A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study of VX-770 to Evaluate Safety, Pharmacokinetics, and Biomarkers of CFTR Activity in Cystic Fibrosis (CF) Subjects with Genotype G551D (VX06-770-101)
The purpose of this research study is to evaluate the safety and tolerability of multiple doses of an investigational (study) drug VX-770 given to patients with Cystic Fibrosis. VX-770 is being studied to see how it helps chloride ion movement in Cystic Fibrosis patients. Adults 18 years and older may be able to participate. This study will last for about 49 days and may involve both inpatient and outpatient visits. You will have blood and urine tests, sweat tests, nasal potential difference testing, pulmonary function testing, and EKGs. You will answer questionnaires about your health, have physical exams and answer questions by telephone from the research team. You will be compensated for your time and travel.
P.I.: Pamela Zeitlin, M.D., Ph.D.
Contact: Lois A. Brass, R.N.: (410) 955-9782
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Effectiveness and Safety of Intermittent Antimicrobial Therapy for the Treatment of New Onset Pseudomonas aeruginosa (Pa) Airway Infection in Young Patients with Cystic Fibrosis (Protocol # EPIC-001)
This research is being done to evaluate the effect of early treatment of new Pseudomonas aeruginosa (Pa) infection in the lungs of young children between the ages of 1 and 12 years old with CF. It may be easier to treat and eliminate Pa when it first infects the lungs of young patients. The child’s participation in this study will last for approximately 18 months. Except for the initial two study visits, the child will be seen on a quarterly basis at the time of routine clinic visits. Blood work, chest X-rays, lung function tests, sputum and throat cultures will be done. Treatment with TOBI for inhalation and or ciprofloxacin or placebo will be provided. Patients will be compensated for your time and travel.
P.I.: Peter J. Mogayzel,Jr. M.D., Ph.D.
Contact: Lois A. Brass, R.N.: (410) 955-9782
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Mucociliary Clearance and Cough Clearance in Children With Cystic Fibrosis (Hypertonic Saline)
This research study is being done to determine if infection with P. aeruginosa reduces the response of the mucociliary clearance (MCC) apparatus to salt solutions that are likely to rehydrate the mucus by increasing the airway surface liquid. Children with CF who are between the ages of 8 and 12 will have 1 screening visit and 2 study visits in the Pediatric Clinic Research Center (PCRU) at Johns Hopkins Hospital. Lung function testing (PFTs) and a method used to produce sputum from deep within the lungs (induced sputum) will be done on the screening visit. PFTs, vital sign measurements and the inhalation of a salt solution mist will be done on each study visit. MCC will also be measured on each study visit. To measure MCC volunteers will inhale a mist that contains a radioisotope and will undergo repeated imaging of the lungs with a gamma camera. Volunteers will be compensated for their time and parking.
P.I.: Beth L. Laube, Ph.D.
Contact: Beth L. Laube at 410-502-5791
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A randomized, double-blind within dose, placebo-controlled study to investigate the safety, tolerability and pharmacokinetics of increasing single and multiple doses (28-day dosing) of tiotropium bromide administered once daily via the Respimat Device in cystic fibrosis patients
This purpose of this research study is to investigate the safety and effectiveness of increasing single and multiple doses (28 days) of once daily investigational medication - tiotropium bromide compared to placebo via the Respimat inhalation device in patients with Cystic Fibrosis. Adults 18 years and older may be able to participate. This study may last up to 28 days, based on which group you are randomized to. You may enroll in either or both of the single dose and multiple dose arms of the study. You will have blood and urine tests, sputum tests, and pulmonary function testing. You will have physical exams and answer calls by telephone from the research team. You will be compensated for your time and travel. P.I.: Pamela Zeitlin, M.D, Ph.D.
