Dr. Michael Boyle with CFTR image in background

Clinical Trials: Ongoing Trials

Share This Page    

Standardized Treatment of Pulmonary Exacerbations II (STOP 2)

The Standardized Treatment of Pulmonary Exacerbations II (STOP 2) is a randomized, controlled, open-label study being done to learn more about CF patients who have a pulmonary exacerbation treated with IV antibiotics. The main purpose of this research is to study the effectiveness and safety of differing durations of IV treatment, given in the hospital or at home for a pulmonary exacerbation in adults with CF.

People 18 years of age and older with CF who are experiencing a pulmonary exacerbation that their doctor wants to clinically treat with IV antibiotics may join. Participation in this study will last approximately 24 to 35 days and will include 3 study visits at Johns Hopkins. Procedures at the Study Visits may include: physical exam, vital signs, height and weight, blood draws, pulmonary function testing, and sputum collection. We are open for enrollment and expect to enroll up to 100 subjects into this study at Johns Hopkins.

You will be compensated for your time. If you expect to be treated with IV antibiotics for a pulmonary exacerbation at home or in
the hospital and are interested, please let us know! We can discuss the study further and answer any questions you may have.

PI: Natalie West, MD, MHS
CONTACT: Abigail Thaxton  at 443-384-7479 or 410-614-4411 or Athaxto2@jhmi.edu.
PROTOCOL: IRB00090863
SEND ME INFO


A Phase 2, Randomized, Double-Blind, Placebo Controlled, Parallel-Group, Exploratory Study to Evaluate Effects of VX-661 in Combination With Ivacaftor on Lung and Extrapulmonary Systems in Subjects Aged 18 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation (Vertex Study Number: VX14-661-111)

The VX14-661-111 study is now enrolling subjects aged 18 years and older with Cystic Fibrosis in this Phase 2 clinical trial. VX-661 is an oral compound developed by Vertex that has been shown to have CFTR corrector properties. Ivacaftor, also developed by Vertex, is a CFTR modulator that has shown improvements in lung function, respiratory symptoms, and weight gain in several Phase 3 studies of subjects with gating CFTR mutations when used in monotherapy.

This VX14-661-111 study is designed to evaluate the efficacy and safety of VX-661 in combination with ivacaftor. Eligible subjects aged 18 years and older, having two F508del mutations, will be randomized to VX-611/ivacaftor combination therapy or placebo. This study includes a Screening Period, a Treatment Period, and a Safety Follow-up Visit.

Subjects who complete the Day 29 Visit and subsequently the Safety Follow-up Visit will be offered the opportunity to enroll into an extension study of VX-661/ivacaftor combination therapy if they meet eligibility requirements for the extension study.

Subjects’ participation in this study will last approximately 12 weeks and will include 6 study visits to the study center. Some of the procedures at the study visits may include: physical exam, vital signs, height, weight, ECG, pregnancy testing, blood draws, urine collection, sweat testing, pulmonary function testing, and nasal potential difference assessment. An eye exam, genotyping, mucociliary clearance assessment, and an assessment to see how your digestive tract is functioning will also be done. Subjects will be compensated for the time and travel.

It is anticipated that enrollment will begin in January 2016, with the expected enrollment of approximately 35 to 45 subjects. We expect to enroll 5 subjects into this study at Johns Hopkins.

PI: Noah Lechtzin, MD, MHS
CONTACT: Azar Nouraky, BS, RN at 410-502-7043 or by email
PROTOCOL: IRB00080322
SEND ME INFO


A Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-440 Combination Therapy in Subjects Aged 12 Years and Older With Cystic Fibrosis (VX15-440-101)

PI: Noah Lechtzin, MD, MHS
CONTACT: Alicia Green at 443-287-7332 or by email
PROTOCOL: IRB00107592
SEND ME INFO


PROSPECT: A Two-part multicenter prospective longitudinal study of CFTR-dependent disease profiling in cystic fibrosis

The PROSPECT observational trial is now enrolling CF patients aged 12 years and older. The purpose of this observational research study is to learn more about the effects of a newly approved (or soon to be approved) combination therapy called lumacaftor/ivacaftor. Ivacaftor treatment has led to rapid,
dramatic and sustained improvements in lung function, weight, quality of life, and measures of CFTR function. It has also shown reductions in pulmonary exacerbations in those with one copy of the G551D mutation. Preclinical testing has shown that combining the CFTR corrector lumacaftor with the potentiator ivacaftor leads to enhanced F508del CFTR activity relative to lumacaftor alone.

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, urine and stool before and after the use of lumacaftor/ivacaftor combination. Subjects will be enrolled in the Part A study for about 4 months. During this time there are 3 visits to gather information about you before starting lumacaftor/ivacaftor. If you are homozygous F508del, you can decide whether to enroll in Part B of the study. Part B will begin after you receive a prescription but before you begin taking lumacaftor/ivacaftor. You will be enrolled in Part B for about 12 months. There are about 7 visits in
Part B. In Part B, you will be asked to do a lung tests called Multiple Breath Washout and FENO. You will also have the option to do a test called a MCC or Mucociliary Clearance Test. If you are interested in this study, your study team will explain more about each of these tests in detail.

This study does not provide lumacaftor/ivacaftor. Participation in this study will not change your care and
usual CF medications are allowed to be continued.

