A Phase IIa, Randomized, Double-Blind, Placebo-Controlled Study To Evaluate Multiple Doses Of Glpg2222 In Subjects With Cystic Fibrosis Who Are Homozygous For The F508del Mutation Save

Date Added
June 13th, 2017
PRO Number
Pro00065901
Researcher
Patrick Flume
Keywords
Cystic Fibrosis, Lung, Pulmonary
Summary

Cystic fibrosis is caused by changes in the genes of your DNA (Deoxyribonucleic Acid) encoding for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein. These changes cause the CFTR to stop working as it should. GLPG2222 is being studied for treatment of cystic fibrosis. The study drug is designed to improve the expression and function of CFTR, which may improve some of the symptoms of cystic fibrosis.

Institution
MUSC
Recruitment Contact
David Longshore
843-792-9697
longshor@musc.edu

Phase 3b, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Safety, Efficacy, and Tolerability of Tezacaftor/Ivacaftor (TEZ/IVA) in an Orkambi-experienced Population Who Are Homozygous for the F508del-CFTR Mutation Save

Date Added
May 23rd, 2017
PRO Number
Pro00066198
Researcher
Patrick Flume
Keywords
Cystic Fibrosis, Pediatrics, Pulmonary
Summary

The purpose of this study is to learn more about the safety and efficacy of the combination of tezacaftor/ivacaftor (TEZ/IVA) in subjects with CF who have previously discontinued Orkambi. Orkambi® is a combination of ivacaftor with another drug called lumacaftor. This study is being conducted to test whether combining ivacaftor with tezacaftor instead will improve symptoms of cystic fibrosis and cause fewer respiratory side effects.

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

Prospective, randomized, placebo controlled trial of the efficacy and safety of inhaled nitric oxide (NO) in cystic fibrosis (CF) patients Save

Date Added
April 11th, 2017
PRO Number
Pro00064789
Researcher
Patrick Flume
Keywords
Cystic Fibrosis, Drug Studies, Lung, Pulmonary
Summary

This study is being done to evaluate an experimental treatment for people with Cystic Fibrosis who have a bacterial infection in their lungs. Subjects will be randomized (selected by chance) to either be treated with an investigational inhaled gaseous drug (in 99.5% nitrogen) that is mixed with room air or breathe 100% nitrogen that is mixed with room air. If you are randomized to the investigational treatment, we are asking you to breathe in a gas called ?nitric oxide?. Participation in the study will take about 43 days

Institution
MUSC
Recruitment Contact
Ashley Warden
843-792-4349
jonesash@musc.edu

A Phase 3, Rollover Study to Evaluate the Safety of Long term Treatment With Lumacaftor/Ivacaftor Combination Therapy in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation Save

Date Added
March 28th, 2017
PRO Number
Pro00064086
Researcher
Patrick Flume
Keywords
Cystic Fibrosis, Drug Studies, Lung, Pulmonary
Summary

This study is being done to learn more about the long-term safety and effects from taking the combination of lumacaftor/ivacaftor in a previous Vertex study (VX15-809-115 Part B).

Institution
MUSC
Recruitment Contact
Ashley Warden
843-792-4349
jonesash@musc.edu

Multiple Patient Program for Lamprene® (clofazimine) for the treatment of Non-Tuberculous Mycobacterial (NTM) Infections Save

Date Added
March 21st, 2017
PRO Number
Pro00065207
Researcher
Patrick Flume
Keywords
Pulmonary
Summary

Lamprene®/Clofazimine, is a product of the pharmaceutical company named Novartis Pharmaceuticals Corporation. Lamprene®/Clofazimine is approved by FDA (the U.S Food and Drug Administration) for the treatment of leprosy. It is being tested in non-Novartis clinical studies for drug resistant tuberculosis and non-tuberculous mycobacteria (NTM).

If you have been diagnosed with NTM, then your doctor may decide that this infection can be treated with Lamprene®/Clofazimine. This medicine is provided to you in an expanded access program. This means that this medicine is not registered for the treatment of NTM, but it can be used in special situations where there are no other possible treatments. For example, this may be because you have a type of Mycobacterial infection that is resistant or failed to respond optimally to other drugs, or because you have had side effects that prevent the use of other drugs.

Institution
MUSC
Recruitment Contact
Robyn Do
843-792-1221
dorobyn@musc.edu

A Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety of VX-152 Combination Therapy in Adults With Cystic Fibrosis Save

Date Added
October 25th, 2016
PRO Number
Pro00059272
Researcher
Patrick Flume
Keywords
Cystic Fibrosis, Drug Studies, Lung, Pulmonary
Summary

This is a Phase 2, 2-part study. Adults over the age of 18 with cystic fibrosis are eligible.

