View clinical trials related to Cystic Fibrosis.
Filter by:This study will assess the relative bioavailability of ganciclovir from the pro-drug valganciclovir in lung transplant recipients with or without cystic fibrosis. Each patient will receive 900mg valganciclovir daily for the period specified at their center, starting as soon as possible after the transplant. Pharmacokinetic assessments will be made provided that steady-state kinetics of ganciclovir and immunosuppressive drugs have been obtained (>=4 days of drug therapy). Blood samples for pharmacokinetic analysis will be taken up to 24h post-dose on one occasion. The anticipated time on study treatment is 3-12 months, and the target sample size is <100 individuals.
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.)
Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the CF trans-membrane conductance regulator (CFTR) protein. CF is the most common inherited disease of Caucasians, with a carrier frequency of 1 in 25-30 individuals. Even with the impressive advances achieved in the understanding of the molecular basis and physiopathology of CF, it remains a life-threatening disorder that causes severe lung damage and nutritional deficiencies. It is generally accepted that early therapy could delay the progression of lung disease. A number of non-invasive methods are available to monitor disease activity in CF patients; however none of the currently used tools are able to monitor real-time events. Recently high resolution computed tomography (HRCT) has been used to monitor changes in lung structure. However, HRCT does not allow differentiating between acute and chronic lesions. Positron emission tomography (PET) with 18fluoro-deoxy-glucose (FDG) has already been used in a variety of settings to visualize inflammation or infection. FDG-PET imaging appears to be a promising new tool to quantify inflammation as it can detect clinically relevant changes even when no changes or minimal ones are detected by morphologic imaging. PET/CT may consequently be used to evaluate the severity of lung inflammation/infection in CF patients, and therefore the aim of this study is to evaluate the use of PET/CT for the assessment of the severity of lung inflammation/ infection in CF patients.
This is a research study where researchers are collecting blood to evaluate the genetic characteristics of individuals with chronic lung diseases, including asthma, chronic obstructive pulmonary disease (COPD), interstitial lung disease, cystic fibrosis, and lung cancer.
People with cystic fibrosis (CF) often develop chronic pulmonary infections which are caused by a variety of organisms, the most predominant being Pseudomonas aeruginosa. Antibiotics are important in managing CF lung infections. Antibiotic use in CF was altered about ten years ago with the approval of inhaled tobramycin, an aminoglycoside which is effective in treating P. aeruginosa. A decade later, CF clinicians are increasingly concerned about the likelihood of induction of aminoglycoside-resistance with prolonged use of inhaled tobramycin to treat chronic P. aeruginosa airway infections. The goal of this study is to examine the current microbiology and susceptibility of organisms from CF sputum, correlate it with antibiotic use, and compare it with previous data.
The purpose of this trial is to evaluate the safety and effectiveness of a one dose strength of denufosol compared to placebo in patients with mild CF lung disease.
In some patients with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein. PTC124 has been shown to partially restore CFTR production in animals with CF due to a nonsense mutation. In an ongoing Phase 2a study being performed at the Hadassah University Hospital - Mount Scopus in Israel, patients with nonsense-mutation-mediated CF have received PTC124 in two 14-day treatment courses. Preliminary results from that study indicate that PTC124 has pharmacodynamic activity of PTC124 in CF and that PTC124 can be safely administered in this patient population. This Phase 2b extension study is designed to evaluate the activity and safety of 3 months (approximately 84 days) of continuous treatment with PTC124 in the same patients who were enrolled to the Phase 2a study. The main purpose of this study is to understand whether PTC124 can be safely administered and achieve pharmacodynamic activity in patients with CF due to a nonsense mutation.
In 2006, the International Patient Decision Aids Collaboration (IPDAS) reached agreement on criteria for evaluation. Accordingly, the primary outcome is 'decision quality' 3 weeks after using the decision aid. Decision quality is defined as the extent to which a patient decision aid improves the match between the chosen option and the features that matter most to the informed patient. Measures include: a) patients' knowledge of essential facts [options, benefits, harms, and outcome probabilities]; and b. the congruence between the option chosen and patients' informed values regarding benefits and risks. In the past twenty years lung transplantation has become the most widely accepted option of treating cystic fibrosis patients with severe lung disease. Lung transplantation can be a good experience for many patients, improving their quality of life and their survival. However there are potential risks of lung transplantation including infection, organ rejection and early death. Cystic fibrosis patients are often faced with making a choice of whether to be referred for lung transplantation when they are very sick and there is the immediate need to survive. Our group has developed a tool called a decision aid which we hope will assist the patient and family in making this choice. The decision aid guides the patient through a series of steps where they weigh the benefits and risks of being referred for lung transplantation and the benefits and risks of receiving 'usual care' without the option of referral.
Cystic Fibrosis patients continue to have bowel problems even after adequate pancreatic enzyme supplementation. There may be pathology of the lining of the bowel. Capsule endoscopy will be used to photograph the entire bowel.
Mucociliary clearance, in which mucus secretions are cleared from the breathing airways, is the primary defense mechanism for the lungs. Inhaled particles, including microbes that can cause infections, are normally entrapped in mucus on the airway surfaces and then cleared out by the coordinated action of tiny hair-like structures called cilia. Individuals with primary ciliary dyskinesia, variant cystic fibrosis, and pseudohypoaldosteronism have defective mucociliary clearance. The purpose of this study is to collect clinical and genetic information about these three airway diseases to improve current diagnostic procedures.