Active clinical trials for "Cystic Fibrosis"

A prospective single center observational cohort of patients with cystic fibrosis to determine whether adequate serum levels of posaconazole, after administration of the newer modified release once daily oral formulation, can be achieved.

There have been substantial advances in care for individuals with CF, including improved nutrition and respiratory care. Women with CF are now likely to survive into adulthood with a median predicted survival of 49.7 years in 2012, which have resulted in increasingly normal lifestyles, including successful pregnancies in women with CF. The number of pregnancies in CF women has increased as reported by the 2013 Annual Data Report from the French CF Registry (6 pregnancies in 1992 to 53 in 2012). Pregnancy adds many physiological stresses to the body, particularly on pulmonary function (decrease in residual volume, expiratory reserve capacity) and nutritional status (increase in nutritional needs). In women with CF, these added stresses could theoretically affect survival, with difficulties to maintain adequate nutrition and an unpredictable effect on lung function. A number of studies published over the past 15 years have attempted to clarify the risk of pregnancy in women with CF to guide clinicians and women. Many authors compared the outcomes of pregnant CF women to non-pregnant CF women regardless of nutritional status, lung function, diabetes mellitus, bacteriological colonization. In literature, pregnancy was possible and well tolerated in CF women with mild disease if associated with more intensive monitoring and aggressive treatment during pregnancy, with particular emphasis on nutrition and weight gain. CF women have an increased likelihood of receiving treatment for diabetes both during and after pregnancy. Furthermore diabetes mellitus has been associated with more severe pulmonary disease, more frequent pulmonary exacerbations, poorer nutritional status and a reduced life expectancy. Pre-pregnancy diabetes mellitus is associated with a poorer prognosis for pregnancy in general population. Severe forms of CF, including pre-pregnancy diabetes mellitus might as well do badly with accelerated decline after pregnancy. To determine the effect of pre-pregnancy diabetes on maternal decline after pregnancy, we will compare FEV1 and BMI rates of decline, during a two-years follow-up period after pregnancies, reported in the French CF Registry, according to their diabetic status.

Patients and parents of patients will be asked to answer a questionnaire on paper or online, by the CRCMs and through communication campaigns distributed by the association Vaincre La Mucoviscidose (VLM). This questionnaire includes about fifty items that will take 5 to 12 minutes to complete. The study aims to interview 250 patients / parents of patients, users and non-users of essential oils. Thirty patients or volunteer parents will then meet with a health anthropologist for qualitative interviews to document very precisely the modalities of use of essential oils: which ones, at what dose, advised by whom, the dedicated budget. Relationships with CRCM doctors, the effects felt, the side effects will also be explored. The global data of the two phases will be integrated and put into perspective with the scientific data currently available on essential oils.

Our project is to describe retrospectively and prospectively CF patients treated with biotherapy in French CF centers. Main objective: To describe the clinical and paraclinical course of CF patients before and after treatment with anti-IL5 and other biotherapies since 2019. Secondary objective: To describe adverse events potentially related to the biotherapies.

The main objective of this study is to assess the ovarian reserve of CF patients via measurement of AMH and AFC. Secondly, we aim to correlate between the ovarian reserve and demographic and clinical characteristics of these patients. Outcomes measured will be levels of AMH and AFC, as compared to standard means in the general population. There is no need for a control group in this study since the standardized means of AMH and AFC in the general population are a more accurate comparable measure, which is based on data collected from large-scale populations, and thus accounts for confounding age factor in a more complete manner than can be obtained via a control group (Almog et al., 2011, La Marca et al., 2012, Penzias et al., 2020).

The purpose of this study is to explain the provision of palliative care at the end of life by the implementation of the ELNEC course, as WBT Program using the Normalization Process Theory, that focus attention on how complex interventions become routinely embedded in practice. In addition to, identify the changes implemented by the participant nurses (intervention group) in their clinical practice, after participating in WBT Program to provide Palliative Care alongside with usual care versus usual care only (control group) for children with life-limiting conditions or in the case of accidents/sudden death, at the end of life. And finally, provide findings that will assist in the interpretation of the trial results.

