A Phase 1b, Randomized, Double-Blind, Placebo-Controlled, Dose Escalation Study of N6022 to Evaluate Safety and Pharmacokinetics in Subjects with Cystic Fibrosis Homozygous for the F508del-CFTR Mutation. 

This purpose of this research study is to learn more about the safety, tolerability, pharmacokinetics of investigational study drug called N6022 in people with Cystic Fibrosis (CF) as compared to placebo. A placebo is a substance which has no effect. The drug is administered once daily through an IV for 7 days. This will be done both inpatient and outpatient. Researchers will also look at the effects of these drugs on markers of inflammation, sweat chloride results, nasal potential difference (NPD) and pulmonary function tests. 

People 18 years of age and older with CF, who are heterozygous for the F508del‑CFTR mutation and who meet other inclusion criteria may qualify to participate. 

Your participation in this research study will last for up to 6 weeks. At the study visits, you may have a physical exam including vital signs, height and weight, blood and urine tests, sweat chloride tests, pulmonary function testing, nasal potential difference test, induces sputum and electrocardiograms (ECG) testing of your heart.  You will have up to 3 overnight stays in the Johns Hopkins Hospital and up to 8 outpatient visits in the Clinical Research Unit (CRU). You will be compensated for your time, travel and parking.

PI: Pamela L. Zeitlin, MD, PhD
Contact: Karen Callahan, RN, CCRP at 410-955-1167 
Protocol No.: NA_00079361
Send Me Info

 A Phase 3, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis who have the R117H-CFTR mutation.

The purpose of this research study is to test a newly approved drug called Kalydeco (formerly VX770) in cystic fibrosis patients, ages 6 and up, with at least one R117H genetic mutation. This study is a randomized trial. That means you will be put in one of two groups in a randomized way, like flipping a coin. One group will get 150mg of Kalydeco to take every 12 hours for 24 weeks. The other group will get a placebo, or sugar pill that does not have any active ingredient in it. All subjects who complete the study will be eligible for an open label extension study. This means everyone will get Kalydeco free of charge in the extension study. There are 2 screening visits, 6 study visits and one follow up visit over about 32 weeks. You will need to come to Johns Hopkins Hospital for all your visits. They are all done in the outpatient clinical research units. We will ask you to read the consent and decide if the study is right for you.

PI: Michael Boyle, MD
Contact: Karen Callahan, RN, CCRP at 410-955-1167 
Application No.: NA_00073258
Send Me Info

G551D Observational Study (GOAL)

The purpose of this observational research study is to learn more about the effects of a newly approved (or soon to be approved) drug called ivacaftor (formerly VX770). This drug has been tested in cystic fibrosis patients with at least one G551D genetic mutation.

This study is observational. That means the investigators will not be putting anyone in groups or assigning them to a group. They are interested in the possible effects this drug has on CFTR by analyzing blood, sputum and urine before and after the use of ivacaftor. Subjects will be enrolled and followed for up to 18 months.
We will ask you to read the consent and decide if the study is right for you. If you agree, we will ask you to come in for a screening visit. Then we will wait to hear from you if you are put on the drug. If your doctor puts you on ivacaftor anytime during those 12 months after the first screening visit, we will ask you to come in for 4 additional study visits and call you approximately 5 times. Study visits are done one before the first dose, and then at 1, 3 and 6 months after that first dose for those subjects who are prescribed ivacaftor.

For patients who are not prescribed ivacaftor within 12 months of screening, we will ask you to come in one time between 12-18 months after that initial screening visit.
This study does not provide ivacaftor. Participation in this study will not change your care and usual CF medications are allowed to be continued.

PI: Pamela L. Zeitlin, MD, PhD
Contact: Carolyn Chapman, RN at (410) 955-1167
Application No.: NA_00067774
Send Me Info

 Early Intervention in Cystic Fibrosis Exacerbations

This study compares usual care to a program using home electronic devices that measure lung function and CF symptoms, to identify and treat pulmonary exacerbations in CF patients ages 14 and older.  The purpose of this study is to determine if earlier treatment of exacerbations will improve lung function and quality of life.

Interested participants will be randomized (like flipping a coin) into two groups: usual care or early intervention.

The early intervention group will use home spirometers twice a week to record lung function and answer short questions.  They will then send the information electronically to the cystic fibrosis center using either a telephone or computer.   The usual care group will not use home monitors and will be asked to call the clinic if they have symptoms of a pulmonary exacerbation.

In both groups, you will be asked to come to the CF clinic every 3 months for examination and to answer questionnaires. These visits can be arranged to match with your regularly scheduled clinic appointments. Additional visits to the CF clinic may also be appropriate when the spirometers or you detect an exacerbation.

All participants will be enrolled in the study for 12 months. Leaving this study early will not stop you from getting regular medical care. 

You will receive compensation and parking for clinic visits required by the study.

PI: Noah Lechtzin, MD, MHS
Contact: Noah Lechtzin, MD, MHS at 410-502-7044.
Research Study # NA_00047034
More 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: Currently Enrolling

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 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 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: Currently Enrolling

P.I.: Pamela L. Zeitlin, M.D., Ph.D.
Contact: Karen Callahan, RN, CCRPat 443-287-8983 
Send Me Info