
Efficacy of Simeox Airway Clearance Therapy in Children With Cystic Fibrosis
Cystic Fibrosis in ChildrenAirway Clearance ImpairmentChest physiotherapy plays a crucial role in treatment of lung disease in cystic fibrosis (CF). New airway clearance techniques (ACTs) adapted to individual needs are still being sought to achieve the best effect of airway clearance. The primary aim of this study is to assess the efficacy of a new ACT (Simeox) on pulmonary function in children with CF. 40 CF patients with stable respiratory function will be randomized 1:1 to Simeox or conventional chest physiotherapy (CCPT) therapy (control group) and treated at home during 1 month. After a short washout period, patients will be treated at home onto the alternative treatment for 1 month (crossover design). Lung function, quality of life, pulmonary exacerbation and safety will be evaluated at 1 month for each therapy period.

Study of Aldafermin (NGM282) in Subjects With Compensated Cirrhosis (ALPINE 4)
Compensated CirrhosisNonalcoholic SteatohepatitisA multi-center evaluation of aldafermin in a randomized, double-blind, placebo-controlled study in subjects with compensated cirrhosis.

Cystic Fibrosis and Gut Dysmotility: The Effect of Polyethylene Glycol (PEG) on Intestinal Transit...
Cystic Fibrosis Gastrointestinal DiseaseThe investigators will recruit 15 patients with cystic fibrosis 18 years of age and older who present with constipation. The investigators will assess baseline motility symptoms with a survey. Patients will then ingest a SmartPill (trademark) to obtain baseline motility within the GI lumen. All patients will undergo intervention with taking polyethylene glycol (PEG) or Miralax (brand name) 17 grams once daily. After two weeks of therapy, the patient will repeat the motility survey and again ingest a smart pill to assess the change in motility symptoms while on therapy.

Fecal Microbial Transplant for Alcohol Misuse in Cirrhosis
CirrhosisAlcohol AbuseThere is an epidemic of alcohol use disorder in the US. Alcoholism is an epidemic that spans all ages and socio-economic strata, which has a major impact on healthcare expenditure. Alcohol-associated liver disease can take the form of mild fatty liver, chronic liver disease including cirrhosis and a very acute active form known as alcoholic hepatitis. However, most patients with alcohol abuse issues with cirrhosis do not develop alcoholic hepatitis and are not willing to quit drinking. These patients are neither liver transplant candidates due to their drinking nor have any recourse to therapies directed towards the liver as is the case with alcoholic hepatitis. This is very large proportion of cirrhotic patients who do not have many therapeutic options. Prior studies have demonstrated that these patients have an altered gut-liver axis which is exacerbated by dysbiosis and a higher production of potentially toxic secondary bile acids. These secondary bile acids in turn have the potential to worsen the already impaired gut barrier in these patients, creating a vicious cycle of inflammation and further liver injury that is led by the altered microbial composition. A gut-based strategy that has the capability of "resetting" this dysbiosis could help in the amelioration of this inflammatory load and improve the prognosis of these patients.

Tackling 30-day Readmissions in Patients With Cirrhosis: The SALTYFOOD Trial
Hepatic EncephalopathyAscites2 moreThe purpose of this study is to determine the feasibility of a home meal delivery program for patients with cirrhosis and ascites and to determine the effectiveness of a salt-restricted (2 gram sodium) meal delivery program in reducing the need for therapeutic paracenteses and/or all-cause re-admissions for these patients. Many patients with cirrhosis don't have enough nutrients in the body and are frail and these meals may help them maintain a good diet and lead to improved quality of life.

An Phase 1 Study to Evaluate the Pharmacokinetic (PK) Profile of FDL169 New Formulations in Healthy...
Cystic FibrosisTwo parts, two periods, crossover study with part 2 is optional. In both parts, subjects will be randomized to sequentially receive both sublingual and oral formulations of FDL169.

A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for...
Cystic FibrosisThis study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).

Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis...
Cystic FibrosisThe trial will consist of a single treatment group enrolling adult subjects with CF on background therapy with KALYDECO®. Approximately 16 subjects will be enrolled.

AeroVanc in the Treatment of Methicillin-resistant Staphylococcus Aureus Infection in Patients With...
MRSACystic FibrosisThis is a multi-center, randomized phase III study to evaluate the clinical effectiveness of AeroVanc in persistent methicillin-resistant Staphylococcus aureus (MRSA) infection in patients with cystic fibrosis (CF).

Open-label Study to Assess the Effectiveness of Pirfenidone in Participants With Idiopathic Pulmonary...
Idiopathic Pulmonary FibrosisThis study is a national, multicenter, interventional, non-randomized, non-controlled, open-label study to assess the effectiveness of pirfenidone in participants with IPF in Russian clinical practice.