PI: Peter J. Mogayzel, Jr. MD, PhD
CONTACT: Britany Zeglin at 433-287-8983  or by email
PROTOCOL: IRB00052569
SEND ME INFO


A Phase 2, Multi-Center, Randomized, Placebo-Controlled Study of IV Gallium Nitrate in Patients with Cystic Fibrosis (The IGNITE study)

Pseudomonas aeruginosa infections account for approximately 60% of the chronic lung infections in people with cystic fibrosis. Pseudomonas exists in biofilms in airways of people with CF, which are difficult for traditional antibiotics to penetrate. Current antibiotic therapies are only effective at suppressing these infections.

This phase 2 randomized study uses a five-day continuous gallium nitrate intravenous infusion as an antimicrobial agent to kill Pseudomonas strains. We will study the efficacy gallium in cystic fibrosis patients by measuring both lung function and sputum culture results. There will be a total of 120 participants enrolled into this study at 20 cystic fibrosis sites in the United States. Interested participants will be randomly assigned to one of two groups. This is like flipping a coin. The two groups are the gallium nitrate infusion group and the placebo infusion group (saline infusion). The assignment group will not be disclosed to the participant or the investigators. This is called a “doubleblinded study”.

Participants will come to Johns Hopkins Bayview Medical Center for up to 6 study visits over a 2 month period. A special intravenous line (PICC, midline) may be placed at Johns Hopkins Bayview for the administration of the study drug, or, if already in place, a Port-A-Cath may be used. At the discretion of the principal investigator, the infusion will be started either in the outpatient research center and continued at home, or may be started and continued in the inpatient research unit.

If you receive the study drug infusion at home, you may be visited by a home healthcare nurse for support and education regarding the infusion. If you receive the study drug infusion as an inpatient, you will be admitted to Johns Hopkins Bayview Medical Center research unit for up to 6 days and 5 nights for the duration of the infusion. All participants will have blood, urine, and sputum samples collected on day 1, day 6, day 14, day 28 and day 56 of the study for routine screening, gallium nitrate levels, and cultures for bacteria. The blood may be drawn from the placed intravenous line. A separate screening visit may take place up to 7 days prior to day 1. Spirometry measurements for lung function will also be performed at each study visit. All participants who participate in the study will receive compensation and parking validation for every

PI: Noah Lechtzin, MD, MHS
CONTACT: Sarah Allgood at 410-614-4411.
PROTOCOL: IRB00083095
SEND ME INFO


Use of High Flow Nasal Insufflation in Children and Adults with Cystic Fibrosis

People with cystic fibrosis (CF) often lose weight because they have problems with breathing and lung infections. As lung disease gets worse, breathing becomes more difficult and leads to worse nutrition. People who do not have lung diseases normally slow down their breathing when they sleep. We have found that children with CF breathe much harder during sleep, and this could be one reason why they may have trouble growing or gaining weight.

In this research study, we are studying an investigational device called nasal insufflation that delivers a flow of warm and humid air through the nose at night while people are sleeping. We believe that this device will help people with CF breathe easier and more comfortably while they sleep. In this research study, we would like to see if breathing easier at night while asleep can help people with CF grow or gain weight.

We will ask people with CF to use this device at home every night for six months to see if and how much nightly use will change their weight and help improve their sleep. We will give you a device and show you how to use and clean it. We would also like to visit you at your home every month to check your weight and see how you are doing with the device. After six months of using the device, we would like to do a special type of study, called a sleep study, at Johns Hopkins to measure how much this device will (1) ease the work of breathing and (2) reduce the calories needed for breathing. Then we will ask you to not use the device for six months. We would still like to visit you at home to see how you are doing in terms of your weight, your health, and your sleep.

By doing these studies, we hope to learn more about how people with CF can breathe more comfortably while they sleep, and to see if they can grow or gain weight better if they are sleeping better at night.

PI: Hartmut Schneider, MD PhD
CONTACT: Audrey DeWitt,  at 410-955-2035
PROTOCOL: NA_00089089
SEND ME INFO


Sleep-Related Ventilatory Mechanics & Energy Expenditure in Children and Adults with Cystic Fibrosis

People with cystic fibrosis (CF) often lose weight because they have problems with breathing and lung infections. As lung disease gets worse, breathing becomes more difficult and leads to worse nutrition. People who do not have lung diseases normally slow down their breathing when they sleep. We have found that children with CF breathe much harder during sleep, and this could be one reason why they may have trouble growing or gaining weight.

In this research study, we are studying an investigational device that delivers a flow of warm and humid air through the nose at night while people are sleeping. We believe that this device will help people with CF breathe easier and more comfortably while they sleep. In this research study, we would like to see if breathing easier at night while asleep can help people with CF grow or gain weight.

By doing a special type of study, called a sleep study, at Johns Hopkins, we will measure how much this device will (1) ease the work of breathing and (2) reduce the calories needed for breathing. By doing these studies, we hope to learn more about how people with CF can breathe more comfortably while they sleep, and to see if they can grow or gain weight better if they are sleeping better at night.

PI: Shruti Paranjape, MD
CONTACT: Shruti Paranjape, MD  at 410-955-2795
PROTOCOL: NA_00089087
SEND ME INFO