A 5-year Longitudinal Observational Study of the Natural History and Management of Patients with Hepatocellular Carcinoma Save

Date Added
February 28th, 2017
PRO Number
Pro00062918
Researcher
David Koch

Silhouette
Keywords
Cancer, Cancer/Gastrointestinal, Cancer/Other, Digestive System, Genetics, Liver, Obesity, Weight Control
Summary

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.

Institution
MUSC
Recruitment Contact
Francis Beylotte
843-876-4273
beylott@musc.edu

A 5-year Longitudinal Observational Study of Patients with Nonalcoholic Fatty Liver or Nonalcoholic Steatohepatitis Save

Date Added
February 28th, 2017
PRO Number
Pro00062843
Researcher
David Koch

Silhouette
Keywords
Disease Prevention, Genetics, Liver, Obesity, Weight Control
Summary

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.

Institution
MUSC
Recruitment Contact
Francis Beylotte
843-876-4273
beylott@musc.edu

Phase 1/2 Study of Intravenous or Intrapleural Administration of a Serotype rh.10 Replication Deficient Adeno-associated Virus Gene Transfer Vector Expressing the Human Alpha-1 Antitrypsin cDNA to Individuals with Alpha-1 Antitrypsin Deficiency Save

Date Added
October 25th, 2016
PRO Number
Pro00059083
Researcher
Charlie Strange

Silhouette
Keywords
Genetics, Lung, Pulmonary, Rare Diseases
Summary

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.

Institution
MUSC
Recruitment Contact
Danielle Woodford
843-792-6280
woodfordd@musc.edu

Alcohol Research Center. Shared clinical assessment core for the Alcohol Research Center clinical projects. Save

Date Added
December 1st, 2015
PRO Number
Pro00050179
Researcher
Konstantin Voronin

Silhouette
Keywords
Alcohol, Drug Studies, Genetics
Summary

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.

Institution
MUSC
Recruitment Contact
Mark Ghent
843-792-1222
ghent@musc.edu

Enroll-HD: A Prospective Study in a Global Huntington's Disease Cohort Save

Date Added
September 10th, 2015
PRO Number
Pro00048038
Researcher
Miroslav Cuturic
Keywords
Genetics, Movement Disorders
Summary

The primary objective of Enroll-HD is to develop a comprehensive repository of prospective and systematically collected clinical research data (demography, clinical features, family history, genetic characteristics) and biological specimens (blood) from individuals with manifest HD, unaffected individuals known to carry the HD mutation or at risk of carrying the HD mutation, and control research participants (e.g., spouses, siblings or offspring of HD mutation carriers known not to carry the HD mutation). Enroll-HD is conceived as a broad-based and long-term project to maximize the efficiencies of non-clinical research and participation in clinical research while ensuring privacy and protections for consenting research participants.

Institution
USC
Recruitment Contact
Alyson Grant
803-545-6104
alyson.grant@uscmed.sc.edu

Alcohol Use and Neural Connectivity Save

Date Added
August 4th, 2015
PRO Number
Pro00046264
Researcher
Joseph Schacht

Silhouette
Keywords
Alcohol, Brain, Genetics
Summary

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.

Institution
MUSC
Recruitment Contact
Lindsay Meredith
843-792-1017
meredith@musc.edu

Analysis of genetic variant and treatment based variations in infants at risk for retinopathy of prematurity (ROP) Save

Date Added
July 31st, 2015
PRO Number
Pro00041164
Researcher
Lakshmi Katikaneni

Silhouette
Keywords
Children's Health, Genetics, Infant, Vision/ Eye
Summary

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.

Institution
MUSC
Recruitment Contact
Kinsey Shirer
843-792-2799
evanssa@musc.edu

LAM Pilot study with imatinib mesylate Save

Date Added
July 21st, 2015
PRO Number
Pro00044389
Researcher
Charlie Strange

Silhouette
Keywords
Genetics, Lung, Pulmonary, Rare Diseases
Summary

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.

Institution
MUSC
Recruitment Contact
Kimberly Brown
843-792-6474
Brownkl@musc.edu

MUSC Alpha-1 Biorepository Save

Date Added
December 16th, 2014
PRO Number
Pro00039387
Researcher
Charlie Strange

Silhouette
Keywords
Genetics, Liver, Lung, Pulmonary
Summary

This study is recruiting participants who are affected with Alpha-1 Antitrypsin Deficiency (Alpha-1) or are carriers of abnormal Alpha-1 Antitrypsin genes. Participants will be asked to provide a sample of blood that will be stored in a biorepository at the Medical University of South Carolina. Participants will be asked to complete surveys including demographic information and information about medical history and pulmonary disease. Survey information and samples will be linked and stored for use in future research related to Alpha-1 and genomics. Researchers with Alpha-1 related interests may request to use deidentified samples and data from the biorepository. The identity of donors is protected. Participants will not receive individual results; however, the availability of samples from people with rare genetic disease such as Alpha-1 is essential for researchers to better understand disease and risks, recommend risk reduction strategies and develop new treatments.

Institution
MUSC
Recruitment Contact
Kimberly Brown
843-792-6474
BrownKL@musc.edu

Alpha-1 Foundation Clinical Resource Center (CRC) Research Registry Save

Date Added
November 18th, 2014
PRO Number
Pro00026071
Researcher
Charlie Strange

Silhouette
Keywords
Asthma, Breathing, Environmental Factors, Genetics, Liver, Lung, Pulmonary, Rare Diseases, Shortness of Breath
Summary

The purpose of the Alpha-1 Foundation Clinical Resource Center (CRC) Research Registry is to collect and store medical information from individuals with alpha-1 antitrypsin deficiency (AATD or Alpha-1) or individuals that carry a deficient Alpha-1 gene. The Registry will collect medical information on your disease and diagnosis. This information will include family history, lung and liver symptoms, and exposure to cigarette smoke, dusts and fumes.The goal of this project is to obtain and share information that defines the natural history of alpha-1 antitrypsin deficiency. Biological samples from either blood or tissue may be collected and stored as part of this research. This project will assemble a library of these biological samples, some of which will be saved at MUSC.

Institution
MUSC
Recruitment Contact
Eryn Varano
877-886-2383
alphaone@musc.edu

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