OPTIMIZing Treatment for Early Pseudomonas Aeruginosa Infection in Cystic Fibrosis

Clinical Trials

Offered by: Nemours Children's
Location: Jacksonville, FL

Trial Name

OPTIMIZing Treatment for Early Pseudomonas Aeruginosa Infection in Cystic Fibrosis

What is the trial about?

People with Cystic Fibrosis (CF) often have thick mucus that is  a good environment for bacteria (germs) to live and grow, rapidly leading to infections. One bacteria, Pseudomonas aeruginosa (Pa) is particularly good at living in the CF lung. We want to find out what treatment(s) work best for clearing up Pa.

Who can participate?

People with CF who are between the ages of 6 months and 18 years of age and have recently had a lab result (culture) showing the presence of Pa in the throat or lungs for the first time or after at least two years of negative cultures. Several tests and procedures would be completed at the first study visit to see if it is safe to be treated with the study medicines.

What is involved?

If you join the study, you would have some tests and exams over an 18-month period. These tests and exams help us find out if being in this study causes any effects that are important to know about. We use them to check on the safety of people in the study. We also use them to learn if an experimental treatment is helping or not.

During the first visit, you will be assigned to one of two groups. One group will take azithromycin orally (by mouth) three times per week for 18 months. The amount you take will depend on how much you weigh. The other group will take a placebo orally three times per week for 18 months. A placebo is a pill or liquid with no active medicine but looks just like the active medication. The amount you take will depend on how much you weigh. The group assignment is done by chance. It is similar to flipping a coin. You will not know which group you are in. Also, the CF research team members will not know which group you are in.

Both groups will get a 28-day course of TIS (tobramycin inhalation solution, 300 mg/5mL (TOBI®)). You will take it two times a day for the first 28 days of the study. After that, you will be prescribed TIS only if Pa is found in your throat or lung sample taken at a study visit.

Although there are other types of tobramycin for inhalation, you will be encouraged to use TIS while participating in this research study. If you are using another type of tobramycin for inhalation at the start of this study you would complete that cycle of treatment and then be asked to switch to TIS if you need to have treatment because Pa is found in your throat or lung sample.