
Inspiratory Muscle Training in Individuals With Cystic Fibrosis
Cystic FibrosisCystic fibrosis is a genetic disease that affects some organs of the human body. Among them, the lungs tend to be the most affected due to the accumulation of mucus in the airways, which in addition to avoiding the passage of air, favors pulmonary infections. With the evolution of the condition, secondary complications arise, such as postural changes, decreased respiratory muscle strength, decreased functional capacity and, consequently, quality of life. Therefore, respiratory muscle training may be an intervention that improves the respiratory condition of these individuals, allowing an improvement in the quality of life and may delay the evolution of respiratory symptoms. Thus, this study aims to investigate a home protocol of respiratory muscle training on respiratory muscle strength, lung function, quality of life, posture and functional capacity in adolescents and adults with cystic fibrosis. The researchers believe that the training can cause an improvement in the studied variables, and can be inserted in the usual treatment of these patients.

Longitudinal Assessment of Transient Elastography in Cystic Fibrosis
Cystic Fibrosis Liver DiseaseTo determine if transient elastography (TE), when combined with ultrasound (US) pattern characterization can improve the prediction of progression to a nodular pattern on US. To confirm the feasibility of obtaining TE measurements in children with Cystic Fibrosis (CF) To prospectively assess whether TE data are associated with conventional laboratory markers of hepatic fibrosis To determine the variability of TE measurements taken at different sites in the same patient

SynRinse Irrigation Pilot (SIP) Trial
SinusitisChronic1 moreAssess if the use of SYNRINSE can improve short-term subjective and objective outcome measures after one week in patients with active Chronic Rhinosinusitis (CRS) who have had prior sinus surgery.

PTC Study to Evaluate Ataluren in Combination With Ivacaftor
Cystic FibrosisThe purpose of this study is to explore the combination of ataluren and ivacaftor as a treatment for patients with nonsense mutation cystic fibrosis

Feasibility of Outpatient Closed Loop Control With the iLet Bionic Pancreas in Cystic Fibrosis Related...
Cystic Fibrosis-related DiabetesThe current study is designed to test the feasibility of the a wearable bionic pancreas system that automatically delivers insulin and glucagon can provide superior regulation of glycemia versus usual care for adults and children with cystic fibrosis related diabetes.

Extracellular RNA Markers of Liver Disease and Cancer
Hepatocellular CarcinomaBiliary Tract Cancer2 moreThe study will examine and evaluate the use of extracellular RNA in blood as markers for the diagnosis of liver disease or cancer, and as markers for prediction of response to treatment or recurrence of cancer after surgery

Emricasan, an Oral Caspase Inhibitor, in Subjects With NASH Cirrhosis and Severe Portal Hypertension...
CirrhosisPortal Hypertension1 moreThis is a multicenter, randomized, double-blind, placebo-controlled trial involving subjects with NASH cirrhosis and severe portal hypertension (defined as HVPG ≥12 mmHg as determined by the central reader assigned to this study). Upon successful screening, subjects will be randomized to receive either emricasan 50 mg BID, 25 mg BID, or 5 mg BID or matching placebo BID.

Prognostic Value of Myocardial Fibrosis in Severe Aortic Valve Stenosis
Aortic StenosisCalcificDegenerative aortic valve stenosis (AS) is the most common valve heart disease in the developed Western countries. The hemodynamic progression of AS occurs over time and leads to LV hypertrophy (LVH) as a compensation mechanism of the heart. Morphological changes such as increasing muscle fibre thickness, collagen volume, and interstitial fibrosis occur in AS patients. These changes result in left ventricular (LV) diastolic and systolic dysfunction and, consequently, to with AS related symptoms. When symptoms associated with AS appear, patients' prognosis is poor if surgical aortic valve replacement (SAVR) or a trans-catheter aortic valve implantation (TAVI) is not performed. Primary hypothesis of the research: fibrotic changes in the myocardium are related to immediate (in hospital) or long-term complications (MACE and all-cause mortality) in patients with severe AS. The goal of the study is to determine the prognostic implications of focal as well as diffuse myocardial fibrosis in patients with severe aortic valve stenosis.

Non Invasive Ventilation During Physical Training in Cystic Fibrosis
Cystic FibrosisPatients with cystic fibrosis older than 18 years will performance a Bruce test with non invasive ventilation (once) after 30 minutes they perform the same test with oxygen supplementation. Measurements: Transcutaneous level off carbon dioxide, saturation, puls and dyspnea scale.

HERO-2: Home-Reported Outcomes With CFTR Modulator Therapy
Cystic FibrosisThis is an observational cohort study, using data from Folia Health and the Cystic Fibrosis Foundation Patient Registry (CFFPR). Individuals taking elexacaftor/tezacaftor/ivacaftor (ETI) may be enrolled through the Folia application. During the 12-month study period, participants will be asked to track their routine treatment and medication usage, daily symptoms, and monthly review with validated patient-reported outcome (PRO) questionnaires. Participants will also be asked to self-report instances of changes to their treatment plan, and pulmonary exacerbations. There are no study-associated site visits.