VX-152 given alone or in combination with VX-661 and ivacaftor is an investigational drug and is still being tested for safety and effectiveness. VX-661 given alone or with ivacaftor is investigational and is still being tested for safety and effectiveness. Ivacaftor, the third drug used in this study is approved in a number of countries globally for use in patients with CF as young as 2 years, depending on the country. Ivacaftor (Kalydeco) is approved in the United States for use in patients with CF.

Institution
MUSC
Recruitment Contact
Ashley Warden
843-792-4349
jonesash@musc.edu

A Phase 3, Open-label Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of VX-661 in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation Save

Date Added
September 27th, 2016
PRO Number
Pro00057745
Researcher
Patrick Flume
Keywords
Cystic Fibrosis, Drug Studies, Lung, Pulmonary
Summary

This study will evaluate the pharmacokinetic of VX-661 and ivacaftor after administration of multiple doses of VX-661 in combination with ivacaftor over 8 weeks with subjects 6 to 11 years old who are homozygous or heterozygous for the F508del-CFTR Mutation. The 8 weeks will consist of a screening period of 4 weeks, a treatment period that will last 2 weeks, and if needed, a follow up visit taking place 2 weeks after the first dose of the Study Drug.

This is an open-label study with two treatment groups based on weight. The purpose of this study is to learn more about the safety and tolerability of the combination of VX-661 and ivacaftor in subjects with CF. Part A of the study will be receiving one 50mg tablet of VX-661 in the morning, one 75mg capsule of ivacaftor in the morning and one 75mg capsule of ivacaftor in the evening. Part B receiving one 50mg tablet of VX-661 in the morning, one 150mg tablet of ivacaftor in the morning and one 150mg tablet of ivacaftor in the evening.

Institution
MUSC
Recruitment Contact
Ashley Warden
843-792-4349
jonesash@musc.edu

A Multi-center, Randomized, Placebo-Controlled, Ascending Dose Phase I Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects with Cystic Fibrosis Save

Date Added
June 28th, 2016
PRO Number
Pro00056260
Researcher
Patrick Flume
Keywords
Cystic Fibrosis, Drug Studies, Lung, Pulmonary
Summary

This study aims to test the safety and tolerability of the study drug, PTI-428, when used to treat cystic fibrosis. Approximately 36 individuals will take part study-wide and adult males and females of the ages 18-55 years old with a diagnosis of Cystic Fibrosis are eligible to participate.

There are 2 different groups within the study and depending on the group you paticipate in the study will last between 5 to 7 weeks.

Institution
MUSC
Recruitment Contact
Ashley Warden
843-792-4349
jonesash@musc.edu

Standardized Treatment of Pulmonary Exacerbations II (STOP2) Save

Date Added
May 17th, 2016
PRO Number
Pro00054432
Researcher
Patrick Flume
Keywords
Cystic Fibrosis, Lung, Pulmonary
Summary

The study is being used to evaluate the efficacy and safety of differing durations of IV antibiotic treatment for CF pulmonary exacerbations. Subject eligibility includes males and females 18 years of age or older. Participation in the study will take 3 visits over a period of 24 to 35 days.

Institution
MUSC
Recruitment Contact
Ashley Warden
843-792-4349
jonesash@musc.edu

A prospective observational study in cystic fibrosis patients with chronic respiratory Pseudomonas aeruginosa infection treated with TOBI® Podhaler™ (tobramycin inhalation powder) or other FDA approved inhaled antipseudomonal antibacterial drugs Save

Date Added
February 23rd, 2016
PRO Number
Pro00044578
Researcher
Patrick Flume
Keywords
Adolescents, Breathing, Cystic Fibrosis, Lung, Pulmonary
Summary

You are invited to volunteer for this non-interventional study if you have been prescribed tobramycin inhalation powder (TOBI® Podhaler™) or another inhaled medication used to treat Cystic Fibrosis (CF) related Pseudomonas aeruginosa (bacteria).

The purpose of this non-interventional study is to assess how well TOBI Podhaler or other approved inhaled antibiotic medications work on treating this bacterium in the lungs, and their safety as used in medical practice. A non-interventional study is a study in which individuals are only observed and certain outcomes are measured. The study will entail around 20 visits over the course of 5 years.

Institution
MUSC
Recruitment Contact
Ashley Warden
843-792-4349
jonesash@musc.edu

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