A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation Save

Date Added
August 14th, 2018
PRO Number
Pro00078283
Researcher
Patrick Flume
Keywords
Cystic Fibrosis, Lung, Pediatrics, Pulmonary
Summary

The purpose of this stuThe purpose of this study is to learn more about the safety and effectiveness of VX-445 used in combination with TEZ and VX-561 in patients with cystic fibrosis. Participation could last for approximately 100 weeks. This will include a treatment period that will last approximately 96 weeks, and a safety follow-up period that will last approximately 4 weeks after the last dose of Study Drug. Approximately 400 people will participate in this study.

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

A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect of PTI-428 in Subjects with Cystic Fibrosis Save

Date Added
July 24th, 2018
PRO Number
Pro00080286
Researcher
Patrick Flume
Keywords
Cystic Fibrosis, Lung, Pulmonary
Summary

The is a Phase 2, randomized, double-blind, placebo-controlled study that will be
conducted at multiple centers. The study population is comprised of adult subjects with Cystic Fibrosis, and who have been taking tezacaftor/ivacaftor for the treatment of the disease. The planned sample size is approximately 40 subjects. Participation in this study will last up to approximately 10 weeks and include a screening visit and 9 visits to the study center.

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

A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F) Save

Date Added
June 12th, 2018
PRO Number
Pro00078198
Researcher
Patrick Flume
Keywords
Cystic Fibrosis, Lung, Pediatrics, Pulmonary
Summary

The purpose of this study is learn more about the effectiveness and safety of VX-445 used in combination with TEZ, IVA and VX-561 in patients with cystic fibrosis.
Participation could last for approximately 16 weeks. This will include a Screening Period that will last up to 4 weeks, a Run-in Period that will last up to 4 weeks, a Treatment Period that will last up to 4 weeks, and a Follow-up Period that may last up to 4 weeks after the last dose of Study Drug. Approximately 100 people will participate in this study.

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

A Phase 1 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
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 57 days

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

A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) Save

Date Added
May 22nd, 2018
PRO Number
Pro00078126
Researcher
Patrick Flume
Keywords
Cystic Fibrosis, Lung, Pediatrics, Pulmonary
Summary

The purpose of this study is to learn more about the effectiveness and safety of VX-445 used in combination with TEZ and VX-561 in patients with cystic fibrosis. Participation could last for approximately 32 weeks. This will include a Screening Period that will last up to 4 weeks, a Treatment Period that will last up to 24 weeks, and a Follow-up Period that may last up to 4 weeks after the last dose of Study Drug. Approximately 360 people will participate in this study.

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

A Post-approval Observational Study to Evaluate the Long-term Effectiveness and Safety of Orkambi in US Patients Who Completed Study VX12-809-105 Part A Save

Date Added
May 21st, 2018
PRO Number
Pro00076856
Researcher
Patrick Flume
Keywords
Cystic Fibrosis
Summary

This study is being done to evaluate the long-term outcomes of patients with CF who are homozygous for the F508del-CFTR mutation and previously took part in Study VX12-809-105 and are currently participating in the CF Registry.

No part of this study is experimental. There will be no more than 485 people participating in this study. No additional medical tests or procedures are needed to participate in this study.

Institution
MUSC
Recruitment Contact
Allison Patterson
(843) 792-5168
patteall@musc.edu

A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR Mutation Save

Date Added
March 27th, 2018
PRO Number
Pro00075578
Researcher
Sylvia Szentpetery
Keywords
Cystic Fibrosis, Lung, Pediatrics, Pulmonary
Summary

The purpose of this study is to learn more about the long-term safety, tolerability, and effects of the combination of VX-661 and ivacaftor in subjects with Cystic Fibrosis. This is a rollover study from study VX 15-661-113 Part B. Approximately 121 people will participate in this study. Participation could last for approximately 100 weeks. This will include a treatment period that will last up to 96 weeks, and a follow-up period that will last up to 4 weeks.

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
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
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
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

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