A Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis Save

Date Added
April 23rd, 2019
PRO Number
Pro00087417
Researcher
Bryan Garcia

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Keywords
Cystic Fibrosis, Lung, Pulmonary
Summary

The purpose of this study is to learn more about the effectiveness and safety of VX-561 used in patients with cystic fibrosis. Patients who have cystic fibrosis and are currently taking a stable dose of ivacaftor (IVA) are invited to take part in this research study. VX-561 is a form of IVA, an approved medication for some types of CF, that lasts longer in the body, allowing the medicine to be taken once a day instead of twice a day. Participation could last for approximately 20 weeks. There will be approximately 88 people participating in this study.

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

Prospective study of peripherally inserted venous catheters in CF patients Save

Date Added
November 20th, 2018
PRO Number
Pro00083486
Researcher
Patrick Flume

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Profiles_link
Keywords
Cystic Fibrosis, Lung, Pulmonary
Summary

The main purpose of this study is to see if important factors can be identified in each of the three categories (patient, catheter, and catheter management) that are linked to blood clotting complications. This is a multicenter, prospective observational study to evaluate risk factors associated with complications of peripherally inserted central catheter (PICCs) and midline catheters. Both adult and pediatric patients with CF who receive care at participating centers will be eligible for participation. This study can last for 2 weeks.

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

A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults with Cystic Fibrosis Save

Date Added
May 22nd, 2018
PRO Number
Pro00078211
Researcher
Patrick Flume

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Profiles_link
Keywords
Cystic Fibrosis, Lung, Pulmonary
Summary

In this study, PTI-808 will be given alone and in combination with two other experimental drugs, PTI-801 and PTI-428. PTI-428, PTI-801, and PTI-808 are considered cystic fibrosis transmembrane conductance regulator (CFTR) modulators being developed to treat the underlying mechanism of cystic fibrosis. PTI-428 is a CFTR amplifier that increases the amount of CFTR protein in the cell, PTI-801 is a CFTR corrector that improves the processing of CFTR protein in the cell, and PTI-808 is a CFTR potentiator to enhance the activity of CFTR at the cell surface. Individual modulators have been shown to have limited activity, and therefore it is predicted that a combination of modulators will be required to fully address the CFTR protein defects. The study will also obtain information on how the study drug moves into, through, and out of the body. The expected duration of this study is up to 74 days

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

Randomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas aeruginosa (PA) Infection/Colonization Save

Date Added
March 16th, 2018
PRO Number
Pro00072857
Researcher
Daniel e. Brown

List of Studies

Keywords
Cystic Fibrosis
Summary

This is a randomized, double-blind, multi-center study in pediatric
subjects age 3 months to less than 18 years with CF and newly
detected PA respiratory tract colonization/infection. The study
schedule will consist of a minimum of 13 visits: Screening, Day 1
(Baseline and Randomization), Day 29, Weeks 6, 8, 16, and at
12-week intervals thereafter through Week 112. Subjects may be
screened up to 14 days prior to the Baseline visit to determine
eligibility for participation in the study. Screening and Baseline may
occur on the same day for subjects.

Institution
Palmetto
Recruitment Contact
Barbara (Barb) Thompson
803-434-4986
Barbara.Thompson@uscmed.sc.edu

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis Save

Date Added
February 27th, 2018
PRO Number
Pro00075593
Researcher
Patrick Flume

List of Studies


Profiles_link
Keywords
Cystic Fibrosis, Lung, Pediatrics, Pulmonary
Summary

This is a research study to find out if a drug called lenabasum can help people with Cystic Fibrosis (CF) and if it is safe to take without causing too many side effects. There are 9 planned study visits in the institution during this study.

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

The CHEC-SC Cohort Study: CHaractErizing CFTR Modulated Changes in Sweat Chloride and their Association with Clinical Outcomes Save

Date Added
January 10th, 2018
PRO Number
Pro00073414
Researcher
Sylvia Szentpetery

List of Studies

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Keywords
Cystic Fibrosis, Lung, Pediatrics, Pulmonary
Summary

This is an observational study which will collect sweat chloride values from approximately 1000 cystic fibrosis subjects who are currently on cystic fibrosis transmembrane conductance regulator (CFTR) modulators. This study will require one visit to an MUSC clinic which will last approximately 2 hours. Compensation is provided.

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

A PHASE III, RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED STUDY OF AEROVANC FOR THE TREATMENT OF PERSISTANT METHICILLIN-RESISTANT Staphylococcus aureus LUNG INFECTION IN CYSTIC FIBROSIS PATIENTS Save

Date Added
October 24th, 2017
PRO Number
Pro00071904
Researcher
Patrick Flume

List of Studies


Profiles_link
Keywords
Cystic Fibrosis, Lung, Pulmonary
Summary

The purpose of this research study is to evaluate the effectiveness and safety of AeroVanc compared to placebo (an inactive substance) in patients (also called subjects) with CF and a chronic MRSA lung infection.

Institution
MUSC
Recruitment Contact
Julia Palmer
(843) 792-3169
palmerju@musc.edu

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

List of Studies


Profiles_link
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

A Phase 2, Randomized, Double-Blind,Placebo-Controlled Study To Evaluate The Safety and Efficacy of SPI-1005 Capsules in Cystic Fibrosis Patients with Acute Pulmonary Exacerbation receiving IV Tobramycin at risk for Ototoxicity Save

Date Added
August 8th, 2017
PRO Number
Pro00067873
Researcher
Patrick Flume

List of Studies


Profiles_link
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

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

List of Studies


Profiles_link
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

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