View clinical trials related to Cystic Fibrosis.
Filter by:The purpose of this study is to describe the effects of massage therapy on quality of life (QOL) in youth and young adults (ages 8 to 21 years) with cystic fibrosis (CF).
This pilot study will be conducted in adolescents with cystic fibrosis (CF) without diabetes but with abnormal glucose tolerance, and will assess the effects of sitagliptin on glucose regulation. An oral glucose tolerance test (OGTT) and a mixed meal tolerance test (MMTT), will be performed at baseline and again ~4 weeks after treatment with study drug. We will also look at blood sugars throughout the day using a continuous glucose monitor (CGM) before each time the MMTT/OGTT are performed. Several hormones that may affect the way the body regulates blood sugars will be measured in blood when the OGTT and MMTT are done. We will assess the effect this medicine has on blood sugars (using CGM) and the effect the medicine has on the hormones measured during the OGTT and MMTT.
This phase IV, multicenter, randomized, open-label, two-period crossover study will evaluate the comparable efficacy and safety of Pulmozyme (dornase alfa) delivered by the eRapid nebulizer system in patients with cystic fibrosis. Patients who have been receiving Pulmozyme once daily chronically for at least 6 months will continue to receive Pulmozyme once daily for a run-in period of 2 weeks using the Pari LC Plus nebulizer. Patients will then be randomized to receive in a crossover design Pulmozyme once daily for two treatment periods of 2 weeks each using either the Pari LC Plus or the eRapid nebulizer. Anticipated time on study treatment is 6 weeks.
The purpose of this Randomized, Double-blind, Multicenter, Two-period Crossover Study is to Assess the Efficacy and Tolerability of Burlulipase (NM-BL) in Patients with Exocrine Pancreatic Insufficiency due to Cystic Fibrosis
An open label study in 40-60 subjects with diagnosed lung airway disease and in 10-20 normal controls. Each subject will receive PFP as a contrast agent to visualize the airway and alveolar spaces in their lungs using magnetic resonance imaging of inert gas/oxygen mixtures.
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.
To study the effects of an 8-week combined inspiratory muscle training and exercise (resistance+aerobic) program on of a lung volume, inspiratory muscle strength (maximal inspiratory pressure, PImax) and cardiorespiratory fitness (maximal oxygen uptake, VO2peak) (primary outcomes)and dynamic muscle strength, body composition and quality of life (QoL) in children with Cystic Fibrosis (CF) (secondary outcomes).
The purpose of this study is to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD), of ivacaftor in children with cystic fibrosis (CF) who are 2 through 5 years of age and have a CF Transmembrane Conductance Regulator (CFTR) gating mutation in at least 1 allele. Part A is designed to evaluate the safety and PK of multiple-dose administration of ivacaftor in participants 2 through 5 years of age and to confirm the doses for Part B. Part B is designed to evaluate the safety, PK, PD, and efficacy of ivacaftor in participants 2 through 5 years of age.
The purpose of this study is to confirm and extend the Phase 1-2 KB001 findings of an airway anti-inflammatory effect in CF individuals with chronic Pseudomonas aeruginosa (Pa) airway infection. It is hypothesized that steady-state levels of KB001-A in CF subjects with airway Pa infection will be safe and well-tolerated, and will increase the time-to-need for antibiotic treatment (IV, inhaled, or oral) for worsening of respiratory tract signs and symptoms compared with placebo.
Bronchopulmonary infection is the most common and serious complication in the evolutionary course of cystic fibrosis (CF). Administration of antibiotics adapted to infecting pathogens is one of the key issues for its management. However, more than half of patients with CF have chronic respiratory infections for which infectious agent remains unknown leading to empirical antibiotic therapies that are not adapted to the causative agents. Recently, new technologies have been applied for the description and characterization of microbial agents in CF patients including molecular biology techniques that allowed us to detect and to identify new and/or emerging pathogens. Moreover, more sophisticated molecular techniques such as pyrosequencing and PCR amplification and cloning lead us to demonstrate the huge microbial diversity associated with chronic bronchopulmonary infections in this population. Otherwise, a metagenomic approach revealed the extraordinary complexity of the respiratory flora in these patients and, somewhat unexpected, abundance of anaerobes, viruses, and bacteriophages. In addition, it has been shown that some antibiotics commonly used in clinical practice for the treatment of respiratory infections were able to induce these bacteriophages, suggesting the existence of lateral gene transfer by transduction. The human microbiome is the set of microbial communities associated with the human body and represents all living microorganisms in the body. Its role in the immunity development has recently been demonstrated suggesting that changes in this ecosystem play a critical role in evolution of several human diseases. For example, in obesity it has been shown that there is a relationship between the intestinal human microbiota and nutritional and metabolic status of the hosts and specific alterations of these intestinal microbiota may represent a metagenomic signature of this disease. Evolution of the respiratory microbiota in patients with cystic fibrosis, whose nutritional status is often impaired (chronic malnutrition due to disorder of digestive absorption) and receiving regular antibiotic treatments remains unknown to date. Characterization of this ecosystem and its role is a critical step to understand the evolutionary course of the disease. The main objective of this seminal study is to describe and to characterize the respiratory microbiota from sputum samples obtained from a limited number of selected patients with CF from 5 regional care centers (CRCM) from South of France (Mucomed network) (2 patients per center : 6 adults and 4 children), with similar clinical, microbiological and functional status before and after a cure of antibiotics. Different microbiological tools will be used including axenic culture systems, co-culture on amoebae in order to to isolate and to identify the microbial communities. Identification of bacteria will be done using MALDI-TOF mass spectrometry and/or molecular techniques. Moreover, 16S rRNA PCR amplification followed by cloning and sequencing of PCR products from the same sputum sample will be carried out to identify and to compare the bacterial species identified using molecular methods. In a second step, once the respiratory microbiota was characterized, it will be interesting to develop a dedicated microarray that will allow to detect all the bacteria identified in the first stage of the project and to assess its relevance on a larger cohort of patients with CF by studying the correlation between the respiratory microbiota and clinical status of patients according to the prescribed antibiotic treatments. This study will open new clinical perspectives and will help us to determine the potential role of antibiotics on the microbiota evolution during treatment according to regional health care practices. This will contribute to better understand the role of the microbiota in the evolution of these chronic respiratory infections. It could be the first step for innovative therapeutic strategies, taking into account the balance of complex microbial flora and possibly evolving according to antibiotic therapies. It could also form part of a larger preventive strategy against transmission of specific pathogens in CF.