

Spirometry is a useful clinical tool for the assessment and monitoring of lung disease, however, it does not provide information on peripheral airways resistance. On the contrary, impulse oscillometry (IOS) may provide information not only on airway resistance (Rrs) but also on the elastic properties of the lung (Xrs). In addition, multiple breath nitrogen washout (MBNW) utilizes the exhalation of nitrogen gas from the airways to determine changes in lung ventilation and derive small airways indices (that tells us about small airways calibre). This method, like IOS, allows a precise assessment of small airways function. Even though patients with asthma may show some reduction of the caliber of the small airways these changes are more a feature of patients with COPD. The study team hypothesize that IOS and MBNW measurements may detect these differences and provide different resistance profiles for asthma and COPD. Furthermore, the study team would like to investigate the relationship between airway inflammation and small airway disease by measuring exhaled nitric oxide (NO) at multiple exhalation flow rates. This technique allows the partitioning of NO produced in the central airways from that generated more peripherally in the lung, providing valuable information on the activity of inflammation in different parts of the respiratory system. The study team hope that the combined use of IOS, MBNW and NO will identify a possible correlation between inflammation and small airway dysfunction.

The purpose of this study is to better define risk factors preceding first isolation of Pseudomonas aeruginosa (Pa) from respiratory cultures in cystic fibrosis (CF) lung disease and to better define clinical outcomes associated with acquisition of Pa. This study will also collect and bank DNA samples for current and future studies designed to enhance the understanding of the pathogenesis of CF.

The purpose of this study is to evaluate the impact of an internet based program for children and families (CF.DOC) with Cystic Fibrosis on health outcomes. The program provides for virtual visits, a personal health record, messaging with clinicians and several tools for monitoring self-care behaviors. We anticipate that this intervention will provide for more intensive monitoring and feedback of self-care behaviors and will lead to improved health status and in particular nutritional status

Healthy volunteers and patients with diseases that involve problems clearing mucus from the lungs will be examined and tested to better understand the reasons for recurring lung infections in these patients and to try to develop better ways to diagnose and treat them. The study will also try to identify the genes responsible for these diseases. Healthy volunteers 18 years of age and older and patients 2 years of age or older with suspected primary ciliary dyskinesia (PCD), variant cystic fibrosis (CF) or pseudohypoaldosteronism (PHA) may be eligible for this study. Patients enrolled in the Natural History Study of Nontuberculous Mycobacteria at NIH or other NIH natural history protocols may also be enrolled. Participants undergo the following tests and procedures during a 1-day visit at the NIH Clinical Center, as follows: All patients and normal volunteers have the following procedures: Physical examination and review of medical and genetic history and family genetic history. Lung function test and measurement of oxygen saturation level. Nitric oxide measurement to measure the amount of nitric oxide production in the nose: A small tube is placed in the nose while the subject breathes through the mouth into a cardboard tube. All patients have the following additional procedures: Blood tests for liver and kidney function, blood count, immunoglobulins and pregnancy test (where appropriate). Blood test or buccal scrape (brushing the inside of the cheek) to obtain DNA to look for gene mutations that cause PCD, CF or PHA. Scrape biopsy of cell lining the inside of the nose: A small toothpick-sized plastic stick with a tiny cup on the end is used to get nasal lining cells to look at the cilia (hair-like structures that move mucus). Semen analysis (in some men) to test sperm tail function or structure. Patients suspected of having a variant of CF or PHA, including nontuberculous mycobacterial lung disease, have the following additional procedures: Sweat chloride test: A medicine is placed on the arm to produce sweat; then, a very low level of electric current is applied for 5 to 12 minutes. Sweat is collected in a plastic tube and tested for salt content. Blood draw for CF genetic testing, if necessary, and to measure levels of the enzyme trypsin. Saliva collection to measure sodium and chloride content. Nasal potential difference to measure the electrical activity of the cells lining the inside of the nose: A soft plastic tube filled with a salt solution is passed into the nasal passage and a sterile needle is placed under the skin of the arm. This test provides information about how the lining of the nose is able to get used to changes in temperature and humidity. (Normal volunteers also have this test.)

The purpose of this study to to assess the feasibility, acceptability, and satisfaction of a telehealth cognitive behavioral stress management (CBSM) intervention among adults with cystic fibrosis (CF) who exhibit elevated anxiety and/or depression symptoms.

This is an observational cohort study building on an existing protocol at our institution recruiting CF patients who are 6-21 years old with one or two copies of the F508del CFTR mutation. That existing protocol seeks to enroll a total of 38 subjects; it includes three to four (three scheduled, with a fourth optional) study visits, with spirometry, LCI, UTE MRI, and 129Xe MRI being performed at each visit. This present study will utilize existing study visits for the NHLBI study, with a sub-set of 20 subjects opting-in to have all of the procedures performed twice, with an intervening ACT, during one of their study visits.

STeP IT UP CF: STimulating ImProved Health And Well-being In CysTic Fibrosis Using Integration Of Fitness Technology and Port CF. A pilot in integration of wearable fitness tracker data with existing health data provided by CF foundation Patient Registry (Port CF)