
Alterations in Coagulation Factor Levels in Patients With End Stage Liver Disease
Liver CirrhosisHemophilia5 moreThe goal of this observational study is to learn about the changes in coagulation factor VIII and IX levels in patients undergoing liver transplantation to help guide future management of coagulation factor replacement in patients with hemophilia and liver disease. The question we aim to answer is: should the recommendations for factor replacement in patients with hereditary bleeding disorders be altered in the setting of end stage liver cirrhosis? Participants will be asked to provide two blood samples, one at the beginning of their liver transplant, and one after their liver transplant.

Impact of Highly Effective Modulator Therapy on the Cystic Fibrosis Microbiome
Cystic FibrosisThe goal of this observational study is to learn about the effects of a specific cystic fibrosis therapy (Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy) on chronic sinonasal disease. The main questions it aims to answer are: How does this therapy impact bacterial communities in the paranasal sinuses? How does this therapy impact inflammation in the paranasal sinuses and olfactory cleft? How does this therapy impact sense of smell and sinonasal disease burden in individuals with cystic fibrosis? How does this therapy impact disease-specific and general quality of life of individuals with cystic fibrosis? Participants will be asked to provide samples from their nose, complete testing of their sense of smell, and complete surveys about their quality of life and sense of smell in this study. Researchers will compare study results between patients who are currently undergoing Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy and patients who are not currently undergoing therapy.

A Study to Evaluate the Efficacy and Safety of Recombinant Human Pentraxin-2 (rhPTX-2; PRM-151)...
Idiopathic Pulmonary FibrosisThis phase III study will evaluate the efficacy, safety and pharmacokinetics (PK) of recombinant human pentraxin-2 (rhPTX-2; PRM-151) zinpentraxin alfa, compared with placebo in participants with idiopathic pulmonary fibrosis (IPF).

A Pilot Study of a Low Glycemic Load Diet in Adults With Cystic Fibrosis
Cystic FibrosisCystic Fibrosis-related Diabetes1 moreThis pilot study will evaluate the safety and tolerability of a low glycemic load dietary intervention in adult patients with cystic fibrosis (CF) in a rigorous feeding study. Specific emphasis will be placed on changes in weight, body composition, and glycemic measures obtained via continuous glucose monitor (CGM) usage.

Composition for Treating Cirrhosis and Liver Cancer (SB-1121)
Cirrhosis of the LiverThe composition treats cirrhosis and hepatocellular carcinoma (Hcc) at an early stage. The product is extracted from herbs, including Adenosma glutinosum extract, Eclipta prostrata extract, Phyllanthus urinaria extract, Impatiens balsamina extract, Ascorbic acid, pyridoxine 5-phosphate, L-Arginine hydrochloride, Pregnenolone acetate. These components have participated in repairing and regenerating new liver parenchyma, preventing fibrosis cell generation, preventing liver cancer cell growth at an early stage. The composition supplements precursors which help the body strengthen antibodies and reduce the risk of inflammation, restore physiological and biochemical functions of liver cells after chronic inflammation.

Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis...
Cystic FibrosisThis study will evaluate the efficacy, pharmacodynamics (PD) and safety of ELX/TEZ/IVA in participants 6 years of age and older with a non-F508del ELX/TEZ/IVA-responsive cystic fibrosis transmembrane conductance regulator gene (CFTR) mutation.

Empagliflozin in Diuretic Refractory Ascites
Liver CirrhosisCirrhosis3 moreAscites is the most frequent complication of liver cirrhosis and results in increased morbidity and mortality but current medical management options are limited. Here, the investigators will conduct an interventional single-arm pilot clinical trial toevaluate the feasibility of empagliflozin in managing diuretic-resistant ascites in patients with decompensated cirrhosis. This single site, open label pilot study will enroll participants with decompensated cirrhosis at a single site. Participants will receive empagliflozin 10mg oral tablets once daily for 12 weeks with monitoring for safety and adverse events.

A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Participants With Cystic Fibrosis...
Cystic FibrosisThis study was evaluate the effect of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) on glucose tolerance in CF participants, 12 years of age and older who are heterozygous for the F508del mutation and a minimal function mutation (F/MF genotypes), with abnormal glucose metabolism.

Ph 1/2 Study Evaluating Safety and Tolerability of Inhaled AP-PA02 in Subjects With Chronic Pseudomonas...
Cystic FibrosisPseudomonas Aeruginosa3 morePhase 1b/2a, double-blind, randomized, placebo-controlled, single and multiple ascending dose study to evaluate the safety, tolerability and phage recovery profile of AP-PA02 multi-bacteriophage therapeutic candidate administered by inhalation in subjects with cystic fibrosis and chronic pulmonary Pseudomonas aeruginosa (PA) infection.

A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through...
Cystic FibrosisThis study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function (MF) mutation (F/MF genotypes).