Effect of the CFTR-modulating Triple Therapy Elexacaftor - Tezacaftor - Ivacaftor
Cystic FibrosisThe aim of this study is to investigate the frequency distribution, cytokine profile and function of peripheral, mononuclear leukocyte populations (monocytes, NK cells, T/B lymphocytes) and their correlation to clinical and biochemical parameters in patients with cystic fibrosis receiving CFTR modulatory triple therapy consisting of elexacaftor, tezacaftor and ivacaftor and to compare it with patients without CFTR modulatory therapy and healthy control subjects.
Glucocorticoids Versus Placebo for the Treatment of Acute Exacerbation of Idiopathic Pulmonary Fibrosis...
Acute Exacerbation of Idiopathic Pulmonary FibrosisAcute exacerbation of idiopathic pulmonary fibrosis (AE-IPF) is associated with a poor prognosis, with a 3-month mortality rate of over 50%. To date, no treatment has been proven to be effective in AI-FPI. The interest of glucocorticoids is controversial and needs to be confirmed. This confirmation is mandatory to validate the improvement of the prognosis of EA-IPF under this treatment but also to search for unsuspected deleterious effects as it has been shown with immunosuppressants in stable idiopathic pulmonary fibrosis.
Trikafta/Kaftrio and Pseudomonas Aeruginosa
Cystic FibrosisThe goal of this observational study o is to define whether, how and to what extent treatment with Trikafta/Kaftrio directly affects Pseudomonas aeruginosa in individuals with cystic fibrosis. The main questions it aims to answer are: whether Trikafta/Kaftrio affects the bacterial phenotypes and susceptibility to antibiotics; whether Trikafta/Kaftrio impacts the bacterial virulence. Participants will be asked the permission to store and analyze P. aeruginosa isolates collected from respiratory samples for usual care plans before the initiation of treatment with Trikafta/Kaftrio and after 12 and 18 months of treatment. The results of bacterial analysis will be matched with clinical data at the specific time-points. We expect to define effects of Trikafta/Kaftrio on P. aeruginosa and identify bacterial phenotypes as possible risk factors for its efficacy.
Health Outcomes of Parents With Cystic Fibrosis
Cystic FibrosisParenthood StatusThis project will determine the health impact of parenthood on people with cystic fibrosis (CF). The study team will use retrospective data to provide relatively immediate evidence on parenthood's effect on pulmonary health.
A Study to Test How Well BI 1291583 is Tolerated by People With Cystic Fibrosis Bronchiectasis (Clairafly™)...
Cystic FibrosisBronchiectasisThis study is open to adults aged 18 years and older with cystic fibrosis bronchiectasis. The purpose of this study is to find out whether a medicine called BI 1291583 is tolerated by people with cystic fibrosis bronchiectasis. Participants are put randomly into 2 groups. One group takes BI 1291583 tablets and the other group takes placebo tablets. Placebo tablets look like BI 1291583 tablets but do not contain any medicine. Participants in both groups take 1 tablet once a day for 12 weeks. Participants have twice the chance of being placed in the BI 1291583 group than in the placebo group. Participants are in the study for about 6 months. During this time, they visit the study site 7 times. At the visits, the doctors check the health of the participants and note any health problems that could have been caused by BI 1291583.
Efficacy and Safety of ME-015 (Suplatast Tosilate) in Cough Related to Idiopathic Pulmonary Fibrosis...
Idiopathic Pulmonary FibrosisCoughOrally administered ME-015 (Suplatast Tosilate) has been available on the market as a prescription drug for allergy-related conditions in Japan since 1995 with a very good safety and tolerability profile. There is preclinical and exploratory clinical evidence suggesting that ME-015 may be effective in treating cough caused by idiopathic pulmonary fibrosis (IPF-cough). 80% of patients with idiopathic pulmonary fibrosis (IPF) are affected by a devastating dry cough that is often not responsive to standard cough treatments and causes significant psychological and physiological suffering as well as reduced quality of life. As of July 2023, there is no approved treatment for the indication of IPF-cough. There is an enormous unmet clinical need for an effective, safe and well-tolerated oral treatment. The COSMIC-IPF Phase 2 trial is the first clinical trial assessing ME-015 for the treatment of IPF-cough and aims to generate clinical proof-of-concept results regarding the safety and efficacy of ME-015 in this condition.
SGLT-2 Inhibitors in the Treatment of Ascites
Ascites HepaticCirrhosis2 moreThe goal of this observational study is to test the efficacy of glyphozines (SGLT-2 inhibitors) in the control of ascites in patients with liver cirrhosis in class A6-B9, according to the Child-Pugh classification, and type 2 diabetes mellitus. The investigators will compare patients belonging to the intervention group (A), who will be given SGLT-2 inhibitors according to diabetology indications in addition to standard medical therapy for 6, with patients of the control group (B), who will, instead, continue with the standard medical therapy for 6 months. Standard medical therapy will include dietary sodium restriction, treatment with diuretics (furosemide and spironolactone), hypoglycemic therapy (metformin, insulin, or both) and other supportive care. The main questions aims of this study are: Compare the efficacy and safety of a therapeutic approach based on the administration of SGLT-2 inhibitors in addition to optimal medical therapy (MRA and loop diuretic) compared to traditional diuretic therapy only, in cirrhotic patients with saline retention and diabetes. Demonstrate better control of the glycemic profile in cirrhotic diabetic patients using SGLT-2 inhibitors.
Open-Label Dose-Escalation Treatment Study of Patients With IPF
Idiopathic Pulmonary FibrosisIdiopathic Pulmonary Fibrosis (IPF) is a chronic progressive fibrotic lung disease resulting in increasing shortness of breath, cough, and low oxygen levels as a result of lung tissue scarring . The goal of this open-label (no placebo) study is to evaluate the safety and tolerability of artesunate at three different doses in patients with IPF. The secondary goals are to explore the blood biomarkers present in IPF patients at the beginning of the study and to study how those biomarkers change following treatment with artesunate. Participants will have 7 visits to the study site over 20 weeks which will include physician exams, vital signs, questionnaires, research and safety blood samples, and taking artesunate capsules by mouth for 12 weeks. Artesunate is used world-wide for the treatment of severe malaria but has also been found to block specific proteins that cause lung scarring and may provide an additional treatment to slow the fibrotic process in the lung and improve survival and quality of life for patients with IPF.
Atorvastatin Use and Portal Hypertension in Patients With Hepatitis B Virus-related Liver Cirrhosis:...
Hepatitis BPortal HypertensionTo evaluate whether atorvastatin can improve portal hypertension in patients with chronic hepatitis B related compensated cirrhosis with portal hypertension
The Visceral Adiposity Measurement and Observation Study
HIVHIV-Infections17 moreThe Visceral Adiposity Measurement and Observation Study