There is a lot to consider before your child participates in a clinical trial. We’ve put together answers to some of the most frequently asked questions regarding children in clinical research. If you have additional questions, we invite you to contact us or visit the National Institutes of Health’s “NIH Clinical Research Trials and You.”
What is a clinical trial?
Clinical research that evaluates a treatment or intervention are referred to as clinical trials. Clinical trials can be found at both the private and public level for children and adults. These studies provide researchers with data necessary to create treatments and interventions that can be used to combat diseases and improve human health. It’s important to note that clinical trials should not be considered the same as individualized medical care by a provider. In a clinical trial, patients follow a pre-determined plan of care and may or may not receive benefits from whatever is being tested.
Why are you performing clinical trials in children?
Children are not just small adults. They are a vulnerable and unique population that requires special consideration in healthcare and in research. Children differ developmentally and physically from adults; however, the majority of medications given to hospitalized children is prescribed off-label, which means that it has not been studied in children. Their organs and systems are in different stages of development, and may work differently and process things differently. Performing clinical trials in pediatric populations addresses this gap and helps develop new therapies and interventions to give to children in need.
Should my child participate? Is it safe?
How do I find a clinical trial for my child?
It is always the choice of the patient and their family whether to enroll in a study. Participation is voluntary and you can change your mind at any time. Your decision not to enroll in a study does not and will not impact the clinical care you or your child receives at UNC.
Many guidelines and measures are in place to protect people participating in clinical trials, with particular attention being given to children as members of a vulnerable population. The health of each participant is a top priority and is carefully protected throughout the duration of the study.
Clinical trials must be approved by an appropriate institutional or federal Institutional Review Board (IRB) before participants can enroll. An IRB is composed of a team of medical professionals who perform critical oversight of the research in the study, in order to ensure the clinical trial is safe for participants as well as scientifically relevant. The Office of Human Research Ethics (OHRE) oversees all IRBs at UNC and is responsible for ethical and regulatory oversight of research at UNC-Chapel Hill that involves human subjects.
What do different phases of a study mean?
- Research for Me: Learn about clinical research and find or volunteer for different research studies at UNC.
- Clinical Trials.Gov: ClinicalTrials.gov is a database of privately and publicly funded clinical studies conducted around the world maintained by the U.S. National Library of Medicine.
What is the difference between a blind study and an open study?
Clinical trials are conducted in a series of phases, or steps, to test the efficacy of the treatment or intervention in different ways. The definitions below are courtesy of the NIH’s “NIH Clinical Research Trials and You
- Phase I: Researchers test a drug or treatment in a small group of people (20–80) for the first time. The purpose is to study the drug or treatment to learn about safety and identify side effects.
- Phase II: The new drug or treatment is given to a larger group of people (100–300) to determine its effectiveness and to further study its safety.
- Phase III: The new drug or treatment is given to large groups of people (1,000–3,000) to confirm its effectiveness, monitor side effects, compare it with standard or similar treatments, and collect information that will allow the new drug or treatment to be used safely.
- Phase IV: After a drug is approved by the FDA and made available to the public, researchers track its safety in the general population, seeking more information about a drug or treatment’s benefits, and optimal use.
Blinding refers to a procedure in which one or more parties in a clinical trial are kept unaware of the treatment assignments. In a single-blind study, the subjects are unaware of the treatment assignments, while in a double-blind study, the subjects, investigators, and study personnel are unaware of the treatment assignments. An open or open label study has no blinding of the subject or the study staff.