Early Access Program (EAP) for a Triple Combination CFTR Modulator (elexacaftor-tezacaftor-ivacaftor) in Subjects With Cystic Fibrosis who are Homozygous or Heterozygous For the F508del Mutation Save

Date Added
October 8th, 2019
PRO Number
Pro00092175
Researcher
Patrick Flume

List of Studies


Profiles_link
Keywords
Cystic Fibrosis, Lung, Pulmonary
Summary

The purpose of this program is to make available the TRIPLE COMBO to patients with Cystic Fibrosis to improve their health. The program drug, elexacaftor-tezacaftor-ivacaftor, is not available to patients in the US except through a special access program filed with the FDA. Elexacaftor-tezacaftor-ivacaftor (no trade name as of yet) is a drug combination that is currently under FDA review, but the FDA has approved expanded access to the TRIPLE COMBO while it is reviewing the application for final drug approval. Those eligible for the TRIPLE COMBO must have at least one copy of the CF gene mutation F508del.

In order to participate in this program, participants must be at least 12 years of age or older (there is no upper age limit). Participation in this program may last until a decision has been made on the approval of the TRIPLE COMBO. If the drug is approved and available by prescription, a prescription will be submitted. Once that has been filled, the participant will be discontinued from this expanded access program. There may be up to 20 subjects enrolled at this site.

Institution
MUSC
Recruitment Contact
Max Lento
(843) 792-3169
lento@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) Save

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

List of Studies


Profiles_link
Keywords
Genetics, Lung, Pulmonary, Shortness of Breath
Summary

The primary purpose of conducting this study is to see if TLD (Targeted Lung Denervation) Therapy in addition to standard optimal medical care is better at reducing a moderate or severe exacerbation (also known as a COPD flare-ups or worsening of symptoms) and related hospitalizations than optimal medical care alone. 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 radiofrequency (or RF) energy to the nerves located on the outside of the airways. As with many bronchoscopic procedures, this is done while under anesthesia. TLD Therapy does cause a permanent change to a person's lungs. To test this, participating patients will be randomly assigned (in a 1:1 ratio) to receive one of two different treatments, either TLD Therapy in addition to optimal medical care or optimal medical care alone.
Some sites, including MUSC, will also be collecting 3 airway brushes to look at inflammatory biomarkers in the lungs. A biomarker is anything that can be used as an indicator of a particular disease state.
If you choose to participate in this study, it is estimated that you will be involved in this study for approximately 62 months. Participation will take around 11 clinical site visits and 9 follow up phones calls over a period of 5 years. The participant and person obtaining consent will sign the informed consent form and the participant will receive a copy before any study procedures occure.

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

Routine Cardiac Screening in Sarcoidosis Patients Save

Date Added
August 20th, 2019
PRO Number
Pro00090373
Researcher
Walter James

List of Studies

Silhouette
Keywords
Lung, Pulmonary, Sarcoidosis
Summary

This is a trial for cardiac sarcoidosis in patients seen during routine clinical care who have not been clinically suspected to have cardiac sarcoidosis according to conventional criteria. The addition of echocardiogram and ambulatory ECG to usual clinical follow-up will be compared. This is a multicenter study, coordinated by the Foundation for Sarcoidosis Research. Up to 12 sites in the US and Europe will participate. Each site will enroll 50 consecutive consenting individuals to a total of 600 enrolled subjects in a randomized trial of additional screening versus usual care.

Institution
MUSC
Recruitment Contact
Abbi Reed
(843) 792-1820
reedab@musc.edu

Registry for Advanced Sarcoidosis (ReAS) Save

Date Added
August 13th, 2019
PRO Number
Pro00090539
Researcher
Walter James

List of Studies

Silhouette
Keywords
Lung, Pulmonary, Sarcoidosis
Summary

The purpose of this research study registry is to identify patients with advanced sarcoidosis. This is an observational research study, which means you will not take study medication(s). The study will determine how many and how often patients are diagnosed with advanced disease in sarcoidosis clinics. The study will determine how many and how often patients are diagnosed with advanced disease in sarcoidosis clinics. This study will look at genetic markers; determine the risk factors for advanced sarcoidosis and the natural course of symptoms in advanced disease. You will be in the research study for approximately 36 months (3 years)and will be seen every 6 (six) months during the research study.

