View clinical trials related to Cystic Fibrosis in Children.
Filter by:Some parents may be more protective of children with CF due to concerns about worsening of the disease due to infection, which can affect their functional level. The goal of this observational study is to learn about the family's protective approach to the functioning and disease course of children with cystic fibrosis (CF) to determine whether there are possible negative effects. There will be an alternative viewpoint offered to clinicians regarding the management of CF with outputs of this study.
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 is the most common severe genetic disease with autosomal recessive transmission in the Caucasian population. Its prognosis has improved considerably since the creation of Cystic Fibrosis centers (CF centers) and the improvement of symptomatic management (nutrition, antibiotic therapy, transplantation, etc.). Thus, the median survival rate is now 46 years, whereas it was 5 years in 1963. The current challenges for cystic fibrosis patients are therefore twofold: to continue to improve their survival, and to improve their quality of life (QoL) to promote "healthy ageing" with this pathology that begins in childhood. In 1980, the World Health Organization (WHO) stated that functional capacity explorations best reflected the impact of chronic disease on health-related quality of life. Impairment of physical activity is common to chronic diseases, as in cystic fibrosis, where respiratory impairment and denutrition have been shown to contribute to reduced exercise tolerance and increased dyspnoea. Measurement of the maximum oxygen consumption (VO2max) by a cardiopulmonary exercise test (CPET) is regarded as the gold standard exercise test in the measurement of aerobic exercise capacity. In 2005 Pianosi et al. found that for children with cystic fibrosis, the rate of decline of VO2max measured by CPET was predictive of poorer quality of life. Continuing to study the determinants associated with impaired aerobic fitness in cystic fibrosis offers the hope of considering appropriate therapies to further improve the quality of life of these patients. In recent years, the arrival and widespread use of CFTR protein modulators in children has been a real turning point and makes it possible to envisage a drastic change in the history of this disease and its prognosis in the long term. Thus, in this study, the investigators aimed to assess the aerobic fitness, assessed by a CPET, of children with cystic fibrosis, and to compare the results with healthy controls. Secondly, investigators wanted to identify the predictive factors of VO2max in children with cystic fibrosis.
This is a prospective, observational study examining the impact of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators on chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) in young children with cystic fibrosis (YCwCF). This study involves two groups: children 2-8 years old, inclusive at initial visit, receiving highly effective modulator therapy (HEMT), and a control group of children 2-8 years old, inclusive at initial visit, not receiving HEMT. Outcomes will include sinus magnetic resonance imaging (MRI) scans, olfactory tests, and quality of life surveys obtained over a two-year period.
Cystic fibrosis (CF) is the most common hereditary life-threatening condition in Belgium. Because of a dysfunctional cystic fibrosis transmembrane conductance regulator (CFTR) channel, chloride is unable to move to the cell surface and mucus becomes more viscous. Consequently, CF patients are not able to clear their lungs efficiently, and trapped bacteria can lead to chronic infection and inflammation of the lungs, and ultimately respiratory failure. CF lung disease starts at birth due to muco-inflammatory processes and is associated with a significantly altered microbial colonization of the infant airways compared to infants without CF. Additionally, young children with CF suffer from viral infections as often as their healthy peers, but the episodes are more severe and often prolonged. Moreover, frequent viral infections in children with CF contribute towards a more pathogenic airway microbiome at a young age. Although this link has been previously reported, the exact mechanisms by which this occurs need to be elucidated. A pulmonary exacerbation in CF is characterized by an increase in respiratory symptoms, general symptoms and a decline in lung function. Most young children with CF suffer from a mean of 4 exacerbations per year for which antibiotics are prescribed. Despite the current novel therapies in CF, treatment of respiratory infections stay relevant and is a greater challenge with increasing survival. The key objective of this study is to gain insights into the mechanisms by which viral infections leading to pulmonary exacerbations induce a more pathogenic microbiome in young children with CF. About forty participants will be recruited at the paediatric CF clinic of the Antwerp University Hospital. Inclusion criteria are an age of less than 5 years and a diagnosis of CF. There are no exclusion criteria. Duration of the study is 1 year to cover for seasonality of clinical symptoms. Study visits are scheduled at 3-month intervals corresponding with the regular follow up, or unscheduled during an acute pulmonary exacerbation. From all participants, two oropharyngeal swabs (for microbiome analysis and for immunological/mucin analysis) will be collected at set time points. For the linking of the laboratory data to the clinical characteristics, we will examine demographics, environmental exposures, and disease markers of CF. Next to the collection of the oropharyngeal swabs, a history, physical examination, and technical investigations will be performed at the study visits.
