View clinical trials related to Cystic Fibrosis.
Filter by:Assessment of the outcome of patients not concluded fir cystic fibrosis, either symptomatic patients or asymptomatic children detected by newborn screening for cystic fibrosis. The aim is to identify patients potentially at risk of progressing to the clinical spectrum of cystic fibrosis
The purpose of this study is to evaluate the relative bioavailability, food effect, and dose proportionality of a granule formulation of VNZ/TEZ/D-IVA.
The purpose of this study is to evaluate the flavor (basic tastes, aroma, texture, mouthfeel) of VNZ/TEZ/D-IVA fixed dose combination (FDC) granules.
Newborn bloodspot screening (from now on referred to as screening) for cystic fibrosis (CF) became part of the national screening programme in 2007. Screening for CF is also well established internationally. The current process works well but has some disadvantages: carrier reporting - which is not the intention of CF screening in the UK (~200 pa); need for repeat samples which can be costly and contribute to parental worry (~300 pa.); mutation panels not fully reflecting the ethnic diversity of the birth population; identification of children designated as CF screen positive, inconclusive diagnosis (CFSPID) which can cause uncertainty (~20-30 pa). A trial of NGS in one centre in the UK, for one year found that it was technically feasible at reasonable cost and with an acceptable turn around time. In addition, the trial determined that using NGS could mitigate against some of the disadvantages described above. The purpose of this piece of work was to: 1. Gather, compare and analyse the views of a range of stakeholders on the proposed CF screening protocol incorporating NGS. 2. Use the outcomes to inform discussions and decisions by the fetal, maternal and child health (FMCH) group and UK National Screening Committee (NSC) about the proposed protocol 3. Consider what generalisable information on the views of stakeholders on newborn screening could be generated from this exercise to inform other FMCH and UK NSC discussions 4. Evaluate and learn from the exercise to inform future stakeholder engagement activities by the UK NSC and screening programmes.
The goal of this observational prospective study is to determine the health impact of parenthood on United States (US) people with CF in the era of CF transmembrane regulator protein (CFTR) modulators. The investigators will collect physical and mental health data to comprehensively evaluate the impact of parenthood in CF with widespread highly effective CFTR modulator use. The main hypotheses this study aims to examine are: H1: Parents with CF and moderate-to-severe depression have more rapid change in ppFEV1 (percent predicted forced expiratory volume in one second) versus those with mild or no depression. H2: Parents with CF who have more parental responsibility and/or stress have more rapid ppFEV1 (percent predicted forced expiratory volume in one second) change than those with less responsibility/stress H3: Parents using CFTR modulators have decreased ppFEV1 (percent predicted forced expiratory volume in one second) change versus those not using CFTR modulators Participants will complete quarterly surveys during the first year of parenthood and biannual surveys, thereafter, using the computer-based survey system on an iPad protected for infection control or via personal device or computer via emailed survey link.
The goal of this methodological study is to determine the reliability and validity of the The Breathlessness Beliefs Questionnaire Caregiver Version (BBQ-C) version in assessing dysfunctional breathlessness beliefs in caregivers of children with cystic fibrosis.
The aim of the present study is to investigate the effect of probiotic supplementation on GI related quality of life, through a randomised placebo-controlled clinical trial. Moreover, the invetigators wish to study CF microbiota and intestinal inflammation in the setting of probiotic supplementation and newly started treatment with a highly effective CF-specific treatment, elexacaftor-tezacaftor-ivacaftor (ETI). The proposed project has the potential to increase QoL and decrease GI morbidity in children with CF. If successful, the results of this study can contribute to alter the care of CF patients by including supplementation of probiotics in routine CF care. Morever, the study can provide much needed insights to GI microbiota and inflammation in pediatric CF patients.
Cystic fibrosis (CF) is a life-threatening exocrine gland disease that is often diagnosed in childhood, and its incidence tends to increase and affect physical and mental health.The purpose of this study is to evaluate the effectiveness of the Intervention Program Based on Strengthening the Psychological Resilience of Children Diagnosed with Cystic Fibrosis and Their Mothers, prepared for children with CF (8-12 years old) and their mothers.In the first stage, the CF experiences of children with cystic fibrosis and how they perceive these experiences will be examined with visual phenomenology, and their mothers' CF experiences and perceptions will be examined with phenomenology. In this regard, 10 children will be asked to draw pictures and their pictures will be analyzed. In-depth individual interviews will be held with mothers.In the 2nd stage, the effectiveness of the Intervention Program Based on Strengthening Psychological Resilience for Children Diagnosed with Cystic Fibrosis and Their Mothers will be determined with a pre-test-post-test randomized controlled experimental design.In the literature review, Gpower analysis was performed (α: 0.05, β: 0.99, d: 1.88) based on the results of studies conducted with children diagnosed with CF and their parents, similar to this study, and 15 samples were used for each group. It is planned to recruit people. Considering the possibility of data loss in the study, it was always deemed appropriate to include 22 people for the group. It was planned to sample a total of 88 children with CF and their mothers, 44 in the intervention group and 44 in the control group. In the 3rd stage, after the experiment, the participants' experiences regarding the intervention program will be examined through interviews and qualitative research method. Thus, qualitative interviews will be conducted before and after the experiment. Data will be collected using these tools: Children; Child and Mother Descriptive Characteristics Form, Semi-Structured Interview Form,Respiratory Function Test, Reorganized Cystic Fibrosis Questionnaire, Psychological Resilience Attitude and Skills Scale and Functional Disability Inventory; Mothers; The Revised Cystic Fibrosis Questionnaire, The Depression Anxiety and Stress Scale, Post-Traumatic Growth Scale and Brief Psychological Resilience Scale. Measurements will be taken before the intervention, after the intervention, at the 1st and 3rd months.
Coronavirus disease 2019 (COVID-19) which is caused by the virus SARS-CoV-2 has resulted in an ongoing global pandemic. It is unclear whether the relatively low number of reported cases of COVID-19 in people with CF (pwCF) is due to enhanced infection prevention practices or whether pwCF have protective genetic/immune factors. This study aims to prospectively assess the proportion of pwCF, including both adults and children with CF who have evidence of SARS-CoV-2 antibodies over a two-year period. This study will also examine whether pwCF who have antibodies for SARS-CoV-2 have a different clinical presentation and what impact this has on their CF disease. The proposed study will recruit pwCF from paediatric and adult CF centres in Europe. Serological testing to detect antibodies will be performed on blood samples taken at month 0, 6, 12, 18 and 24 with additional time-points if bloodwork is available via normal clinical care. Clinical data on, lung function, CF-related medical history, pulmonary exacerbations, antibiotic use, and microbiology and vaccination receipt, will be collected during routine clinical assessments. Associations will be examined between socio-demographic and clinical variables and serologic testing. The effects of SARS-CoV-2 infection on clinical outcomes and analyse end-points will be examined to explore any age-related or gender-based differences, as well as subgroup analysis of outcomes in lung-transplant recipients and pwCF receiving CFTR modulator therapies. As pwCF receive COVID-19 vaccination a comparison of the development and progression of anti-SARS-CoV-2 antibodies in pwCF following natural infection and vaccination SARS-CoV-2 over time will be performed.
The goal of this observational study is to translate and validate the Adt-Physio scale in French, and to validate on a large number of patients the French translation of the Adt-Physio scale as a tool for evaluating adherence and perception of the intervention of a physiotherapist in patients with cystic fibrosis. participants will have to answer the French trans-AdT scale, the Brief Cope and GSES questionnaire.