A Multi-Center, Randomized, Placebo-Controlled, Phase 1, Two-Part Study Designed to Assess the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects with Cystic Fibrosis Save

Date Added
September 26th, 2017
PRO Number
Pro00070908
Researcher
Patrick Flume
Keywords
Cystic Fibrosis, Lung, Pulmonary
Summary

This is a research study to test a new investigational drug, PTI-801. An investigational drug is one that is not approved by the US FDA. In this study, PTI-801 will be compared to placebo (a capsule that looks like the investigational drug, but does not contain an active drug substance). Although PTI-801 has been given to normal healthy adults, this is the FIRST time that PTI-801 has been given to people with CF.

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

Cystic Fibrosis Patients With Active Pulmonary Exacerbation Receiving IV Tobramycin At Risk For Ototoxicity: Part 1: An Observational Study With Audiologic And Vestibular Assessments: Part 2, A Phase 1b, Randomized, Double-Blind, Placebo-Controlled Study To Evaluate The Safety, Pharmacokinetics And Pharmacodynamics Of SPI-1005 Capsules Save

Date Added
August 8th, 2017
PRO Number
Pro00067873
Researcher
Patrick Flume
Keywords
Cystic Fibrosis, Lung, Pulmonary
Summary

This study has two parts and will assess your hearing and the potential effects of antibiotic-induced ototoxicity. Ototoxicity is a potential effect of drugs used to treat your lung infections, and its effect on your ears (oto=ears), particularly structures in your inner ear that enable hearing and balance. Part One of the study will assess your inner ear function that may be associated with hearing loss and / or loss of balance in cystic fibrosis patients taking tobramycin. Part Two is looking to determine if SPI-1005, an investigational drug, influences hearing loss, due to mediations (tobramycin).

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

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

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 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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