Diabetes affects half of cystic fibrosis patients aged 30 years and older. It develops asymptomatically for a long time. Also, two options are possible: start insulin treatment now with the additional constraints associated with cystic fibrosis or wait while monitoring the patient's clinical status and initiate insulin treatment when he has developed symptoms and therefore later. In practice, the choice between these two options takes place over two medical consultations without a formalized shared decision-making process between the doctor and the patient. Shared decision-making is a decision-making process in which the healthcare provider and the patient learn about patients care options and then deliberate to reach a common agreement on the decision taken. Shared decision-making seemed particularly relevant to us in cystic fibrosis where there are complex treatment options with variable short-, medium- and long-term side effects and where the disease and its treatments have a high impact on the patient's quality of life.

This project entitled "Impaired secretory IgA and mucosal immunity in cystic fibrosis" is a research program which aims to determine, owing to national (KULeuven) and international (Descartes university Paris, university of Torino) collaborations for expertise and access to human material, whether a defect exists for the production of IgA antibodies in the lung from patients with this serious genetic disease. These antibodies line and protect normally the airways, and are secreted through a specific epithelial receptor called pIgR (polymeric immunoglobulin receptor); its expression and regulation will be studied in lung tissue and in cell cultures of the lung epithelium from these patients. The link between the putative IgA defect and chronic bacterial infection with Pseudomonas aeruginosa, which often complicates the evolution of the disease, will also be evaluated ex vivo and in vivo, in an animal model of lung infection.

The general objective is to elucidate the mechanisms whereby sex hormones may modulate the severity of respiratory disease. An important component of this proposal is a systematic and intensive approach to characterize how the cellular and cytokine components of airway inflammation respond to fluctuations in sex hormone levels. The effects of menstrual fluctuations in levels of sex hormones on inflammation and bacterial load in respiratory secretions of CF patients will also be determined.

n2015, VERTEX company - producing already KALYDECO (IVACAFTOR, VX-770) potentiator molecule that is recommended for the treatment of CF patients aged ≥ 6 y, with CFTR mutation altering the channel regulation (class III mutations) as G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549Nou S549R) -was allowed by the Federal Drug Administration (FDA) and European Medicines Agency (EMEA) for producing and using ivacaftor combination (such as lumacaftor/ ivacaftor initially, and more recently tezacaftor/ivacaftor, tezacaftor/ivacaftor/VX-659, tezacaftor/ivacaftor/VX-445 and tezacaftor/ivacaftor/VX-152) in clinical trials for patient with cystic fibrosis, according to age and mutation eligibility criteria. Since 2016, the French patients homozygous for the p.Phe508del mutation and older than 12 years are able to be treated with the association LUMACAFTOR-IVACAFTOR and this French authorization is being extended for 6-11 years old children (while the European Commission has already granted an extension of the Marketing Authorization for lumacaftor/ivacaftor to include 6-11 years old children with cystic fibrosis since January 2018). Patients treated by lumacaftor/ivacaftor (or other ivacaftor new combinations) are closely monitored according to criteria established by the working group "New Therapeutic Approaches" of the French Society Cystic fibrosis. This study is a phase IV observational trial for a period of 1 year. In this context, the team aims at initiating a comprehensive monitoring of the lung and gut mycobiota and microbiota evolution under LUMACAFTOR-IVACAFTOR (or other ivacaftor combinations) treatment. This project is directly linked to the monitoring of cystic fibrosis patients who begin treatment with LUMACAFTOR-IVACAFTOR (or other ivacaftor combinations) in France. The pro- and eukaryotic microbiota analysis is based on the secondary use of sputum and stool samples associated with several clinical data of CF patients under ivacaftor combinations and follow-up during the 1st year of therapy. According to the French law, Lum-Iva-Biota project is a non-interventional study. It aims at demonstrating that changes in the hydration of secretions at the pulmonary and intestinal levels related to LUMACAFTOR-IVACAFTOR therapy (or other new generation of ivacaftor combinations) promote a change in the lung and gut mycobiota and microbiota profiles which may achieve the characteristics of the "healthy type" (in terms of composition, richness and diversity).