What is a clinical research study?
A clinical research study, also called a clinical trial, looks at an investigational drug or medical device to see if it is safe, how it works in the body, and if it works to treat a specific disease. The term “investigational” means that the drug or medical device being studied for use in a condition or disease has not been approved by the country-specific regulatory health authority, such as the Food and Drug Administration (FDA) in the United States. Clinical research studies are conducted by doctors who are responsible for the study participants’ study-related care. In most countries, the regulatory health authority requires that several phases of clinical research be performed to better understand the safety and effects of new investigational drugs and certain medical devices.
Why are clinical research studies needed in infants and children?
Biologically, children are not simply small adults. An investigational medication may work differently in children compared to adults, and some formulations may be better suited for children. Also, some conditions affect only children. As a result, it is important for researchers to understand what the safest and most effective dose of an investigational medication is for children at all ages.
What should study participants expect?
Before participating in a study, a detailed description of the study, as well as possible risks and benefits, will be provided in writing in an “informed consent form” and discussed with you or your child.
Parents will be asked to review and sign the parental or legal guardian informed consent form prior to participating.
Each participant’s medical history will be reviewed, and he or she will be given a study-related physical exam and laboratory tests.
Throughout the study, participants and their parents or legal guardians should feel free to discuss the child’s care with the study doctor or research staff members at any time. Participants and their parents or legal guardians will also have the right to withdraw from the study at any time, for any reason.