Cystic Fibrosis Center

Research

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 CFF and Genentech (ESCF). 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 Nancy Jenks at (585) 275-8580 or email nancy_jenks@urmc.rochester.edu.

CF Registry

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.

Age: any

Visits: information taken from clinic visits

Twin/Sibling CF Study

This research is being done to find the genes and other factors that are responsible for variation among persons with cystic fibrosis.  You and a sibling (or two or more of your children) have CF, which involves lung disease, problems with digestion, abnormal perspiration, and a shortened life span.  The underlying cause of CF is changes in a gene called CFTR, but even individuals with the same change in the same family may have differences in the severity of lung, sinus, liver, or intestinal problems.  This study is attempting to understand what other genes and other factors may be responsible for the variation or similarity of illness between you and your siblings or between your children.

Age: any age siblings

One time blood draw from entire family

iCARE

We are trying to learn what tolls, data, and training are most useful to clinicians to help them communicate better with their cystic fibrosis patients and to improve the patients' management of their medications. Your care team may be able to give you more personalized advice about how to take care of your CF. You may learn new ways to take care of your health.

Age: 11-20

Visits: completed during regular clinic visits

VX-809 Alone and in Combination with VX-770

Vertex is developing two drugs, VX-809 and VX-770, as a possible treatement for people with cystic fibrosis. VX-809 and VX-770 are experimental drugs, which means that they have not been approved by the United States Food and Drug Administration (FDA), or by any other regulary agencies for sale or use by the public. This study is being done to learn more about the safety, tolerability, changes in study drug concentration in your blood, and the effect of VX-809 when first given alone and then given in combination with VX-770 to subjects with CF.

Age: 18 and older

Visits: completed in the Clinical Research Center or in the Pulmonary Clinic

Connect with Golisano Children's Hospital

Golisano Children's Hospital Development

What is Cystic Fibrosis?

Cystic fibrosis (CF) is a chronic condition in children and adults. It most often affects the lungs and digestive system of the body. Children with CF may have chronic lung infections and/or poor digestion of their food. Symptoms which are sometimes seen in CF but are also common in other illnesses may include:

  • A chronic cough
  • Frequent infections in the lungs
  • Wheezing/asthma
  • Nasal polyps
  • Difficulty gaining weight
  • Salty taste of the skin