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

A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects

Date Added
February 25th, 2020
PRO Number
Pro00096021
Researcher
Charlie Strange

List of Studies

Keywords
Liver, Lung, Pulmonary
Summary

Alpha-1 Antitrypsin (AAT) is a naturally occurring protein involved in the protection of lungs from inflammation. A mutation in the AAT gene (a change in the body's genetic instructions on how to make AAT) causes it to be made incorrectly and very little of it gets into the bloodstream. Severe AAT deficiency (lack of AAT in the blood) causes emphysema, which causes holes in the lungs. This study is being done to learn more about the safety, tolerability and effectiveness of VX-814 in patients with Alpha-1 Antitrypsin Deficiency. There are 3 parts to this study: Parts A1, A2, and B. Participants can only join one part of the study. Participants in all groups will be randomly assigned to the treatment drug or a placebo (sugar pill). Participants in part A1 will have an 80% chance of receiving the treatment drug and participants in Part A2 and Part B will have a 75% chance of receiving the treatment drug.

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

A Multicenter, Randomized, Sham-controlled Study to Evaluate Safety and Efficacy After Treatment with the Nuvaira™ Lung Denervation System in Subjects with Chronic Obstructive Pulmonary Disease (COPD) (AIRFLOW-3)

Date Added
August 27th, 2019
PRO Number
Pro00087312
Researcher
Charlie Strange

List of Studies

Keywords
Genetics, Lung, Pulmonary, Shortness of Breath
Summary

The primary purpose of this study is to see if the Targeted Lung Denervation (TLD) therapy (Active Treatment) is more effective than a sham procedure (Sham Control/no TLD therapy) at decreasing moderate or severe exacerbations in patients with COPD on optimal medical care. In addition, the study seeks to compare long-term safety, and other efficacy assessments, between the Active Treatment arm and the Sham Control arm.

TLD Therapy is done by passing a bronchoscope, with a special device (catheter) inserted through it, into the lungs. This special catheter delivers a type of electrical energy called radio frequency (or RF) energy to the nerves located on the outside of the airways. As with many bronchoscopic procedures, this is done while under anesthesia.

Participants will be randomly assigned (like flipping a coin) to receive one of two different treatments, either TLD Therapy in addition to optimal medical care (Active Treatment) or optimal medical care only (Sham Control). No matter which treatment you receive, you will undergo the same type of procedure, testing and follow-up while remaining on optimal medical care for COPD. You will have an equal chance of being assigned to either Active Treatment or the Sham Control group (1:1 randomization). Neither you or your study doctor will know which treatment you have received until after your 12-month follow-up visit. At the 12-month visit you will find out whether you received the active or sham procedure. If you received the sham procedure you have the option of crossing over into the treatment group and receiving TLD therapy.

Participation in the study will last for approximately 62 months. Depending on which group you are randomized to and if you decide to crossover to the treatment group, there will be 1-2 visits for TLD Therapy or sham control (non-active) procedure, 9-12 in-person clinic visits, and 13-23 phone visits.

Institution
MUSC
Recruitment Contact
Eryn Varano
843-792-1219
varanoe@musc.edu

Alvelestat (MPH996) for the Treatment of ALpha-1 ANTitrypsin Deficiency

Date Added
June 18th, 2019
PRO Number
Pro00088962
Researcher
Charlie Strange

List of Studies

Keywords
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 study is designed to test the effectiveness of an drug (Alvelestat) on lung damage caused by Alpha-1 Antitrypsin Deficiency. This is blinded study and there is a 50% chance of receiving a placebo.

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

A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters in Response to ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency (AATD)

Date Added
June 11th, 2019
PRO Number
Pro00088982
Researcher
Charlie Strange

List of Studies

Keywords
Drug Studies, Lung, Pulmonary, Rare Diseases
Summary

Alpha-1 Antitrypsin (AAT) is a naturally occurring protein involved in the protection of lungs from inflammation. A mutation in the AAT gene (a change in the body's genetic instructions on how to make AAT) causes it to be made incorrectly and very little of it gets into the bloodstream.This results
in the lung damage known as emphysema. ARO-AAT is an investigational drug, which means that it is not approved by the Food and Drug Administration. ARO-AAT works by interrupting a step in the production of AAT. In a patient with AATD, this would stop the mutated protein from being made. This study is being carried out to see how safe and well tolerated ARO-AAT is, and to see if low, medium and high doses of the study treatment will decrease Alpha-1 Antitrypsin in the blood and in the liver compared to a placebo, or dummy injection. The Study medication is given via injection on Day 1, 29 and 133 and then every 84 days. The study includes approximately 17 visits over a period of 24 month. Compensation will be provided for study site visits. .

