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

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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

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

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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

Prospective study of peripherally inserted venous catheters in CF patients Save

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

List of Studies


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 Proof of Concept Study of Inhaled Nitric Oxide for Adults with Pulmonary Non-Tuberculous Mycobacterial Infection Save

Date Added
September 18th, 2018
PRO Number
Pro00081838
Researcher
Patrick Flume

List of Studies


Profiles_link
Keywords
Lung, Pulmonary
Summary

The purpose of this study is to evaluate the efficacy and safety of open-label exposure of gNO in patients with NTM lung disease. Subjects will receive the study drug by inhaling through a nasal mask. Subjects will be treated for 3 weeks (5 days per week) and followed monthly for 3 months.

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

List of Studies


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

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

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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