Infant Study of Inhaled Saline in Cystic Fibrosis The purpose of this research study is to determine if hypertonic (7%) saline improves lung function in young children with CF. Children diagnosed with CF who are aged 4 months to up to 60 months can enroll in this study. Children will receive either hypertonic or a placebo twice daily by nebulizer for 1 year. Lung function will be tested in infants who are aged 4 months up to 16 months using infant pulmonary function testing. The parent (or parents) and child will be asked to come in for 6 visits to the Pediatric Clinical Research Center (PCRU). If your child is participating in the Infant Pulmonary Function testing, you will be asked to come for 7 visits to the PCRU and, during 2 of those visits, your child will have pulmonary function testing performed. Each study visit lasts about 3 hours. Volunteers will be compensated for their time and will receive a parking voucher for each visit. For additional information click here. STATUS: Currently Enrolling
P.I.: Peter Mogayzel, M.D., Ph.D.
Contact: Karen Callahan or Carolyn Chapman 410-955-1167
Protocol #: NA_00017484
Send Me Info 
A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis This purpose of this research study is to test a study drug called PTC124. Researchers hope to learn more about whether PTC124 can improve lung function, reduce the symptoms associated with CF, decrease the number of hospitalizations and the use of antibiotics for CF-related lung infections, reduce cough, improve overall patient quality of life, and to determine if it is safe for children and adults to take as a long-term treatment.The overall recruitment goal for this study is 208 subjects in about 38 sites, internationally. Our site will recruit 5 children and/or adults with cystic fibrosis (CF) who have a nonsense mutation and who meet eligibility criteria.
People 6 years of age and older with cystic fibrosis, who have a nonsense mutation (a genotype with an ‘X’ in its name such as G542X, W1282X, or R553X) may qualify to participate
The study drug will be taken 3 times a day (morning, noon and nighttime) within 30 minutes of a meal. It is a powder that can be mixed in 1-2 fluid ounces of water or milk and will be taken every day for 48 weeks. PTC124 will be given to half of the subjects and the other half will receive the "fake" drug or placebo. A placebo has no effect but looks just like the study drug. Should you agree to participate in this research, you will be in the study for about 1 year, and will be followed for safety 5 years after your last study doses. After the first year, the study team will call you to follow up on your health every 6 months in YR 1 and 2, then once a year in YR 3, 4 and 5. At the study visits, you will have a physical exam including vital signs, blood and urine tests, sweat chloride tests, pulmonary function testing, nasal potential difference (NPD) tests, and EKG testing of your heart. You will answer questionnaires about your health, be asked to complete an electronic diary daily, wear a LifeShirt vest to capture cough for 24 hours four times over the first year, and have a renal ultrasound and CT scan of your lungs twice. You will be compensated for your time, travel and parking. STATUS: Currently Enrolling P.I.: Pamela Zeitlin, M.D, Ph.D.
Contact: Karen Callahan or Carolyn Chapman or Erin Felling at 410-955-1167
Protocol #: NA_00028826
Send Me Info
Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis (PUSH) The purpose of this research study is to learn more about liver disease in people with cystic fibrosis (CF) using a test called a liver ultrasound. CF is an inherited disease that involves the lungs and the digestive system, including the liver. It is hard to predict which people with CF are at greater risk for liver disease. It is also hard to identify liver disease in the early stages in people with CF. The liver ultrasound test may help to diagnose and treat liver disease in people with CF. In this 5-year study, participants will have labwork and complete questionnaires once a year and have a liver ultrasound test every two years. STATUS: Enrollment has not yet started PI: Shruti M. Paranjape, M.D.
Contact: Karen Callahan or Carolyn Chapman or Erin Felling at 410-955-1167
Protocol #: NA_00026772
Send Me Info
A Phase 3, International, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Efficacy and Safety Study of Denufosol Tetrasodium Inhalation Solution in Patients with Cystic Fibrosis Lung Disease and FEV1 ? 75% Predicted (TIGER-2 08-110)
The purpose of this study is to investigate the safety and effectiveness of the investigational drug denufosol in patients with mild Cystic Fibrosis (CF) lung disease. Denufosol is being studied to see if it helps the body clear secretions out of the lung. Children and adults 5 years and older may be able to participate. Denufosol is an inhalation solution that is delivered via a nebulizer. It is being investigated as an early intervention approach to improve clearance of secretions out of the lungs in CF patients.
The study may last for approximately 6 months. 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 one chest x-ray. 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, travel and parking. For additional information click here. STATUS: No longer enrolling P.I.: Pamela Zeitlin, M.D, Ph.D.
Contact: Karen Callahan, R.N. 410-955-1167
Protocol #: NA_00015316
Send Me Info
Long Term Administration of Inhaled Mannitol in Cystic Fibrosis--A Safety and Efficacy Study STATUS: No longer enrolling P.I. Pamela Zeitlin, M.D, Ph.D.
Contact: Karen Callahan, R.N. at 410-955-1167
Send Me Info
A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study of VX-809 to Evaluate Safety, Pharmacokinetics, and Pharmacodynamics of VX-809 in Cystic Fibrosis Subjects Homozygous for the deltaF508-CFTR Gene Mutation
The purpose of this research study is to learn more about the safety and effect that the study drug, VX-809 has on people with Cystic Fibrosis (CF) as compared to placebo. A placebo is a “sugar pill” which has no effect. VX-809 is a study drug being tested to see if it improves the function and quantity of a protein called CFTR. Problems with CFTR in patients with CF lead to thick secretions in the lungs and other organs. This study will determine how the body absorbs and processes the study drug and determine drug dosing. People 18 years of age and older with Cystic Fibrosis, who have a delta F508 CFTR mutation, may qualify to participate. Your participation in this research study will last for about 60 days. The study drug will be a capsule taken once a day over a 28 day period. You will have physical exams including vital signs, blood and urine tests, sweat chloride tests, pulmonary function testing, nasal potential difference (NPD) tests, and EKG testing of your heart. You will answer questionnaires about your health and have 2 overnight stays in the hospital. You will be compensated for your time, travel and parking. Study number: NA_00025569 STATUS: No longer enrolling P.I.: Michael Boyle, M.D.
Contact: Karen Callahan, RN, CCRP at 443-287-8983 or Carolyn Chapman, RN at 410-955-1167
Send Me Info
Correlation of Self-Reported Physical Activity to Exercise Tolerance, Pulmonary Function, and Nutritional Status in Cystic Fibrosis
The purpose of this research study is to see if people with cystic fibrosis (CF) can use exercise to help improve their overall physical health and nutrition. CF is an inherited disease that involves the lungs. When lung disease is severe, people with CF can have problems with exercise and not know how much or how little exercise they are able to do. Exercise has been shown to make lungs healthier in CF and make people feel better in general. In this study, participants will take part in the 8-week exercise program, complete questionnaires and perform walking tests. Application No.: NA_00018509
STATUS: No longer enrolling PI: Shruti M. Paranjape, M.D.
Contact: Karen von Berg, P.T. at 410-614-3234
Send Me Info
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. STATUS: Enrollment has not yet started P.I.: Pamela L. Zeitlin, M.D., Ph.D.
Contact: Karen Callahan, RN, CCRPat 443-287-8983
Send Me Info
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. STATUS: Enrollment is complete P.I.: Peter J. Mogayzel, Jr., M.D., Ph.D.
Contact: Peter J. Mogayzel, Jr., M.D., Ph.D. at 410-955-2795
Send Me Info
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. STATUS: Currently enrolling 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 or visit the website.
 | 
