View clinical trials related to Cystic Fibrosis.
Filter by:This study aims to assess the effects of programmed exercise with or without electrical stimulation in the cardiorespiratory fitness, strength, functional capacity and agility in a group of young patients with Cystic Fibrosis: A randomized controlled trial comparing two interventions with a control group.
To obtain prospective real world data of the effect of lumacaftor/ivacaftor or tezacaftor/ ivacaftor on small airway disease in children aged 6-18 years with cystic fibrosis (CF) homozygous for F508del. The effect of the medication on small airway disease is evaluated by measurement of multiple breath washout (MBW) with its outcome parameter lung clearance index (LCI) and the Perth-Rotterdam Annotated Grid Morphometric Analysis for CF (PRAGMA-CF) cpmputed tomography (CT) score. In addition the relation between changes in LCI and PRAGMA-CF score is evaluated.
This is a Phase 2 open-label, dose-escalation study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 with and without ivacaftor in patients with CF with at least one G542X allele. In total, up to 16 patients will be enrolled in the trial; up to 4 patients will be homozygotes for G542X, and the remaining patients will be compound heterozygotes with one G542X or phenotypically similar nonsense allele and any Class 1 or Class 2 mutation. Each patient will receive up to 5 escalating doses as follows: - ELX-02 0.3 mg/kg per day SC - ELX-02 0.75 mg/kg per day SC - ELX-02 1.5 mg/kg per day SC - An individualized dose of ELX-02, as high as 3.0 mg/kg per day SC, based on the patients observed safety and tolerability, PK at previous doses and the results of laboratory tests. - ELX-02 1.5 mg/kg per day SC plus 150 mg ivacaftor every 12 bid
The purpose of this study is to explore and collect the perceptions, expectations and needs of CF patients about parenting. This will be done in the context of several small groups of patients led by a psychologist who will ensure that all the participants express themselves; he will encourage them to develop their points of view, their divergences and their common points about what constitutes to be a parent. The collected information should make it possible to develop and propose adapted medico-psycho-social interventions, if necessary, in connection with patient associations
This is a Phase 2 open label study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 with and without ivacaftor in patients with CF with at least one G542X allele or phenotypically similar nonsense allele. Up to 16 patients will be enrolled in the trial; up 4 patients will be homozygotes to G542X, and the remaining patients will be compound heterozygotes with G542X or phenotypically similar nonsense mutation and any Class 1 or Class 2 mutation. Each patient will receive 5 escalating doses as follows: - 0.3 mg/kg per day SC - 0.75 mg/kg per day SC - 1.5 mg/kg per day SC - An individualized dose, as high as 3.0 mg/kg per day SC, based upon the patients observed safety and tolerability, PK at previous doses and the results of laboratory tests - ELX-02 1.5 mg/kg per day SC plus 150 mg ivacaftor every 12 bid
The study will assess if administration of high-dose vitamin D and a commonly used prebiotic (inulin) is effective to reduce gastrointestinal dysbiosis and to improve critical intestinal functions in Cystic Fibrosis with the additive or synergistic effects of the combination of vitamin D + inulin.
The objective of the study is to assess the utility of "Acceptance and Commitment Therapy" (ACT) in which subjects learn new ways to manage uncomfortable experiences and feelings and to engage in positive behaviors, over "Supportive Psychotherapy" in which subjects talk about their experiences to date in a cohort of adult Cystic Fibrosis patients. The hypothesis is that six telehealth/webcam sessions of ACT will lead to an improvement in medication and visit compliance, as well as an overall improved sense of well-being and coping skills, particularly as compared with 6 telehealth/webcam sessions of supportive psychotherapy.
This study will evaluate the efficacy, safety, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for F508del.
Regular bronchial clearance is essential in patients with cystic fibrosis for their bronchial health. SIMEOX® (Physio-Assist, Aix en Provence, France) is an innovative medical device for the drainage of the bronchial tree. By changing the rheological properties of mucus, SIMEOX® helps to mobilize secretions and assists their transport to the upper airways. This technology is based on fundamental research on bronchial mucus rheology. At the present time, SIMEOX® device is mainly used over a short period at the time or after an exacerbation in healthcare structures (hospitals, physiotherapy practices, postcare, and rehabilitation units, etc…). The clinical effects observed in the short term encourages long-term autonomous use by the patients themselves. The overall objective of this study is to evaluate the efficiency and acceptability of SIMEOX® used at home by the patient himself for bronchial clearance in patients with cystic fibrosis.
"The usefulness of respiratory physiotherapy and its execution modalities remains highly debated even though reviews of the literature show that respiratory physiotherapy is able to improve the drainage of bronchial secretions and pulmonary function tests during cystic fibrosis in periods of stability. Different physiotherapy techniques have been developed but the choice of one or the other facing a patient can not currently be recommended. The VirtualChest project, supported by a grant from the National Agency for Research (ANR), aims to develop and validate a physical model of respiratory physiotherapy (6 stages including model establishment pulmonary: bronchial tree and pulmonary mechanics and parietal [steps 1 and 3], a model of mucus [step 2] and modeling the effect of physiotherapy [step 5]). This project is integrated with stages 3 and 5 of this broad project and aims to get on a limited number of children with cystic fibrosis a proof of concept (prediction of drainage efficiency) and especially to feed the proposed physical models in order to subsequent optimization of the model (step 6). The choice cystic fibrosis of the child was justified by the effect demonstrated respiratory physiotherapy, particularly on respiratory functional criteria, and the fact that the parietal mechanics varies physiologically at this age."