Contact: Lois Brass, R.N., CCRP (410) 955-1167
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A Multi-Center, Double Blind, Placebo-Controlled Randomized, Efficacy and Safety Study of Denufosol Tetrasodium (INS37217) Inhalation Solution in Patients with Mild Cystic Fibrosis Lung Disease
The purpose of this study is to investigate the safety and effectiveness of one dose strength of denufosol study medication compared to placebo in patients with mild CF lung disease. Denufosol is being studied to see how it helps the body clear secretions out of the lung. Children and adults 5 years and older may be able to participate. This study will last for about one year and will be in two parts: denufosol or placebo and then after six months, denufosol only. The study medication will be taken three times daily by inhalation. You will have blood and urine tests, sputum tests, pulmonary function testing, and as many as three chest xrays. You will answer questionnaires about your health, have physical exams and answer questions by telephone from the research team. You will be compensated for your time and travel. P.I.: Pamela Zeitlin, M.D., Ph.D.
Contact: Lois Brass, R.N., CCRP (410) 955-9782
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A Pilot Study of the Safety and Efficacy of Digitoxin to Suppress Inflammation in Cystic Fibrosis
This research study is being done to see if Digitoxin can decrease inflammation in the airways of CF patients. Digitoxin is a drug that is used in Europe for treatment of heart failure and is known to decrease inflammation in airway cells. People with CF over 18 years of age will make 6 study visits to the General Clinic Research Center at Johns Hopkins Hopkins over about 56 days. PFTs, EKGs, blood tests, nasal brushings and urine tests will be done at the visits. We will also use a method to get you to produce sputum from deep within your lungs called induced sputum. You will be compensated for your time and travel. P.I.: Pamela L. Zeitlin, M.D., Ph.D.
Contact: Lois Brass, R.N., C.C.R.P (410) 955-1167
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Identification of Patients with Cystic Fibrosis Due to Decreased CFTR Expression
This study is enrolling children and adults with cystic fibrosis (CF) whose genetic tests have only revealed one CFTR (CF gene) mutation. This research is being done to understand why some people have cystic fibrosis without having changes in both CF genes (CFTR). Participants will undergo several routine medical tests in addition a small brush will be used to obtain cells lining your nose and blood will be drawn for genetic tests. Volunteers will receive compensation for participating. P.I.: Peter J. Mogayzel, Jr., M.D., Ph.D.
Contact: Peter J. Mogayzel, Jr., M.D., Ph.D. at 410-955-2795
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Cystic Fibrosis Twin and Sibling Genetic Modifier Study – A Multicenter Study
The reason why we are doing this study is to understand more about CF and how we could better treat people that have it. This research is being done to find the genes and other factors that are responsible for variation among persons with CF. We are interested in twins and siblings because they share similar genes and a similar environment. We want to know why twins and siblings with the same CFTR mutation have very different diseases. We think that other genes, besides CFTR, are playing a role in CF. We also think that the environment influences disease course. For this reason we are collecting blood samples (2 tablespoons) to look at a person’s DNA. We are also collecting personal (work/home environment) and clinic information about the disease course. We plan on comparing clinical symptoms with DNA results to see if we can identify genes that affect the course of illness. All information collected is coded and de-identified to keep it confidential. When possible, the blood sample will be obtained at the same time blood is needed for reasons that are medically indicated. There will be no cost to the patient for any tests done as part of this study. Participating in this study is strictly on a volunteer basis. Patients will not be compensated for their participation. We appreciate your consideration to be part of this study. P.I.: Garry R. Cutting, M.D.
Contact: If you and your sibling would like to participate please feel free to contact us at 1-866-245-8065 and visit our website.
Genetic Modifiers in Cystic Fibrosis Lung Disease P.I.: Pamela L. Zeitlin, M.D., Ph.D.
Contact: Lois A. Brass, R.N., C.C.R.P. (410) 955-1167
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Specimen Collection in Patients with Cystic Fibrosis P.I.: Pamela L. Zeitlin, M.D., Ph.D.
Contact: Lois A. Brass, R.N., C.C.R.P. (410) 955-1167
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