The goal of this clinical trial is to to assess the nutritional status of children diagnosed with cystic fibrosis between the ages of 2-14, to determine the changes in the nutrition education given to the mothers of the patients on the nutritional status of the children and the nutritional knowledge of the mothers, and to compare them with the control group.. The main questions it aims to answer are: 1. Will be better the nutritional status of children of mothers in the education group will be better compared to the control group? 2. Will the macro and micronutrient Recommended Dietary Allowance (RDA) and diet quality of children with cystic fibrosis in education group increase after nutrition education? 3. Will the nutrition knowledge test scores of mothers of children with cystic fibrosis in education group on "Nutrition in Cystic Fibrosis" increase after nutrition education? Mothers of children diagnosed with cystic fibrosis will participate in the study as a result of the call of the Cystic Fibrosis Association in Turkey. Mothers participating in the study will be divided into 2 groups. While the mothers in the first group will be given nutrition education 3 times a week, 1 hour a day, no education session will be given to the mothers in the control group. The nutritional status and nutrient consumption of children in the training group and the control group will be compared.
This study capitalizes on the emerging technology of 19F MRI, using conventional 'thermally' polarized perfluorinated gas (perfluoropropane, or PFP) mixed with oxygen and studied with magnetic resonance imaging (MRI) to visualize ventilation. This technique has not been studied in children. Children and adolescents (6-17 years old) with cystic fibrosis (CF) who have normal spirometry will undergo 19F MRI with the inhalation of an inert contrast gas to study ventilation. Comparisons will be made to a cohort of healthy children (6-17 years old) who will perform the same measures. The primary outcome measure is the feasibility of conducting these studies in the pediatric population. Parallel performance of multiple breath nitrogen washout (MBW) and spirometry will be used to compare the sensitivity of these outcomes to the presence of mild lung disease in these children. Finally, the investigators will compare data obtained during standard breath holds with a novel "free-breathing" technique that will eliminate the need for breath holds during MRI acquisition.
The goal of the clinical trial is to test whether a mental health program that is delivered through the Internet works well for children and adolescents with cystic fibrosis (CF) and their healthy siblings. The main questions it aims to answer are: - Does the program improve the mental health such as depression and anxiety symptoms? - Does the program improve overall quality of life? - Does the program improve self-efficacy - an individual's belief in their ability to complete tasks to achieve their goals? Participants will: - Fill out an online survey asking questions about their personal and health information, as well as their mental health before the program - Complete the online mental health program - Fill out an online survey asking questions about their mental health after completing the program, and 1-month and 3-months following completing the program Participants be compared against another group of children with CF and their healthy siblings who are on a waitlist and receiving usual CF treatment. Researchers will compare participants scores before starting the program with their scores immediately following completing the program, 1-month, and 3-month after completing the program. Researchers hope to develop a program that improves mental health, quality of life, self-efficacy, and knowledge about CF.
The goal of this clinical trial is to investigate the effects of a-12-week virtual reality exercise program, applied as tele-exercise, on the exercise capacity of children with cystic fibrosis, whose physical activity level is further restricted during the pandemic. The main questions it aims to answer are: Does aerobic tele-exercise increase functional capacity in cystic fibrosis patients? Will there be a difference in treatment outcomes between the virtual reality group and the online exercise group? Participants will be randomized into two groups; virtual reality group and online group. Physical activity will be provided to the virtual reality group in the form of team activities in a virtual environment, and the other group will be provided online exercises at their home. For this purpose, virtual reality headsets will be provided to patients to give the impression of a natural, immersive environment and realistic experience. Researchers will compare exercise compliance and functional capacity between the virtual reality group and the online group.
This study will investigate if parental physical activity levels, assessed by providing a physical activity questionnaire to parents of children aged 6-16 with Cystic Fibrosis (CF), is associated with their child with CF's physical activity levels. Children's activity levels will be taken from electronic records where a questionnaire is routinely given at annual reviews to analyse this. Parental activity levels will also be compared against adherence to nebulisers as a proxy for adherence to treatment, this data is again in the electronic records of patients and is collected at annual reviews.