Anamaria Robles serves as BAPP's Research Coordinator. Please contact Ana with your questions or for more information about any of these studies.

The registry is a database of Cystic Fibrosis patients around the United States. The registry contains information about the patient including demographics, nutrition and PFT's. This registry allows us to have a centralized location for information about our CF patients. It also allows us to compare our center to those around the nation and compare how our CF patients perform relative to the national averages.

Eligible Candidates: Any individual diagnosed with Cystic Fibrosis

A Multi-Center, Double-Blind, Randomized, Efficacy and Safety Study of Denufosol (INS37217) Inhalation Solution in Patients with Mild to Moderate Cystic Fibrosis Lung Disease.

This study will last for about one year and is divided into two parts. In the first part, the patient will either get Denufosol or placebo. A computer will randomly assign the patient to either get study drug or placebo. In the second part of the study every patient will receive study drug. There are ten clinic visits spread throughout the year as well as some phone contacts.

The investigational drug in this research study, Denufosol Tetrasodium (denufosol), turns on a different salt channel than the one that does not work in CF patients. Investigational drug means that it has not been approved by the Food and Drug Administration (FDA) to be sold to treat your child's condition. Denufosol may let liquid into the airway, unclogging mucus and allowing the cilia to work better. The purpose of this research study is to find out if the drug is safe and effective.

Treatment consists of inhaling study drug/placebo three times a day for fifteen minutes over the duration of the study.

Eligible Candidates:

• Be a diagnosed CF patient 5 years old or older.
• Have a forced expiratory volume at one second (FEV1) = 75% of predicted normal.
• Be able to reproducibly perform spirometry maneuvers.
• Be clinically stable for at least 4 weeks prior to screening with no evidence of acute upper or lower respiratory tract infection or current pulmonary exacerbation.

Compensation: $30 stipend per visit $6 Parking $25 for food for visits longer than two hours $50 gas card if you live 50 miles or more from the hospital

This study is attempting to understand what other genes and other factors may be responsible for the difference or similarity of illness between siblings.

The family completes a medical history questionnaire and each CF patient will complete a personal questionnaire. Blood samples are taken from both siblings as well as the parents.

Eligible Candidates:

• Families with 2 or more siblings with CF may join.

The investigators want to find out whether certain DNA mutations affect lung and liver diseases in CF patients. To find out, investigators need to look at the DNA in the blood samples from CF patients.

This is a one-time blood draw requiring 35ml of blood.

Eligible Candidates:

• A CF patient with any combination of the following Two known CFTR Genotype alleles (both CFTR alleles must be identified): DF508, G542X, G551D, R553X, W1282X, N1303K, 3905insT, 1078delT, 621+1G>T, 1717-1G>A, DI507, R560T, S549N, 3659delC, G480C, Any stop mutation (CFTR Genotype ending in "X")
• All 15-28 years old with a consistent baseline FEV1 % predicted value in the upper 20th percentile.

Compensation: $25 Stipend $6 Parking

Anti-microbial Resistance in Sputum Obtained from Patients with Cystic Fibrosis

The purpose of this study is to see if the antibiotics are still effective for certain bacteria that often infect CF patients. To do this, we need to see how well the drugs kill bacteria in CF patients now. We will learn this by looking at mucus samples from current CF patients. Then, we will compare that information to how well the drugs killed bacteria about 10 years ago.

This is a one-time sputum sample.

Eligible Candidates:

• 6 years of age or older
• Documented sweat chloride =%gt; 60 mEq/mL by quantitative pilocarpine iontophoresis test or homozygous for DF508 genetic mutation (or heterozygous for two well-characterized mutations) and one or more clinical features consistent with CF.
• Most recent FEV1 between <= 75% and => 25% predicted when clinically stable; must be obtained at or within 6 months prior to study visit.
• P. aeruginosa present in the most recent sputum or throat culture obtained within 6 months prior to study visit.
• Able to expectorate sputum on a routine basis.

Compensation: $10 Stipend $6 Parking