
Assessment of a PERT-free Nutritional Therapeutic Delivery System for Cystic Fibrosis
Cystic FibrosisExocrine Pancreatic InsufficiencyThis is a single center, randomized, double-blind, cross-over trial (with a follow-on single-blind safety evaluation stage), assessing a ready-to-drink nutritional supplement used without PERT ("PERT-free"), nutritional supplement for blood lipid levels, safety and tolerability compared to a standard of care nutritional supplement used concomitantly with PERT.

A Study to Evaluate ELX/TEZ/IVA on Cough and Physical Activity in Participants With Cystic Fibrosis...
Cystic FibrosisThis study will evaluate the effects of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on cough and physical activity using wearable technology in CF participants.

Safety of Anticoagulant Therapy After Endoscopic Treatment
CirrhosisPortal Vein Thrombosis2 moreThe investigators aimed to verify the efficacy and safety of nadroparin calcium warfarin sequential (NWS) anticoagulation therapy after endoscopic therapy in PVT patients with cirrhosis and AVB.

Cystic Fibrosis and Urinary Incontinence
Urinary IncontinenceCystic FibrosisEvaluate the feasibility of a randomized controlled trial of non-surgical urinary incontinence (UI) management options for women with CF. The investigators will complete a pilot, feasibility study (n=30) to compare tolerability and symptom relief in women with CF and UI. Subjects will be recruited from the University of Pittsburgh Cystic Fibrosis Center after demonstrating bother from UI on initial phone script. Participants will undergo UI questionnaires and undergo a pelvic examination, non-invasive bladder scan ultrasound and a provocative stress test and then be randomized to either a disposable urethral support device (Impressa®), an absorbent product (Speax Reusable Underwear), or Pelvic floor muscle therapy. The primary outcome will be to determine the feasibility and tolerability of these options. Hypothesis: All three non-surgical UI management options for women are feasible (as measured by 80% adherence to treatment assignment over 7 days) and tolerable (as measured by patient report via questionnaire). The results from the proposed aims will provide important information about the experiences and symptom burden of women with CF and UI. Importantly, the investigators will also be able to answer the important questions of "Can it work?" and "Does it work?" as the investigators seek to construct the definitive, adequately powered trial of these therapies in women with CF and UI.

Thromboelastography Guided Blood Product Transfusion for Upper Gastrointestinal Bleeding in Cirrhosis...
CirrhosisLiver2 moreThe goal of this clinical trial is to compare resuscitation strategies in patients with cirrhosis and gastrointestinal bleeding. The main question it aims to answer is whether thromboelastography guided resuscitation decreased the amount of fresh frozen plasma patients receive. Patients will receive blood products guided by thromboelastography in the intervention group. Researchers will compare the patients who undergo thromboelastography guided resuscitation to those who receive usual care to see which strategy leads to the use of less blood products, specifically less fresh frozen plasma.

To Assess the Efficacy of Midodrine in Prevention of Cirrhosis Related Complications in Children...
CirrhosisLiverChildren with cirrhosis awaiting transplantation are more proned 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 [1]. The development of complications in these children may result in death or may preclude them from reaching upto liver transplantation [2]. 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[2-4]. One group will receive only standard medical therapy and other group will receive midodrine and standard medical therapy for 6 months. Mean arterial pressure will be monitored at every OPD visit. At the end of 12 weeks, and 24 weeks, plasma renin activity, incidence of complications related to cirrhosis like new onset ascites, increase in grade of ascites, hyponatremia, acute kidney injury and spontaneous bacterial peritonitis will be assessed. Also the transplant free survival and need for albumin transfusion will be compared between the two groups. In case of liver transplantation or death before 6 months, midodrine will be stopped

First in Human Study in Healthy Volunteers Followed With Dosing in Participants With Lung or Liver...
FibrosisFirst in Human single ascending dose followed by multiple ascending doses in healthy volunteers.

Management of Decompensated HCV Cirrhotic Patients
Decompensated Cirrhosis-The limited treatment varieties of decompensated cirrhosis due to hepatitis C virus (HCV) remain a challenge. In patients with reduced hepatic reserve, DAAs may be associated with complications as worsening decompensation. The impact of DAAs therapy on mortality in decompensated cirrhosis was not investigated.

JUNIPER: A Phase 2 Study to Evaluate the Safety, Biological Activity, and PK of ND-L02-s0201 in...
Idiopathic Pulmonary FibrosisA phase 2, randomized, double-blind, placebo-controlled, multicenter study to evaluate the safety, tolerability, biological activity, and pharmacokinetics (PK) of ND-L02-s0201 for Injection in subjects with IPF.

A Study Looking at the Safety, Tolerability and Efficacy of the Combination of the Study Drugs GLPG2451...
Cystic FibrosisThis is a Phase Ib, multi-center, open-label, nonrandomized multiple cohorts study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple doses of a combination treatment of GLPG2451 and GLPG2222, with and without GLPG2737, in adult subjects with Cystic Fibrosis.