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.
This is a 5-year, longitudinal, observational study of patients with NAFL or NASH designed to specifically address important clinical questions that remain incompletely answered from registration trials. In addition to the study database, a bio specimen repository will also be included so that translational studies of genomics and biomarkers of response may be performed.
Alpha-1 antitrypsin (Alpha-1, AAT) deficiency is an inherited disease which results from a defect in the alpha-1 gene. Severe AAT deficiency causes emphysema predominant chronic obstructive pulmonary disease (COPD). This is a first in man study of gene therapy to insert a normal Alpha-1 gene into the cells of the body and attempt to make a normal Alpha-1 antitrypsin protein. The purpose of this Phase I/II study is to test the safety of a new gene therapy called AAVrh.10h ?1AT. This gene therapy uses a viral vector called Adeno-Associated Virus to insert the normal Alpha-1 gene into the cells of the body when the vector is placed into the bloodstream or pleural space.
The Southern Network on Adverse Reactions (SONAR) has just described a new adverse drug reaction termed fluoroquinolone (FQ)-associated disability (FQAD). The FQs ciprofloxacin, levofloxacin, and moxifloxacin are among the most commonly prescribed oral antibiotics administered to cancer patients and have Black Box warnings for tendinitis, tendon rupture, and neurotoxicity among myasthenia gravis patients. In 2014, SONAR filed a Citizen Petition with the Food and Drug Administration (FDA) requesting Black Box Warnings for severe FQ-associated psychiatric toxicity. Our case-definition included oral FQ use and: toxic psychoses, hallucinations, paranoia, suicidal thoughts or acts, altered mental status, depressed level of consciousness, amnesia, coma, memory impairment, and/or inability to work/perform daily activities for greater than one month. A November 2015 FDA Advisory Committee meeting reviewed risks and benefits of FQs. An FDA safety reviewer described FDA?s first reports of the same toxicity, termed FQAD, noting that these events were reported since 2010. It is imperative that if there is a genetic rationale for FQAD, it be identified. This study aims to find out association between fluoroquinolone exposure and genomic risk factors which leads to long-term disability.
Research study looking for participants who have recently been hospitalized for a myocardial infarction (heart attack) or ischemia (decreased blood flow to the heart) and are identified as having a genetic variant by a simple blood test. This study is being carried out to see if a new investigational drug called dalcetrapib provides cardiovascular benefits and decreases the risks of future cardiac events when taken by patients with the the genetic variant found in 20% of people.
This research protocol will be using for initial screening and assessment potential study subjects for their farther participation in the other specific research protocols/components. These research components are part of larger research protocol - Alcohol Center Grant.
This study uses magnetic resonance imaging (MRI) to compare differences in brain connectivity between people with different levels of alcohol use. The study requires two visits: a clinical interview and assessment, and a 1-hour MRI scan. Subjects must be between the ages of 21 and 35. Compensation is available for eligible subjects.
Infants born early who are in the neonatal intensive care unit will be included if they meet national guidelines for retinopathy of prematurity (ROP) screening exams. Informed consent will be given to the parent(s) or legal guardians. 1.5-2 ml of blood will be drawn from a vein when the child is enrolled in the study and may be drawn again if the child requires treatment of eye disease. A cheek swab will also be obtained. These biologic samples will be shipped overnight to the University of Utah for genetic analysis. Analysis will determine if a change in gene expression causes retinopathy of prematurity. Infants enrolled in the study will be followed clinically per established ROP screening guidelines. They will not require additional study exams.
The purpose of this medical research study is to evaluate the safety and effectiveness of a new medication called imatinib mesylate in the treatment of Lymphangioleiomyomatosis (LAM). LAM is a rare disease in which abnormal cells (called LAM cells) grow out of control. Over time, LAM cells destroy healthy lung tissue and cause respiratory disease or failure.
Many patients with LAM are currently treated with a medication called sirolimus (rapamycin). Sirolimus slows the growth of LAM cells.
Imatinib mesylate (hereafter called imatinib) is approved by the Food and Drug Administration (FDA) for the treatment of some cancers that share common pathways with LAM cells. Laboratory studies suggest that imatinib could completely block the growth of LAM cells through initiation of targeted cell death.
An important purpose of this research is to determine the safety of imatinib in people with LAM. This study will also evaluate the short-term effectiveness of imatinib. Participants will be randomized to receiving imatinib (study medication) or placebo (no treatment) for the 56 day duration of participation. The study is being conducted at the Medical University of South Carolina and at Columbia Univerity in New York. Each site will enroll 10 participants.
Schizophrenia is a highly heritable neurodevelopmental disorder, accompanied by impairment in a variety of day-to-day cognitive functions that are associated with exposure to certain herpesviruses. This project investigates the role of a major gene of the immune system in the etilogy of herpesvirus-spurred cognitive impairment. Results from these investigations could help devise novel immunotherapeutic interventions in this devastating disorder.