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We list hundreds of Clinical Trials about "Cystic fibrosis drug available England after pricing deal" on BioPortfolio. We draw our references from global clinical trials data listed on ClinicalTrials.gov and refresh our database daily.
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Cystic fibrosis is a genetic disease caused by mutation of the cystic fibrosis transmembrane conductance regulator (CFTR). The purpose of the study is to investigate the effects of miglustat on CFTR function in cystic fibrosis patients.
The long-term goal of this project is to examine the effects of cystic fibrosis (CF) and cystic fibrosis related diabetes (CFRD) on brain structure and function
In order to further enable physiopathology studies, the aim of this project is to validate an in vitro model of cystic fibrosis created using induced pluripotent stemcell (iPS) differentiated bronchial epithelium from cystic fibrosis (CF) patients homozygous for the p.Phe508del mutation of the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
A Study to Compare US Marketed Pancrelipase Drug Product With Drug Product Manufactured With a Modernized Process at an Alternate Manufacturing Site, in Participants With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis.
This is a study to demonstrate that pancrelipase Delayed Release (DR) capsules manufactured with a Modernized Process (MP) is non-inferior to currently marketed pancrelipase DR capsules in participants with EPI due to Cystic Fibrosis (CF), as measured by Coefficient of Fat Absorption (CFA). Safety will also be evaluated.
The effect of comprehensive respiratory physiotherapy applications on respiratory function, functional capacity and peripheral muscle strength in children with cystic fibrosis and non-cystic fibrosis will be compared.
Exercise is an important clinical feature in cystic fibrosis. Better exercise capacity has been associated with better patient outcomes and quality of life. Exercise-induced bronchospasm is a condition, often associated with asthma, which may make exercise difficult. The role that exercise-induced bronchospasm has in people with cystic fibrosis is unknown. This study is designed to determine how often exercise-induced bronchospam occurs in cystic fibrosis.
OBJECTIVES: Evaluate the efficacy and safety of lipid-mediated transfer of the cystic fibrosis transmembrane conductance regulator gene to nasal epithelium in patients with cystic fibrosis.
OBJECTIVES: I. Determine the stability of uridine triphosphate (UTP) and examine the metabolism of exogenous nucleotides on airway epithelial surfaces in patients with cystic fibrosis. II. Determine the acute safety and efficacy of aerosolized UTP in children with cystic fibrosis.
For some years, the investigators observe an increase of the arisen of diabetes in cystic fibrosis patients However, this diabetes may be reversible. The investigators speak about " Cystic fibrosis related diabetes.". The objective of this project, is to know better what facilitates the appearance and the reversibility of the diabetes, such as the genetic mutations, the respiratory state and the lung infections. Theses knowledges should allow to adapt the screening of diabetes,...
This study is a multicenter, prospective cohort study of patients diagnosed with cystic fibrosis, the clinical information of recruited patients, including clinical manifestations, lung function, chest imaging, quality of life and other indicators, will be followed for 10 years.
Children with cystic fibrosis require increased caloric intake to maintain appropriate growth, an important determinant of long-term outcomes. This study seeks to determine the feasibility of using a novel therapeutic food to promote weight gain and growth in children with cystic fibrosis.
The purpose of this study is to confirm the improvement in pulmonary function and cytokine levels observed in the recently completed multidose aerosol study for the treatment of Cystic Fibrosis (CF).
Background Chronic airway inflammation is present in cystic fibrosis. Non-invasive inflammometry may be useful in disease management. Objective We studied 1) the ability of fractional exhaled nitric oxide and inflammatory markers (acidity, nitrite, nitrate, hydrogen peroxide, 8-isoprostane, interferon-γ, tumor necrosis factor-α, interleukin-2,-4,-5,-10) in exhaled breath condensate, to discriminate between cystic fibrosis and control children, and, 2) the relationship of bio...
The purpose of this research is to determine how a person's lungs will uptake [18F]fluorodeoxyglucose (FDG), as measured with positron emission tomography (PET) scanning in young cystic fibrosis patients.
The purpose of this study is to examine the role of a well-known and well-tolerated antibiotic, doxycycline, in the treatment of cystic fibrosis patients who are hospitalized. This antibiotic does not effectively treat the bacteria in airways of cystic fibrosis patients, but may reduce the activity of inflammatory molecules in the disease.
The purpose of this study is to compare the effectiveness of different mucous clearance techniques in cystic fibrosis patients
The aim of the study is to assess the diagnostic sensitivity of MRI to detect changes in Helbich-Bhalla scoring over time in patients with cystic fibrosis
This study will evaluate the pharmacokinetics (PK), safety, tolerability, efficacy, and pharmacodynamic effect of VX-659, tezacaftor (TEZ), and ivacaftor (IVA) when dosed in triple combination (TC) in Cystic Fibrosis (CF) subjects with F/F or F/MF genotypes.
This study will evaluate the pharmacokinetics (PK), safety, tolerability, efficacy, and pharmacodynamic effect of VX-445, tezacaftor (TEZ), and ivacaftor (IVA) when dosed in triple combination (TC) in Cystic Fibrosis (CF) subjects 6 through 11 years of age with F/F and F/MF genotypes.
This research is being done to find the genes and other factors that are responsible for differences among persons with cystic fibrosis. We are particularly interested in the factors that relate to the development of Cystic Fibrosis Related Diabetes (CFRD).
The objective of this study is to evaluate the relevance of Porphyromonas as a biomarker predicting the risk of P. aeruginosa primocolonization in children form 0 to 18 years old with cystic fibrosis
Exploratory proof of concept study to determine whether intranasal administration of QR-010 in subjects with cystic fibrosis, homozygous or compound heterozygous for the ΔF508 mutation, can increase the function of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR).
The purpose of this research study is to evaluate the safety and effectiveness of Interferon gamma-1b (IFN-g 1b) on lung function when given to patients with cystic fibrosis by inhalation (breathed into the lungs) three times a week for 12 weeks. The FDA has not approved Interferon gamma-1b for use with cystic fibrosis patients, which is the condition being examined in this study.
Vitamin D deficiency is common in cystic fibrosis. Vitamin D deficiency frequently persists despite aggressive treatment with ergocalciferol, a vitamin D preparation also known as vitamin D2. Cholecalciferol, a vitamin D preparation also known as vitamin D3,may work better to increase vitamin D levels. Vitamin D is important for absorption of calcium from the diet and bone health. Vitamin D more recently has been found to play a role in regulating the normal inflammatory proc...
This is a phase II, dose ranging, multicenter, randomized, double-blind, placebo-controlled study. The aim of this study is to assess the safety of increasing doses of roscovitine administered orally for 4 cycles of 4 consecutive days (treatment "on") separated by a 3 days treatment free period (treatment "off") in adult CF subjects with Cystic Fibrosis carrying 2 Cystic Fibrosis causing mutations with at least one F508del-CFTR mutation and chronically infected with Pseu...