The members of our CF team are actively involved in research that evaluates better treatments for CF. We are collaborative investigators on two national epidemiologic databases sponsored by the Cystic Fibrosis Foundation (CFF). Data is collected and analyzed to better understand the natural history, progression, and results of treatment for CF.
Below is a listing of the research currently underway for our Cystic Fibrosis population. To learn more, call our Research Coordinator, Barbara Johnson, R.N. at (585) 276-4123 or email Barbara_Johnson@URMC.Rochester.edu.
The Patient Registry was established in 1966 as a means to monitor important trends in the CF population and to improve understanding, treatment, and survival. The CF Patient Registry is used by both clinicians and researcher to better understand CF and to improve care of individuals with CF. Each year the data in the Patient Registry is analyzed and an annual report of CF health trends is created. Using this information, CF clinicians can look at nutritional status, infection control, pulmonary treatment and/or metabolic issues quickly. The Patient Registry also has played an important role in directing clinical care in the design of clinical research studies. Researchers may request information from the CF Patient Registry through the CF Registry Committee. This committee evaluates the scientific merit of data requests for epidemiological studies often from respected scholars who specialize in treating people with CF.
Visits: information taken from clinic visits
Fibrosing Colonopathy (FC)
The first reports of FC in the 1990's were linked to the use of high-dose pancreatic enzyme supplements. Dosing guidelines were put in place and the use of high-dose supplements was stopped. Although rare, FC still occurs in some patients with CF. It is important to monitor the use of pancreatic enzyme supplements and other standard treatments in patients to learn more about FC. The FDA has asked manufacturers to study FC patients to see if there is a link between taking pancreatic enzyme medicines and developing FC.
CF-FC (Cystic Fibrosis – Fibrosing Colonopathy)
This is an observational study looking at patients with CF who are treated with pancreatic enzyme replacement therapy. Fibrosing colonopathy (FC) is a health problem that affects the colon and is seen almost only in patients with Cystic Fibrosis. FC is a painful swelling, shortening and fibrosis (scarring) of the colon. Patients with FC may have abdominal pain, diarrhea, rectal bleeding and in some cases, partial or complete blockage of their colon. It is not known exactly what causes FC. The purpose of this study is to determine the number of patients affected by FC and to learn more about what might cause it, including use of pancreatic enzyme supplements.
This study is looking at patients with CF who are experiencing a pulmonary exacerbation and will be treated with antibiotics by their doctor. Doctors and CF patients have questioned about the best way to treat pulmonary exacerbations and to make sure the antibiotics given are taken long enough to get better, but not taken for too long which expose patients to unnecessary risks. This study is evaluating the optimal length of IV antibiotics (between 10 and 21 days) for a patient with an exacerbation. This study is currently enrolling patients. Study visits are during the time you are being treated for your pulmonary exacerbation. If you are interested in participating, please speak to your provider for more information.
Age: 18 and older
TEACH: Testing the Effect of Adding Oral Azithromycin to Inhaled Tobramycin in People with CF
People with CF commonly get chronic infections in their lungs. Certain bacteria or germs found in the lungs are normally treated with antibiotics, azithromycin and inhaled tobramycin. This study is evaluating if the routine azithromycin taken as an anti-inflammatory and inhaled tobramycin work well if they are taken at the same time. Researchers want to find out if azithromycin reduces the benefit of inhaled tobramycin or not. This study is currently enrolling patients. Study visits are scheduled once a month for up to 5 months. If you are interested in participating in this study, please speak to your provider for more information.
Age: 18 and older
The PROMISE study aims to answer questions about cystic fibrosis (CF). CF affects the body’s ability to move salt (chloride) and fluids in and out of the cells of the lungs, intestines, pancreas, sweat glands and other organs.
In the PROMISE study, we will collect health information and specimens from people with CF before and after they start a new FDA approved Vertex triple combination modulator therapy.
Ivacaftor works by opening the “gates” of the CFTR protein to help those gates to stay open longer. Tezacaftor works by allowing more CFTR protein “gates” to reach the right place in the cell to work effectively. Tezacaftor with ivacaftor is only modestly effective, but when a third drug is added (Vertex triple combination modulator therapy), the combination brings many more gates to the right place in the cell. The combination of these 3 drugs allows many more chloride ions to move into and out of the cells. This helps to keep a balance of salt and water in certain organs such as the lungs.
The goal of this study is to see how treatment with a new FDA approved Vertex triple combination modulator therapy might affect people who have CF.
Age: 12 and older