This is a clinical research study of an investigational new drug that is being developed as a possible treatment in patients with hepatic insufficiency, cirrhosis and or/elevated blood ammonia levels. Ammonia is a substance that is built during the breakdown of the proteins in blood; in case of errors in the breakdown of proteins, ammonia levels might increase. Elevated ammonia may lead to other medical conditions. The study will help to determine the safety and tolerability of the study medication in male and female patients; in addition, the study investigates the study medications pharmacokinetics and pharmacodynamics.
This study is an inpatient study.
This study consists of 2 parts (part 1 and part 2). However our institution will only be taking part in part 2 of the study (described below) because recruitment for part one has been closed (as indicated by our sponsors).
Patients will initially present for a screening visit where eligibility will be determined. If patients are eligible for this study they will be admitted to an inpatient unit for a total of 12 days. During the 12 days of hospital stay, patients will receive study drug/placebo, regulated diet (predetermined as per study protocol) and several blood tests will be withdrawn measuring serum ammonia levels during this hospital stay. Once patients are discharged from hospital stay, they will be expected to follow up 1 week and 70 days after last study drug/placebo was administered (i.e last day in the hospital) with a stool sample.
When a person swallows, squeezing pressure is created to drive food and liquid down the throat to the esophagus (food tube). If a person has a swallowing impairment, meaning it is hard for him/her to swallow, he/she may need to use more squeezing pressure to drive the food or liquid down the throat to the esophagus. For this study, we want to examine the effect of making it harder to swallow (by placing a device around your neck that applies pressure to your neck) on how much squeezing pressure is needed to swallow liquids in normal people. After numbing the inside of your nose with numbing cream, we will use two instruments at the same time to measure this: 1) a small scope placed through your nose into the upper part of your throat, so that a camera can record the movements of your throat before and after swallowing and 2) a small catheter placed through your nose and fed into your stomach while you swallow, which records the squeezing pressures of the muscles in your throat and esophagus. We also want to see how much liquid remains in the throat after swallowing and how well the windpipe is protected from liquid entering it before, during, and after swallowing.
A clinical study for people with recurrent Clostridium difficile infection (CDI), which is an infection in the intestines. This infection causes severe diarrhea. Recurrent Clostridium difficile (C. diff) infection is being studied as part of a medical research study for an investigational new drug called RBX2660, an enema made of a solution of stool (poop or feces).
The purpose of this research study is to determine which dose is effective and how the body processes the study drug, vedolizumab in children ages 2-17 who have ulcerative colitis or Crohn's disease.
This phase 2 study includes a 4-week Screening Period, a 22-week
Treatment Period (with last dose at Week 14). Subjects who do not
enter the long term extension study will have an 18-week Follow-up Period starting
from their last dose of study drug and a long-term follow-up safety
survey by telephone 6 months after their last dose of study drug.
This is a long-term extension study enrolling male and female pediatric subjects with ulcerative colitis (UC) or Crohn's disease (CD) who initiated vedolizumab intravenous (IV) treatment in the phase 2 Study MLN0002-2003 between the ages of 2 and 17 years of age. The study will evaluate the long-term safety of vedolizumab administered by IV infusion in pediatric subjects with UC or CD. The study will also evaluate the effect of long-term vedolizumab IV treatment on the time to major IBD-related events (hospitalizations, surgeries, or procedures), health-related quality-of-life measurements, patterns of growth and development, and exploratory efficacy measures. Up to 80 rollover subjects from Study MLN0002-2003 will participate in this study. Subjects who weigh 30 kg or greater will receive vedolizumab 300 mg (high dose) or 150 mg (low dose) Q8W. Subjects who weight less than 30 kg will receive vedolizumab 200 mg (high dose) or 100 mg (low dose) IV. Patients will receive treatment and perform assessments every 8 weeks for up to 5 years or until the subject withdraws from the study or the sponsor decides to close the study, whichever comes first.
Non-Alcoholic fatty liver disease is the most common liver disease, and involves fat deposition in the liver. The fat in the liver can lead to inflammation, scarring, end stage liver disease and potential liver cancer. Some patients with fat in their liver do not see these changes, and our current understanding of why some people are not affected while others see progression of their disease is poor. We are currently in process of initiating studies to learn more about fatty liver disease, and having a database of patients at the VA medical center who are willing to participate in these studies and future studies would help both the patients learn about the new and upcoming therapies, and help the clinical investigators to quickly screen their patients and invite them to participate in their studies.
TARGET-HCC is a 5-year, longitudinal, observational study of the natural history and management of patients with HCC. The study will address important clinical questions that remain unanswered in the management of HCC with a unique research registry of participants with HCC from academic and community real-world practices. TARGET-HCC is disease focused, not drug specific, which allows for continuous acquisition of real-world evidence regarding the natural history, management, and outcomes of treatment with current therapies and new treatments that may be utilized in usual clinical practice.
The purpose of this research study is to learn more about the outcomes of total pancreatectomy with islet autotransplantation (TPIAT). Total pancreatectomy is the removal of the pancreas and islet autotransplantation is the placement of the insulin producing cells back into you to prevent diabetes. This study is looking to enroll patients who are scheduled to have a TPIAT surgery to treat pancreatitis (inflammation and scarring of the pancreas).
In addition to the routine care for pancreatitis and TPIAT surgery, participation in this study will involve completion of some brief surveys about the subject's health before TPIAT, at 6 months after TPIAT, and each year after the TPIAT surgery for 4 years, as well as a lab test conducted at each of the follow-up visits.
A Crohn's Disease Registry for pediatric patients (between the ages of 6 and 17 years) with moderately to severely active Crohn's Disease (CD), who are already taking Humira® (Adalimumab) to treat their condition. Data from the participant's medical record will be collected to study the long-term safety and effectiveness of Humira®. The Registry expects to collect data for 10 years. No study drug will be given.
Treatment of chronic hepatitis C virus (HCV) infection is now possible with all oral medications. While most patients achieve a sustained virologic response (SVR) after treatment, synonymous with cure, some patients relapse after treatment for reasons that are unclear. The goal of this research is to understand how a person's immune system changes during treatment of HCV infection with all oral therapy, and how these changes might impact the chances of relapse after treatment. To address these questions, blood and clinical information will be collected from study participants over the course of receiving standard of care treatment for HCV infection. This blood and clinical information will be used to conduct laboratory research focused on the immune system.