What is a Clinical Trial?

A clinical trial is a research study that involves people. Many clinical trials are performed to investigate the safety and effectiveness of new medications. Researchers also conduct studies of people with CF to better understand the effects of CF on symptoms and how this could change over time. 

Trials that investigate the use of medications in people are performed in a stepwise fashion. The process of testing new drugs takes several years and is separated into four phases:

Phase I

Phase I clinical trials are designed to determine if a drug is safe for use in humans. These studies test a drug in a small number of people to determine the best method of giving the drug and how much can be given safely.

Phase II

Phase II studies look at both safety and evidence of effectiveness. Determining the best dosing schedule is also part of a Phase II trial.

Phase III

Phase III trials determine if a drug really works. Phase III trials often require hundreds if not thousands of volunteers and often require multiple locations to get enough participants. 

Phase IV

Phase IV trials can be performed before as well as after a drug has been approved for use by the Food and Drug Administration (FDA). These are often large open-label studies investigating the ongoing safety of a drug. This means that all the patients in the study receive the drug; a placebo is not used.

If you are interested in participating in a clinical trial at Johns Hopkins All Children’s Hospital, please contact our Clinical Research Office at 727-767-7247.

What clinical trials do you have?

DRUG DEVELOPMENT: In addition to clinical trials to treat the underlying cause of Cystic Fibrosis, there are also many trials to treat key symptoms of CF in an effort to improve quality of life. The Cystic Fibrosis Foundation provides helpful resources and information about clinical trials, including a database of clinical trials.
 
CLINICAL TRIALS: Check out the registry for clinical trials and results database of public and private clinical studies of human participants conducted around the world.