Institution
MUSC
Recruitment Contact
Abbi Reed
(843) 792-1820
reedab@musc.edu

A Phase 3, Randomized, Placebo-controlled, Double-blind, Adaptive Study to Evaluate the Safety and Efficacy of Inhaled Treprostinil in Patients with Pulmonary Hypertension due to Chronic Obstructive Pulmonary Disease (PH-COPD) Save

Date Added
July 23rd, 2019
PRO Number
Pro00090282
Researcher
Rahul Argula

List of Studies


Profiles_link
Keywords
Lung, Pulmonary, Pulmonary Arterial Hypertension (PAH)
Summary

The purpose of this study is to evaluate the effectiveness of inhaled treprostinil in improving exercise ability in patients with PH (Pulmonary Hypertension) -COPD (Chronic Obstructive Pulmonary Disease) when compared to an inactive solution, or placebo. This is a 34-week study. Approximately 136 subjects will be enrolled at approximately 60 US centers in the Original Design. If the study is adapted to the Contingent Design, approximately 314 subjects will be enrolled at approximately 80 US centers.

Institution
MUSC
Recruitment Contact
Abbi Reed
(843) 792-1820
reedab@musc.edu

Multicenter Randomized Pragmatic Clinical Trial Comparing Two-versus-Three Antibiotic Therapy for Pulmonary Mycobacterium Avium Complex Disease Save

Date Added
July 16th, 2019
PRO Number
Pro00089685
Researcher
Patrick Flume

List of Studies


Profiles_link
Keywords
Infectious Diseases, Lung, Pulmonary
Summary

The purpose of this study is to better understand treatment practices for Mycobacterium avium complex (MAC) disease. This study is comparing the effectiveness and tolerability of taking 2 drugs compared to 3 drugs in people with MAC disease.

Institution
MUSC
Recruitment Contact
Abigail Grady
843-792-2072
gradyabi@musc.edu

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

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

List of Studies


Profiles_link
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
Whitney Billingsley
843-792-5526
billingw@musc.edu

Study to Understand Disease Course Inflammation and Endotype of South Carolinians with Nontuberculous Mycobacteria Save

Date Added
June 18th, 2019
PRO Number
Pro00089284
Researcher
Bryan Garcia

List of Studies

Silhouette
Keywords
Healthy Volunteer Studies, Infectious Diseases, Lung, Non-interventional, Pulmonary
Summary

The goal of this cohort and biorepository is to collect data and blood specimens on individuals with Nontuberculous Mycobacteria (NTM), pulmonary disease, and healthy adults to better understand the illness and ultimately improve the care and survival of those with these conditions.

Institution
MUSC
Recruitment Contact
Abigail Grady
843-792-2072
gradyabi@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) Save

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

List of Studies


Profiles_link
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

Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging, Efficacy and Safety Study with Inhaled RVT-1601 for the Treatment of Persistent Cough in Patients with Idiopathic Pulmonary Fibrosis (IPF): SCENIC Trial Save

Date Added
May 28th, 2019
PRO Number
Pro00087983
Researcher
Timothy Whelan

List of Studies


Profiles_link
Keywords
Lung, Pulmonary
Summary

The purpose of this study is to test the effects of the study drug, RVT-1601, in research subjects with persistent cough due to idiopathic pulmonary fibrosis (IPF) in improving cough (frequency and severity) and quality of life. RVT-1601 is delivered as a mist through an eFlow® nebulizer that research subjects will inhale through a mouthpiece attached to the nebulizer. The Screening period will last up to 4 weeks, in order to give the study doctor time to determine if you qualify for the study. After the Screening Period, you will participate in the study for approximately 24 weeks (6 months). Your total study participation is expected to be 28 weeks (7 months).

Institution
MUSC
Recruitment Contact
Abbi Reed
(843) 792-1820
reedab@musc.edu

Change_preferences

-- OR --

Create_login