Active clinical trials for "Cystic Fibrosis"

This dose escalation study will evaluate 4 doses of RSP-1502 in sequential cohorts of 8 subjects each. In each cohort, 6 subjects will receive RSP-1502 and 2 will receive active control. Study drug (RSP-1502 or active control) will be administered by inhalation twice daily (BID) for 14 days. Planned RSP-1502 doses include 300 mg tobramycin plus ascending doses of CaEDTA (16 mg, 32 mg, 75 mg and 150 mg). Dose escalation will proceed after Safety Review Committee (SRC) review of the safety and tolerability data from the previous cohort. The SRC will determine the maximum tolerated dose (MTD) after completion of the fourth cohort. Following determination of the MTD, a fifth cohort (n = 20) will be randomized (1:1) to treatment with RSP-1502 (at the MTD) or active control administered BID for 14 days. All subjects will be followed for 14 days after completion of dosing.

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.

Cystic fibrosis (CF)-related diabetes (CFRD) is the most important emerging complication after pulmonary complications. This specific form of diabetes is associated with an increased morbidity and mortality. CFRD prevalence at the age of 10 is 10% and reaches 40 to 50% in adulthood, while a similar percentage is afflicted with milder dysglycemia also called pre-diabetes abnormalities. In order to identify patients at risk and to implement early therapeutic measures, an annual CFRD screening test is recommended for CF patients after 10 years of age. The standard 2-hour oral glucose tolerance test (OGTT) is the recommended screening test. However, this test is perceived by both patients and CF care teams as unpleasant while adding a significant burden and workload, resulting in screening rates lower than 50% in most centers. An ideal alternative test should be simpler, less invasive, more sensitive than an OGTT to establish risks for lung function and/or nutritional deterioration, and predict future CFRD risk. To date, compared to the OGTT, no alternative screening method has demonstrated its effectiveness. However, continuous glucose monitoring (CGM) is emerging as a possible alternative method. In patients living with CF, CGM is easy to use and can identify early dysglycemia, which in turn, can predict increased risk of accelerated decline of pulmonary function and/or weight, higher risk of pseudomonas colonization, and future risk of CFRD. However, these observations are based on studies of small sample size with very limited prospective data. Furthermore, many of the multiple CGM metrics that have been standardized are based on the risk of complications associated with Type 1 and Type 2 Diabetes. Thus, there is a need for prospective studies to identify the CGM metrics and the cut-off level that is relevant as a predictor of clinical deterioration and/or CFRD risk in CF. The identification of such CF-specific criteria would provide important information to target at-risk patients.

In this study, the investigators aim to evaluate changes in lung function in women with cystic fibrosis (CF) during pregnancy and for 2 years after pregnancy based on exposure to highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators.

This observational study evaluates the effect of therapy with cystic fibrosis transmembrane regulator (CFTR) modulators on CFTR function measured by the CFTR biomarker intestinal current measurement (ICM), nasal potential difference (NPD) and sweat chloride in a post-approval setting in patients with cystic fibrosis (CF).

The composition and role of the pulmonary microbiota is not yet well described in cystic fibrosis patients. The objective of our longitudinal follow-up of primary colonised patients is to show the presence of a link between the composition of the microbiota and the effectiveness of antibiotic therapy. All patients followed at the Montpellier CF center will be asked to participate in this cohort. All patients have a regular follow-up every 1 to 6 months and will be asked at each visit to keep their sputum sample in excess of the analyses requested for their follow-up

The purpose of this protocol is to begin an exercise program combined with behavioral counseling for patients who are hospitalized with a cystic fibrosis exacerbation. The exercise program will begin during the hospital stay. Beginning an exercise program during this period of reduced mobility and isolation may be an ideal time to deliver a structured exercise prescription along with a behavioral program to promote long-term adherence to exercise (structured physical activity) . Hospitalized patients have an acute awareness that their lung function is declining and may be more motivated and open to changing their behavior and adding exercise to their treatment regimen.

This study collects data on microbiological factors and lung function parameters (e.g. spirometry, body plethysmography, lung-MRI) to assess their interaction on the lung growth and lung development of infants and children with Cystic Fibrosis (CF).

Cystic fibrosis (CF) is a common inherited condition in the Caucasian population resulting in poor function and/or production of the CF transmembrane conductance regulator (CFTR) protein. The CFTR protein plays a crucial role in the secretion and re-absorption of sodium chloride within the sweat gland. The sweat gland has played a key role in diagnosing and understanding CF with sweat chloride elevation being a key criterion to diagnosing CF. People with CF are thought to be at risk of exertional heat illness during exposure to hot environments or during prolonged periods of exercise and are currently encouraged to take salt supplements during periods of excessive sweating. Kaftrio®, a newly approved pharmacological therapy has shown a rapid and sustained reduction in sweat chloride levels on initiation of this treatment. This study will aim to play a crucial part in understanding the sweat response, sweat composition and the thermoregulatory response to exercise in the heat in people with CF on Kaftrio®.

The purpose of this research study is to examine if the use of antioxidant supplements impacts exercise intolerance in people with CF.