View clinical trials related to Cystic Fibrosis.
Filter by:This is a multi-center, randomized phase III study to evaluate the clinical effectiveness of AeroVanc in persistent methicillin-resistant Staphylococcus aureus (MRSA) infection in patients with cystic fibrosis (CF).
This is a 5-part study of FDL176. Part 1 is a double blind, placebo-controlled, dose escalation study in healthy male participants. Part 2 is a single dose, open-label study in healthy male participants. Part 3 is a single dose, double blind, placebo-controlled study in healthy female participants. Part 4 is a randomised, double-blind, placebo-controlled, dose-escalation study in healthy male and female participants.Part 5 is a single dose, open-label study in male and female participants with CF.
Due to emerging resistance, new antibiotic options are needed to treat CF acute pulmonary exacerbations caused by methicillin resistant Staphylococcus aureus (MRSA). There is established evidence that adult patients with cystic fibrosis (CF) may have altered antibiotic pharmacokinetics compared with non-CF patients. Telavancin is a lipoglycopeptide antibiotic that has activity against gram-positive bacteria including MRSA. This study will determine the pharmacokinetics and tolerability of telavancin in 18 adult CF patients admitted for a pulmonary exacerbation at 1 of 4 participating hospitals in the US.
Over 1,900 mutations in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) protein are implicated in causing Cystic Fibrosis (CF). Potential therapies that directly target defective CFTR are being evaluated in important clinical trials, but most target the most common CFTR mutation F508del. Many patients with rare CF mutations are not able to participate in those studies. The RARE study is specifically designed for people with CF caused by rare mutations. Eligible rare mutations are listed below: • CF patients who are heterozygous for pre-mature stop codons as noted below: i. one allele must be a F508del ii. the other allele must be a pre-mature stop codon mutation • CF Patients with other genotypes that require Study PI permission: i. CF patients with two mutations that are not eligible for Trikafta ii. CF patients homozygous or heterozygous (other allele must be F508del) for rare mutations of special interest (e.g., 711+3A->G, 2789+5G->A, 3272-26A->G, 3849+10kbC->T). Other rare mutations will be considered on a case by case basis This is a multi-site, specimen collection study. Investigators will collect blood, intestinal cells and nasal cells from each participant. Cells from these specimens will be used to test future CFTR modulators to see if they might work for people with study eligible rare mutations. Having cells to test in the lab is an important first step in identifying potential new therapies for people with these mutations.
Assess if the use of SYNRINSE can improve short-term subjective and objective outcome measures after one week in patients with active Chronic Rhinosinusitis (CRS) who have had prior sinus surgery.
Study VX16-661-114 (Study 114) is a Phase 3b, randomized, double-blind, placebo-controlled, parallel-group, multicenter study in subjects aged 12 years and older with CF who are homozygous for the F508del mutation on the cystic fibrosis transmembrane conductance regulator gene (CFTR) gene and who discontinued treatment with Orkambi due to respiratory symptoms considered related to treatment. This study is designed to evaluate the safety and efficacy of Tezacaftor/Ivacaftor (TEZ/IVA).
Introduction: Bronchiectasis is a chronic lung disease in which the underlying condition causes permanent damage to the conducting airways. Bronchiectasis is associated with considerable morbidity and poor quality of life. While cystic fibrosis (CF) is the most common cause of bronchiectasis in childhood, non-CF bronchiectasis is associated with a wide variety of disorders. CF bronchiectasis patients show reduced daily habitual physical activity and exercise capacity. Cardiopulmonary exercise test (CPET) is increasingly gaining importance in clinical medicine and considered the gold standard exercise test for assessing aerobic exercise capacity. The test objectively evaluates exercise physiological functions, may help assess morbidity and predict the outcome and mortality in different clinical circumstances and may serve as a basis for individualized exercise prescription within the limitation of the disease. Unlike CPET in CF, there is a paucity of data on exercise capacity using CPET in non- CF bronchiectasis patients, and on the implications of physical activity on non- CF bronchiectasis morbidity and mortality. Aim: To evaluate and compare exercise capacity in CF and non-CF bronchiectasis patients. Methods: This will be a cross-sectional retrospective/prospective study population. The retrospective study will include data analysis of patients that preformed CPET as part of their clinical evaluation. In the prospective study, patients that are scheduled to perform CPET as part of their clinical evaluation will sign (or legal guardian) informed consent prior to participation. Patients will be recruited from the exercise clinic at the Pediatric Pulmonary Institute at the Rappaport Children's Hospital. Inclusion criteria: 1. Children and adults (age >7 years, height >125cm), with CF and non CF bronchiectasis. 2. Completed a maximal CPET test according to accepted criteria; (maximal VO2 > 80% predicted, maximal heart rate > 80% predicted, acceptable RER (RER > 1.0 in children (under 18 years), RER > 1.05 for adults) or reaching a VO2 plateau..3. Evidence of bronchiectasis in computed tomography (CT). Exclusions criteria: preforming submaximal CPET, lack of data from the exercise test, exacerbation of patient's condition within three days before the exercise evaluation, relevant related chronic diseases that affecting test results.
Prospective, randomized, cross-over, multicenter, trial comparing the efficacy and tolerability of nasal irrigation with Respimer® mineral salts solution versus saline solution both administered with Respimer® Netiflow® medical device among patients aged ≥ 11 years and older with cystic fibrosis and suffering from chronic rhinosinusitis.
This trial will consist of two parts: Part 1 and Part 2. Part 1 will enroll adult healthy volunteers (HV) into four treatment groups. The first group will enroll HV into a single ascending dose (SAD) treatment group consisting of three cohorts. The second group will enroll HV into a multiple ascending dose (MAD) treatment group consisting of three cohorts. The third group will enroll HV into a food effect (FE) treatment group consisting of one cohort. The fourth group will enroll HV into a drug-drug interactions (DDI) treatment group consisting of one cohort. Approximately 76 subjects will be enrolled in Part 1. Part 2 Cohorts 1 through 3 will enroll adult subjects with cystic fibrosis (CF) currently on stable ivacaftor/lumacaftor background therapy for a minimum of three months. Part 2 Cohorts 4 and Cohort 5 will enroll adult subjects with CF not currently receiving cystic fibrosis conductance regulator (CFTR) modulator therapy within 30 days prior to Day 1. Part 2 Cohort 6 will enroll adult subjects with cystic fibrosis on stable tezacaftor/ivacaftor background therapy. Approximately 104 subjects will be enrolled in Part 2.
People with CF have elevated rates of anxiety and depression when compared to the general population. Anxiety and depression can have a negative impact on adherence and disease self-management, leading to worse CF health outcomes such as respiratory symptoms, functional capacity, and health-related quality of life (HRQOL). Project UPLIFT is a group mental health intervention that can be delivered by telephone or Web, though for this study the intervention will be web based only. Project UPLIFT was originally developed as a depression treatment and prevention program for people with epilepsy and was shown to be effective in reducing depression and increasing knowledge and skills. Recently, Project UPLIFT was revised to help people with CF manage their depression and anxiety and shown to be apparently successful in a pilot study that included adolescents and adults with CF. The goals of this project are to determine the effectiveness of Project UPLIFT in reducing anxiety and depression in adolescents and adults with CF, as well as increasing their quality of life and other physical health-related disease outcomes.