Active clinical trials for "Cystic Fibrosis"

The main causes of acute recurrent pancreatitis (ARP) or chronic pancreatitis (CP) in children are defects in genes that have been associated to pancreatitis. Among these gene defects CFTR gene mutations are commonly found, 34% ARP and 23% CP . Since not every CFTR gene mutations clinically manifest, just identifying these CFTR gene mutations may not help to establish a clear role of this defect in the etiology of the individual ARP/CP. The novel beta-adrenergic sweat secretion test is a very sensitive test to detect small abnormalities in CFTR function in form of a linear gene-function relation. By identifying even mild CFTR defects, in future will help in finding the role of CFTR modulators and providing treatment to these patients.

This is a long-term study in cystic fibrosis patients who are participating in the Cystic Fibrosis Patient Registry to assess the occurrence and risk factors for a rare bowel disorder called fibrosing colonopathy (narrowing of the large intestine). Patients will be followed at their regular clinical care visits over a 10-year period and approached if they develop symptoms of fibrosing colonopathy for collection and use of further detailed information.

The purpose of this study is to characterize bacterial diversity and richness in the sputum of cystic fibrosis patients treated with every-other-month TOBI™ Podhaler™ and continuous alternating therapy with TOBI™ Podhaler and colistimethate (Colistin).

This is an observational comparative study to compare the standard-of-care methods for sweat testing used to support diagnosing cystic fibrosis (CF) against a new method of sweat testing called the CF Quantum Sweat Test System. The results of the new device in this study are for comparison ONLY and will NOT be used to diagnose, treat or mitigate the subject's condition.

International, multicenter, observational, longitudinal study to identify biomarker/s for Cystic fibrosis and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s

OBJECTIVES: I. Compare the clinical status of pregnant vs non-pregnant women with cystic fibrosis. II. Determine glucose tolerance during each trimester of pregnancy in these women. III. Evaluate peripheral insulin sensitivity in these women. IV. Evaluate whole body protein turnover and hepatic glucose production in these women. V. Determine resting energy expenditure in these women.

The objective of this study is to determine if interactive technology (i.e. virtual coaching) that facilitates vest therapy goal setting and provides positive reinforcement will increase adherence to vest therapy for airway clearance in individuals with CF.

Children with Cystic fibrosis (CF) have to undergo several needle-related procedures during outpatient visits or hospital stay. It is common for children with CF to display distress and behavioural problems during invasive procedures. Children with chronic diseases have a lower threshold of pain compared to non-chronic patients. Effective management of needle distress in children with CF is critical. Although pharmacological and non pharmacological methods are used during needle -related procedures to lower painful stimuli nearly all patients with CF experience anxiety. Distraction has been shown to be an effective technique that directs children's attention away from noxious stimuli. The application of Virtual Reality (VR) in the medical field has been shown to be successful already 15 years ago. VR has found its use during the medication of burns and in patients undergoing cancer treatments whereas so far few studies have been published to assess its efficacy in reducing needle related pain and distress in children and none in children with CF. Objectives To assess the efficacy of VR in reducing pain and distress during venipuncture in children with CF compared to routine care. Project description Randomized controlled parallel trial with a 1 to 1 allocation ratio. The use of VR compared to standard care during venipuncture in children with CF (age 6-18 years) attending the outpatient clinic of the CF Centre of Florence will be compared over the period of 1 year. Patients randomly assigned to arm A will use VR during the procedure, those patients assigned to arm B will receive routine care. Anticipated output Determination of the efficacy of VR in lowering pain and distress during venipuncture.

Cystic fibrosis related diabetes (CFRD) is a common co-morbidity in patients with CF. The underlying pathophysiology of cystic fibrosis related diabetes (CFRD) is still a matter of investigation. In addition to localized tissue damage developing similar to that of the exocrine pancreas, other mechanisms may be involved. We have shown that a potential contributing factor to the patho-physiology of CFRD may be an abnormal gut derived hormonal profile, specifically of lower incretin hormone responses, prior to development of CFRD. We propose that an altered incretin response, probably due to impaired interaction of nutrients with the gut mucosa due to thickened secretions, may play a role in the development of the disease. Specifically, low GIP and GLP-1, may explain the poor β-cell function observed in these patients prior to CFRD appearance. These incretins have known trophic effects on β-cells, and thus their lower levels may contribute to the development of quantitative as well as qualitative defects in β-cell function and thus may lead to the development of CFRD. Thus, increasing levels of these incretins using a DPP-IV inhibitor may improve glucose metabolism and delay/prevent the development of CFRD. We hypothesize that Saxagliptin will increase the oDI compared to placebo and will thus provide relative protection from diabetes development and in addition we expect that Saxagliptin will lead to overall increased insulin concentrations and thus shift the metabolic milieu to a more anabolic state. This will manifest as weight gain and reduction in inflammation.

Exercise helps people with CF lead long, fulfilling lives. Regular exercise reduces depression and fatigue, and may play a role in preventing exacerbation. However, little is known about exercise habits in adults with CF, or the barriers to exercise that they face. During the COVID-19 pandemic, lockdowns and social distancing saw gym memberships plummet; while sale of personal exercise technology, smart-watches, and interactive home equipment exploded. As this technology becomes more cost-effective and accessible, can it be leveraged to improve CF care? This study will examine barriers to exercise in CF and the role of smart devices in exercise. Adults with CF will participate in the Exercise Behaviors/Barriers Survey, followed by a qualitative discussion on attitudes toward exercise technology, and how CF impacts exercise. We anticipate that many perceived barriers will be universal (e.g. time), while others will be unique to CF. Participants will be fitted with wrist actigraphy (FitBit Inspire 2) to measure baseline exercise. They will receive access to three smartphone apps designed for home exercise, as well as an exercise website designed for the CF community (www.activate65.org), and receive weekly phone calls from the CF care team offering motivational interviewing. Actigraphy, heart and respiratory rate, and app usage will be analyzed. After four weeks, participants will no longer receive calls. Actigraphy will be monitored four additional weeks to determine if exercise is sustained. We anticipate that exercise will increase during the intervention period, and that some activity will be sustained in the follow up period. The findings of this study will pave the way for a larger trial using this "Team and Technology" approach to investigate clinical outcomes. This study highlights the critical and timely need to investigate the barriers to exercise in CF, and effective solutions to develop sustainable exercise habits using widely-available technology from home.