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

A Multi-center, Randomized, 36-month, Parallelgroup, Non-inferiority, Phase III Study to Compare the Effectiveness of 500 mcg QD or alternate regimen of RofLumilast (Daliresp) Therapy Versus 250 mg QD, 500 mg QD three times per week or alternate regimen of Azithromycin Therapy to preveNt COPD Exacerbations (RELIANCE)

Date Added
July 16th, 2018
PRO Number
Pro00079290
Researcher
Charlie Strange

List of Studies

Keywords
Lung, Pulmonary, Shortness of Breath
Summary

The RELIANCE study is comparing two drugs, Roflumilast and Azithromycin, to treat COPD. Participants will be randomized to either take either Roflumilast or Azithromycin during the course of the study. Participation will last up to 3 years depending on when participants join. People who enroll at the beginning of the study will participate for approximately 3 years and people who enroll later in the study will be enrolled for a shorter amount of time. The study team will follow all participants for a minimum of 6 months.
There will be one in person visit at the beginning of the study where participants will be randomized to either take Roflumilast or Azithromycin. The remainder of the study will involve one follow-up phone call at 1 week and follow up phone calls every 3 months. The 3 month follow up phone calls will determine if there have been any hospitalizations, if the study participant is still taking their prescribed study medication and determine if contact information is still correct. The 3 month follow up questions can also be completed via an online patient portal if the patient prefers this method over receiving phone calls.

Institution
MUSC
Recruitment Contact
Eryn Varano
843-792-1219
varanoe@musc.edu

Multicenter International Durability and Safety of Sirolimus in Lymphangioleiomyomatosis (LAM)Trial (MIDAS)

Date Added
September 15th, 2016
PRO Number
Pro00059134
Researcher
Charlie Strange

List of Studies

Keywords
Lung, Pulmonary, Rare Diseases
Summary

Lymphangioleiomyomatosis (LAM) is a rare lung disease that is caused by genetic mutations. It results in the uncontrolled growth and proliferation of an atypical smooth muscle cells in the lung. These cells invade airways, blood vessels, and lymph vessels, and limit the flow of air, blood, and lymph, respectively. The source of the cells is unknown, but available evidence indicates they arise from an extrapulmonary source. Their aberrant behavior is due to mutations in tuberous sclerosis genes that results in mTOR activation. Respiratory failure, lung collapse (pneumothorax), and pleural effusions (chylothorax) are hallmarks of the disease. This study will evaluate the safety and durability of the mTOR inhibitors sirolimus and everolimus, which are FDA approved medications for prevention of rejection of transplanted organs, in stabilizing or improving lung function in people in LAM.

Institution
MUSC
Recruitment Contact
Meghan Blalock
843-792-2813
schneidm@musc.edu

A Phase 3/4 Study to Evaluate the Safety, Immunogenicity, and Effects on the Alpha1-Proteinase Inhibitor (A1PI) Levels in Epithelial Lining Fluid Following GLASSIA Therapy in A1PI-Deficient Subjects

Date Added
January 12th, 2016
PRO Number
Pro00046425
Researcher
Charlie Strange

List of Studies

Keywords
Breathing, Lung, Pulmonary, Rare Diseases, Shortness of Breath
Summary

Individuals with alpha-1 antitrypsin (AAT) deficiency (AAT blood level lower than 11 micro-moles) and emphysema will be invited to participate in this study. This study will determine the impact of IV Alpha-1 proteinase inhibitor (Alpha-1 MP) on the progression of emphysema in patients with AAT deficiency. A participant in this study would receive either GLASSIA dosed at 60mg/kg with a high particle load or GLASSIA dosed at 60mg/kg with a low particle load. Neither the study investigators nor the participants will know which batch of drug is actual given to the participant. Participants will have the IV therapies given to them weekly for 25 weeks, with some infusions given at MUSC and some at home. Safety and side effects of all therapies will be monitored.

Institution
MUSC
Recruitment Contact
M. Gwen Blanton
843-792-8438
blantonm@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 informa┬Čtion 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
Eryn Varano
843-792-1219
varanoe@musc.edu



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