Alpha-1 Foundation Therapeutic Development Network

Date Added
October 23rd, 2023
PRO Number
Pro00132722
Researcher
Charlie Strange

List of Studies


Keywords
Liver, Lung, Non-interventional, Pulmonary, Rare Diseases
Summary

The Alpha-1 Foundation Therapeutic Development Network (TDN) aims to make it easier to design and carry out clinical trials that enhance the treatment of patients with Alpha-1 Antitrypsin Deficiency (AATD). To achieve this, the TDN will establish a network of clinical trial centers that have enough patients to gather a comprehensive database of clinical and genetic information. This data will be crucial in determining the criteria for including or excluding participants in the trials and in recruiting suitable subjects.

Specifically, this study will enroll participants by in person or remote consent who will allow collection of medical records to be entered into an Alpha-1 TDN database. Participants will then be invited to future clinical trials.

Institution
MUSC
Recruitment Contact
Kristin Neff
843-792-1219
neffk@musc.edu

Occult Gastrointestinal Bleeding Caused by Portal Hypertensive Gastropathy in Patients with Cirrhosis

Date Added
May 11th, 2023
PRO Number
Pro00121305
Researcher
Don Rockey

List of Studies


Keywords
Digestive System, Liver, Stomach
Summary

In this study, we will recruit cirrhotic patients who are undergoing endocscopic procedures as part of their standard of care. Their endoscopies will reveal whether they have portal hypertensive gastropathy. After the procedure, we will ask the patients to provide us with a stool sample, which we will assess for occult GI bleeding. For those patients who DO NOT have occult GI bleeding, they will be contacted every 6 months for 2 years to check whether they have developed GI bleeding.

Institution
MUSC
Recruitment Contact
Sara Hatoum
843 792 0650
hatoum@musc.edu

A Phase 2a, Randomized, Double-Blind, Placebo-Controlled, Multicenter, Dose-escalation, Proof-of-Concept Study Evaluating the Safety, Tolerability, Efficacy and Pharmacokinetics of INT-787 in Subjects with Severe Alcohol-Associated Hepatitis

Date Added
April 6th, 2023
PRO Number
Pro00124137
Researcher
Don Rockey

List of Studies


Keywords
Alcohol, Liver
Summary

To evaluate the efficacy of an investigational medication (INT-787) as assessed by disease progression in severe alcohol-associated hepatitis (sAH).

Institution
MUSC
Recruitment Contact
Bridgette Blankenship
843-876-8439
blanke@musc.edu

Collection of Blood to Evaluate Epigenomics and Protein Biomarkers for the Detection of Hepatocellular Carcinoma

Date Added
July 7th, 2022
PRO Number
Pro00117885
Researcher
Don Rockey

List of Studies


Keywords
Liver
Summary

Collection of Blood to Evaluate Epigenomics and Protein Biomarkers for the Detection of Hepatocellular Carcinoma

Institution
MUSC
Recruitment Contact
Bridgette Blankenship
843-876-8439
blanke@musc.edu

Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease Consortium

Date Added
March 16th, 2022
PRO Number
Pro00117423
Researcher
Charlie Strange

List of Studies


Keywords
Autoimmune disease, Liver, Lung, Non-interventional, Pulmonary, Rare Diseases
Summary

The purpose of this study is to create a de-identified, public use,
repository of data of Chronic Obstructive Pulmonary Disease (COPD)
patients with by Alpha-1 antitrypsin deficiency (AATD), a rare genetic
condition that can cause COPD and emphysema.

Institution
MUSC
Recruitment Contact
Kristin Neff
843-792-1219
neffk@musc.edu

MUSC Pulmonary Biorepository

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

List of Studies


Keywords
Genetics, Liver, Lung, Pulmonary
Summary

The purpose of the MUSC Pulmonary Biorepository is to collect and store samples linked to medical and other information from individuals with pulmonary disease as well as healthy controls.

In combination with the clinical data and other approved research studies (that may recruit for and/or utilize samples of the biorepository) this sample repository will provide for uniform, longitudinal, complete and accurate data that can be organized and clinically correlated at the time of sample donation, with longitudinal testing possible as part of future study. Samples will be linked to each participant's unique ID, though will be deidentified and coded for use in future research and subsequent publications with pulmonary disease and control patients.

Institution
MUSC
Recruitment Contact
Mary Hayden
843-792-8438
blantonm@musc.edu



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