A Multi-center, Single-Dose and Repeat-Dose Over Eight Weeks, Sequential Cohort Study to Evaluate Safety and Tolerability as well as Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% Administered Subcutaneously in Subjects with Alpha1-Antitrypsin Deficiency

Date Added
October 26th, 2021
PRO Number
Pro00110691
Researcher
Charlie Strange

List of Studies


Keywords
Lung, Pulmonary, Rare Diseases
Summary

This study is designed to evaluate a new therapy formulation for Alpha-1 Antitrypsin Deficiency (AATD). AATD is an inherited condition in which a person has low blood levels of a protein known as alpha-1 protease inhibitor (called Alpha1-PI). AATD causes an increased risk of chronic obstructive pulmonary disease (COPD) in the form of emphysema (long term lung disease) and, less frequently, other diseases.

This study is being conducted to evaluate the safety and tolerability of 2 different doses of Alpha-1 drugs (Alpha-1 15% and Liquid Alpha1-PI) in participants with AATD. Participants will be placed into one of two groups. Each group will receive both drugs at different points in the treatment period and because this is an "open label", study participants and the study staff know which dose of study drug participants receive. The study will last up to 486 days (16 months). Many visits are able to be conducted through home health care, lessening the need to come into the clinic.

Alpha-1 15% is an investigational product, meaning it is not approved by the U.S. Food and Drug Administration (FDA). The other drug in this study is Liquid Alpha1-PI (licensed as Prolastin®-C Liquid) and is an FDA approved treatment for adults with emphysema due to AATD. However, it is only approved for the recommended dose of 60 mg/kg. This study includes both the FDA approved 60mg/kg of Liquid Alpha1-PI and an experimental dose of 120 mg/kg that is not FDA approved. Alpha-1 15% is given as an injection under the skin and Liquid Alpha1-PI is given as an infusion into the veins.

You may or may not directly benefit from participation. However, you may help advance scientific knowledge in the treatment of AATD. Currently, the only FDA approved treatment for AATD is IV infusions of Liquid Alpha1-PI. Since the drug being studied, Alpha-1 15%, is injected with a small needle under your skin, there may be a benefit to future patients by providing flexibility of treatment route options as well as stability in serum alpha1-antitrypsin levels.

Institution
MUSC
Recruitment Contact
Rachel Millan
843-792-0260
millanr@musc.edu

Adequacy for molecular analysis in EBUS-TBNA specimens vs. liquid biopsies in patients with NSCLC, aka, "The Liquid Trial"

Date Added
June 16th, 2021
PRO Number
Pro00105097
Researcher
Gerard Silvestri

List of Studies


Keywords
Cancer, Cancer/Lung, Lung
Summary

This study will be looking at biopsies taken from mediastinal lymph nodes and a blood sample from patients who have known or suspected metastatic lung cancer. These two samples will be compared using next-gen sequencing to explore if either sampling method offers better results.

Institution
MUSC
Recruitment Contact
Michael Balassone
843-792-6696
Balassom@musc.edu

A Multicenter, Randomized Controlled Trial, Prospectively Evaluating the Clinical Utility of the Nodify XL2 Proteomic Classifier in Incidentally Discovered Low to Moderate Risk Lung Nodules

Date Added
November 5th, 2020
PRO Number
Pro00098515
Researcher
Nichole Tanner

List of Studies


Keywords
Cancer, Cancer/Lung, Lung
Summary

The purpose of this research is to evaluate if including the investigational Nodify XL2 test results in the decision-making process when planning the management of lung nodules will reduce the number of unnecessary surgical and biopsy procedures. The general study procedures include obtaining blood samples for Nodify XL2 tests, collecting relevant medical information (including test results ordered by your doctor in the management of your lung nodule), and documenting your doctor's recommendations for the treatment of your lung nodule. Participation in this research study may last up to 18 months.

Institution
MUSC
Recruitment Contact
Michael Balassone
843-792-6696
balassom@musc.edu

Personalized Lung Cancer Screening (PLuS) Study

Date Added
May 14th, 2020
PRO Number
Pro00098558
Researcher
Gerard Silvestri

List of Studies


Keywords
Cancer, Lung
Summary

This is an observational study that will analyze electronic health records data from real-world populations and settings in order to fully characterize the outcomes of Lung Cancer Screening with Low-Dowse CT scans.

Institution
MUSC
Recruitment Contact
Michael Balassone
843-792-6696
Balassom@musc.edu

LUNGMAP: A Master Protocol To Evaluate Biomarker-Driven Therapies And Immunotherapies In Previously-Treated Non-Small Cell Lung Cancer (Lung-Map Screening Study)

Date Added
May 7th, 2020
PRO Number
Pro00099591
Researcher
John Wrangle

List of Studies


Keywords
Cancer, Cancer/Lung, Lung
Summary

This study is for patients that have been diagnosed with non-small cell lung cancer. The purpose of this study is to find out if a targeted immunotherapy treatment will have an effect on specific genes and proteins in tumors. This is a screening study in which a tumor sample will be submitted for testing. Based on results, the participant will be assigned to one of the treatment sub-studies.

Institution
MUSC
Recruitment Contact
HCC Clinical Trials Office
843-792-9321
hcc-clinical-trials@musc.edu

A Sham Controlled Prospective Randomized Clinical Trial of the RejuvenAir System for the Treatment of Moderate to Severe Chronic Obstructive Pulmonary Disease with Chronic Bronchitis (SPRAY-CB)

Date Added
April 28th, 2020
PRO Number
Pro00090634
Researcher
Charlie Strange

List of Studies


Keywords
Breathing, Lung, Pulmonary, Shortness of Breath
Summary

This research study will be evaluating whether liquid nitrogen sprayed on the cells lining the airways (cryotherapy) can reduce the mucus produced in the lungs of patients with chronic obstructive pulmonary disease (COPD). The study involves bronchoscopies, (placement of a lighted, flexible scope into the lungs) under general anesthesia to deliver the treatment to the lungs. The study is randomized such that 2/3 of individuals get the cryotherapy and the other 1/3 get a sham (control) treatment with no cryotherapy. Participants in the sham control group will be evaluated for eligible for cryotherapy at end of year one. The Study duration is 36 months. Those initially randomized to the treatment group, they will have 7 clinic visits and 2 treatment visits. Participants randomized to sham group will have 11 clinic visits and 4 treatment visits.

Institution
MUSC
Recruitment Contact
M. Gwen Blanton
843-792-8438
blantonm@musc.edu

Investigation of the Efficacy of Antimycobacterial Therapy on Pulmonary Sarcoidosis Phase II Randomized, Double-blind, Placebo-controlled Trial

Date Added
June 22nd, 2017
PRO Number
Pro00067313
Researcher
Walter James

List of Studies


Keywords
Lung, Pulmonary, Sarcoidosis
Summary

You are being asked to take part in this research study because you have been diagnosed with pulmonary sarcoidosis. Sarcoidosis is a disease that can affect the lungs, skin and other organs of the body. Sarcoidosis also involves immune cells which fight bacteria. The purpose of this study is to see if using specific antibiotics will help these immune fighting cells get rid of bacterial proteins and how the antibiotics affect respiratory (breathing) function. The antibiotics used in this study are Levaquin, Ethambutol, Azithromycin, and either Rifampin or Rifabutin. You will by chance be assigned either these medicines or a placebo (an inactive substance).

Institution
MUSC
Recruitment Contact
Kelly French
843-792-3169
frenchke@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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