A master protocol to test the impact of discontinuing chronic therapies in people with cystic fibrosis on highly effective CFTR modulator therapy (SIMPLIFY)

Date Added
September 3rd, 2020
PRO Number
Pro00103466
Researcher
Sylvia Szentpetery

List of Studies


Keywords
Cystic Fibrosis, Lung, Pulmonary
Summary

The SIMPLIFY study is being done to test whether or not it is safe to stop taking inhaled hypertonic saline or Pulmozyme® (dornase alfa) in those people that are also taking Trikafta™. The SIMPLIFY studyl consists of two similar studies conducted within the same protocol that are being done to answer the following questions:
• Study A: For people with CF taking Trikafta, does stopping treatment with hypertonic saline (at least 3%) have a similar effect on lung function as continuing treatment with hypertonic saline?
• Study B: For people with CF taking Trikafta, does stopping treatment with Pulmozyme have a similar effect on lung function as continuing treatment with Pulmozyme?
There are a total of 4 study visits (a screening visit and three additional visits during the active part of the study). There is a 2-week screening period and a 6-week active study period.About 920 people with CF who are 12 years of age or older will take part in this study at approximately 80 hospitals and clinics around the country. Of this total, approximately 520 people will participate in the hypertonic saline (Study A) portion and 400 people with participate in the Pulmozyme (Study B) portion of the study.

Institution
MUSC
Recruitment Contact
Angel Darrow
(843) 792-8092
darrowa@musc.edu

A PHASE 1B RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLED TRIAL TO EVALUATE THE SAFETY OF CB-280 IN PATIENTS WITH CYSTIC FIBROSIS CX-280-202

Date Added
August 12th, 2020
PRO Number
Pro00102299
Researcher
Patrick Flume

List of Studies


Keywords
Cystic Fibrosis, Lung, Pulmonary
Summary

This clinical study is testing the safety (if there are side effects) and tolerability of CB-280 in approximately 32 subjects with CF. This study is being conducted to indentify which CB-280 doses are safe to take without causing too many side effects. CB-280 is an investigational drug that is taken by mouth that blocks an enzyme called arginase, which is thought to play an important role in promoting lung infection in cystic fibrosis (CF). Participation in the study will be up to 56 days long.

Institution
MUSC
Recruitment Contact
Angel Darrow
(843) 792-8092
darrowa@musc.edu

An Observational Long-Term Safety Surveillance Study Of Participants From Corbus Sponsored Lenabasum Pivotal Clinical Trials

Date Added
July 15th, 2020
PRO Number
Pro00101371
Researcher
Patrick Flume

List of Studies


Keywords
Cystic Fibrosis, Lung, Pulmonary
Summary

This observational research study will collect safety information from subjects who received study treatment (lenabasum or placebo) in a Corbus-sponsored pivotal clinical trial. This is being done to understand how stopping the study treatment affects the subject, and to make sure that the subject is not experiencing any side effects from stopping the study treatment. The subject will not be given any investigational drugs, including lenabasum or a placebo, in this study. The subject will be assessed (by telephone or office visit) approximately every 6 months for up to 2 years from the time of the last safety visit in a pivotal trial, unless Corbus stops studying lenabasum or decides to stop the study itself. The number of potential phone calls will depend on when the subject joins the study, but will not exceed 5 total. Approximately 100 subjects are expected to take part in this study in North America, Europe, and Asia-Pacific (APAC).

Institution
MUSC
Recruitment Contact
Nadine O'Connor
(843) 792-6890
oconnorn@musc.edu

Prospective Phase 0 Study to Investigate Pseudomonas aeruginosa and Staphylococcus aureus Bacterial Load, Patient Characteristics and Exploratory Biomarkers in Adult Patients with Cystic Fibrosis or Non-Cystic Fibrosis Bronchiectasis

Date Added
June 25th, 2020
PRO Number
Pro00100720
Researcher
Patrick Flume

List of Studies


Keywords
Cystic Fibrosis, Lung, Pulmonary
Summary

The purpose of this research study is to collect phlegm and blood to learn more about the presence of bacteria in patients with Cystic Fibrosis (CF) or Non-Cystic Fibrosis Bronchiectasis (NCFB). This exploratory research study does not involve the administration of any medication. There are approximately 4 visits (1 visit to see for subject eligibility, and 3 visits during the study) to complete the study participation. The study will take two months. About 100 subjects will take part study-wide.

Institution
MUSC
Recruitment Contact
Angela Millare
(843) 792-3710
millare@musc.edu

APPLAUD: A DOUBLE-BLIND, RANDOMIZED, PLACEBO CONTROLLED, PHASE II STUDY OF THE EFFICACY AND SAFETY OF LAU-7B IN THE TREATMENT OF CYSTIC FIBROSIS IN ADULTS

Date Added
November 12th, 2019
PRO Number
Pro00094338
Researcher
Bryan Garcia

List of Studies


Keywords
Cystic Fibrosis, Lung, Pulmonary
Summary

The purpose of this study is to learn more about the effectiveness and safety of the investigational drug called fenretinide (LAU-7b). The FDA has approved the testing of fenretinide in this study. Researchers hope that treatment with fenretinide will preserve lung function by reducing the persistent inflammation in the lung and to improve its capacity to defend against bacteria such as Pseudomonas aeruginosa. The drug will be administered by capsules. Your participation in this study will last approximately 7 months and includes 8 study visits to the study center (which will last approximately 2-5 hours) and 6 telephone calls. The phone calls are expected to last 10-15 minutes and depend on the amount of information you report.

Institution
MUSC
Recruitment Contact
Max Lento
(843) 792-9697
lento@musc.edu

A Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in Cystic Fibrosis Subjects Who Are Homozygous for the F508del Mutation

Date Added
October 22nd, 2019
PRO Number
Pro00091705
Researcher
Patrick Flume

List of Studies


Keywords
Cystic Fibrosis, Lung, Pulmonary
Summary

This study is designed to evaluate ABBV-3067 alone and in combination with ABBV-2222 to see how safe they are and how well they work for treating cystic fibrosis. The study will also help select the minimum effective dose of both medicines. Both drugs are investigational and will be taken orally. Both help to restore Cystic Fibrosis transmembrane conductance regulator (CFTR) activity, which is when there isn't enough water on membrane surfaces for those who have cystic fibrosis. You will either receive the drug or a placebo. The study will be done in approximately 3 months. Approximately 189 patients with cystic fibrosis who are older than 18 years of age will be enrolled in this study.

Institution
MUSC
Recruitment Contact
Brooke Alexander
843-792-1221
alexanbr@musc.edu

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

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

List of Studies


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

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

List of Studies


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

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

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

List of Studies


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 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

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

List of Studies


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



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