
Longitudinal Observational Study on the Course of Cystic Fibrosis Lung Disease in Patients Following...
Cystic Fibrosis Lung DiseaseThe purpose of this study is to further characterize early CF lung disease in newborns, infants and toddlers with cystic fibrosis (CF).

Cirrhosis, Effects of TRAnsplantation and Diabetes
Liver CirrhosisThis observational study is aimed at assessing insulin secretion and sensitivity by the use of a mathematical modeling from oral glucose tolerance test in subjects with liver cirrhosis undergoing orthotopic liver transplantation (OLT), before and at various time points after OLT.

New Prognostic Predictive Models of Mortality of Decompensated Cirrhotic Patients Waiting for Liver...
Decompensated CirrhosisLiver TransplantationThe MELD score is a predictive model of cirrhosis mortality used in France since 2007 to prioritize access to liver transplantation for patients enrolled in the national waiting list. The predictive value of this score was recently revised downward with a C index of the order of 0.65-0.67 and 20% of the patients enrolled for decompensated cirrhosis have access to liver transplantation by a subjective system of "expert component" independent of the MELD because of this lack of precision. The use of the MELD score to individually define access to the transplant should so be reconsidered. Recently new predictive models of cirrhosis mortality better than MELD have been developed and new mortality predictors independent of MELD have been published. The goal of this study is to design prognostic predictive models of mortality for decompensated cirrhotic patients enrolled on the national liver transplant waiting list including known (MELD, MELD Na) as more recent (CLIF-C AD, CLIF - CACLF) predictive models and new objective predictors studied in combination in order to optimize the system of allocation of hepatic allografts in France. The expected benefits of this search are twofold: At the individual level: The possibility for patients at high risk of death but with intermediate MELD score to be transplanted. Public health plan: Improving the equity of graft allocation system. Decreased mortality in the waiting list by improving the fairness and efficiency of the graft allocation system, a major public health issue

Understanding Gut Symptoms in People With Cystic Fibrosis
Cystic FibrosisGastrointestinal DysfunctionAlthough chest infections affect wellbeing and survival in cystic fibrosis (CF), most people with CF also have difficulty digesting food and must take medication for this. In spite of this treatment, two thirds of people with CF miss school or work because of tummy symptoms (pain, bloating and wind). In some cases these symptoms become severe leading to bowel obstruction and hospital admission. Long term, people with CF have a greater risk of bowel cancer. The investigators asked people with CF and health professionals to suggest the most important questions for research. Treatment of gut symptoms was in their top 10 list. Current treatments are often ineffective because the investigators do not fully understand why symptoms occur. GRAMPUS-CF SRC will describe accurately the categories of gut symptoms in CF and find out why they occur. The investigators will do this using magnetic resonance imaging (MRI) scans and tests which give a detailed description of the germs in the bowel or which measure inflammation. The investigators will also study the effects of diet, using a questionnaire. The investigators will link these results together, using advanced statistics to find the factors causing gut symptoms. The investigators will then identify treatments which are likely to be helpful. In future work the investigators will test these in clinical trials.

A Study Evaluating Atezolizumab, With or Without Bevacizumab, in Patients With Unresectable Hepatocellular...
Hepatocellular CarcinomaThe purpose of this study is to assess the safety and efficacy of atezolizumab and bevacizumab, or atezolizumab alone, as first-line treatment in participants with unresectable, locally advanced or metastatic hepatocellular carcinoma (HCC) with Child-Pugh B7 or B8 cirrhosis.

Midodrine Plus Albumin Versus Midodrine Alone to Prevent Cirrhosis Related Complications in Children...
Liver CirrhosisChildren with decompensated cirrhosis are more prone to develop various complications. The pathogenesis of cirrhotic complications (ascites, hyponatremia, acute kidney injury) includes release of vasodilatory molecules like nitric oxide, damage associated molecular pathogens (DAMPs) and pattern associated molecular pathogens (PAMPs) secondary to bacterial translocation, which causes splanchnic bed vasodilation resulting in activation of renin-angiotensin and aldosterone axis (RAAS) causing sodium and water retention and renal vasoconstriction. The development of complications in these children may result in death or may preclude them from reaching upto liver transplantation. Midodrine is an α1 adrenergic receptor agonist, which increases vascular tone causing rise in the blood pressure, thereby improving renal perfusion and causes RAAS deactivation. The effects of midodrine is documented in reduction of refractory ascites, hepatorenal syndrome and hyponatremia. Albumin is a protien that works by both increasing the colloidal oncotic pressure and improving systemic circulation as well as by effecting the body with anti-inflammatory and antioxidant properties. We have already demonstrated the safety and efficacy of midodrine as well as albumin in cirrhotic children. However, none of these drugs alone provided survival benefit to the patients. Hence, we have planned this study with the ojective to evaluate if combining these 2 drugs (midodrine and albumin) would further reduce the complications and improve the survival in decompensated cirrhotic children.

A Double-Blind, Active-Controlled, Multiple-Ascending Dose Study of Aerosolized RSP-1502 in Subjects...
Cystic Fibrosis LungRespiratory Infections3 moreThis 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.

Longitudinal Changes in Serum KL-6 in IPF
Idiopathic Pulmonary FibrosisIdiopathic pulmonary fibrosis (IPF) is a chronic lung disease with a poor prognosis. More accurate tests to predict disease progression and response to treatment are required. Krebs von den Lungen-6 (KL-6) is a blood marker associated with IPF. Results from previous studies have shown that levels of KL-6 are higher in patients with IPF compared to people without the disease. In addition, it is not clear what impact treatment has on KL-6 levels, and whether this could help us to monitor how effective treatment for IPF is. The investigators plan to perform a study in which KL-6 levels in the blood of patients with a new diagnosis of IPF are measured at baseline, 3, 6 and 12 months to look for and changes in the levels of KL-6 in the blood.

PROtocol of Metabolic and Cryptogenic livEr Disease regisTry for intEgration of Omic Studies
NAFLD - Nonalcoholic Fatty Liver DiseaseDiabetes7 moreThe main aim of the study is to set up an observational cohort with NAFLD (Non-alcoholic fatty liver disease) at different stage of disease (from simple steatosis to cirrhosis and/or HCC-Hepatocellular carcinoma) and for comparative purpose a cohort of subjects with diabetes and/or obesity and/or other risk factors (i.e. psoriasis, IBD (inflammatory bowel disease), dyslipidemia) without NAFLD in order to have a clinical phenotypical characterization and the collection of biological specimens. We will collect clinical data, biological samples and imaging results in order to perform future cross-sectional studies and/or longitudinal studies for elucidating pathways of the disease and develop and validate biomarkers for diagnosis, prognosis and monitoring liver disease and comorbidities in order to contribute to precision medicine in this field.

A Prospective Cohort Study of IRPF in China
Idiopathic Retroperitoneal FibrosisThis is a cohort study to investigate the disease course and treatment response of patients with idiopathic retroperitoneal fibrosis(IRPF). Methods: All the patients fulfilling diagnostic criteria of IRPF would be enrolled around China. A online database system will been established. Endpoints: The primary endpoint is to investigate the clinical manifestations of Chinese IRPF patients; the secondary endpoints including the demographic features,laboratory characteristics, immunological tests, imaging and pathological features, in addition, the treatment and prognosis of the disease.