What are clinical trials?
A clinical trial is a carefully planned research study that involves people. These studies help find new or better ways to diagnose, treat, and prevent diseases.
Clinical trials may test new treatments or other ways to use existing standard treatments. They may collect information to help researchers better understand a disease. Taking part in a clinical trial is your choice. You can decide not to participate or leave the study at any time.
Clinical trials are also called research studies, clinical research, or research protocols.
Purpose of clinical trials
Clinical trials help doctors and researchers learn:
- How well medicines, treatments, or medical devices work
- How different treatments or approaches can be used together
- The safest and most effective dose of medicine or treatment
- Possible risks and side effects
- How a disease develops over time
- Short and long-term effects of a disease and its treatments
There are 2 main types of clinical trials:
- Therapeutic clinical trials test new treatments or ways of treating disease.
- Non-therapeutic clinical trials collect information to help researchers learn more about the disease or its effects. These trials may not directly benefit your child, but what is learned may help others in the future.
Clinical trials may use experimental treatments. These are treatments that have not yet been fully tested or approved by health or regulatory agencies for the purpose that is being studied. In some cases, the treatment may have been approved for other uses and is now being studied for another disease.
An experimental treatment is not part of standard medical care (the widely accepted way of treating a condition). However, a clinical trial may include both standard medical care and experimental treatment.
Safety in clinical trials
Clinical trials must follow steps to help keep participants safe. An Institutional Review Board (IRB) reviews every clinical trial before it starts and oversees it regularly. The IRB is an independent group of doctors, scientists, and other experts. They consider the study's benefits and risks and help protect the health and safety of people who take part. The IRB can approve or reject a study, ask for changes, or stop a trial if needed due to safety or other concerns.
The IRB and medical team work together to monitor safety and help protect participants.
Clinical trials versus standard care
Clinical trials are different from standard medical care. In a clinical trial, researchers follow a specific research plan (called a protocol). They cannot make treatment changes for each participant.
Standard medical treatment is the care a doctor recommends for your child based on their individual needs. An experimental treatment is new and may not yet be fully tested or approved for children. In some cases, it may have already been tested and approved for adults.
Your child’s doctor may recommend a clinical trial if they feel it may benefit your child more than the standard treatment.
Doctors carefully consider the risks and benefits of treatments when choosing the best approach for your child. In some clinical trials, your child might receive both standard medical care and experimental treatment.
Features of a clinical trial
- Only patients who meet certain requirements (eligibility criteria) can participate.
- The study follows a set research plan (called a protocol).
- Treatments may be experimental.
- Benefits and risks are not fully known.
- Patients may receive standard care, experimental treatment, or both.
Features of standard medical care
- Treatments have been tested and widely used by health professionals.
- Treatment is based on your child’s individual needs.
- Benefits and risks are better understood.
- Patients receive standard care only.
Many treatments that are standard care today were first studied in clinical trials. By taking part, patients and families can help researchers learn more about diseases and develop new treatments.
Clinical trial eligibility
People who take part in clinical research are volunteers. To join a clinical trial, patients must meet certain requirements. These are called eligibility criteria or inclusion and exclusion criteria. If your child is eligible, their doctor may talk with you about joining a clinical trial.
Some reasons families choose to take part in clinical research include:
- Trying a new treatment that is not yet available to the public
- Trying a new treatment if the standard treatment may not work well for your child
- Helping researchers learn about a disease or find new treatments
Meeting with the research team
If you are interested in participating in a clinical trial, you will meet with the research team to learn more about the study and what to expect. They will explain the study and answer any questions you have.
You must give permission for your child to join the clinical trial. Before the meeting, it is important that you:
During your meeting with the research team, you should:
- Learn about the clinical trial, including any treatments, tests, procedures, benefits, risks, and side effects, and how it differs from the standard of care
- Receive a written copy of this information
- Learn about other treatment options
- Make sure you understand the information
- Ask any questions you have and get clear answers
Be sure to ask all your questions – your research team is there to help you understand every part of the study. It may help to bring someone with you to take notes. If you do not understand something, ask the team to explain it in simple, clear words in a way that you can understand.
Patient advocates are people who can help support patients and families during a clinical trial. They are not part of the research study team but can talk with you about questions, concerns, or problems related to the study. They can help you find information and may help you share your concerns with the research team or the Institutional Review Board (IRB).
Informed consent is the process of learning about a clinical trial, understanding what to expect, and deciding whether to take part. Depending on your child’s age and understanding, they may be asked to give their agreement (called assent) to take part in the study.
It is your choice whether to participate. You can decide not to join a clinical trial. You can also stop or take your child out of the trial at any time, for any reason.
If your child is 18 or older, they can decide for themselves whether to participate or leave a trial.
If you are thinking about stopping a clinical trial, talk to your child's doctor about other treatment options first.
There are several ways to find clinical trials that may be an option for your child. Your child’s doctor or care team is usually the best place to start. They know your child’s medical history and can help identify clinical trials that may be a good fit.
Many hospitals and research centers offer clinical trials. You can also learn more about clinical trials through trusted websites, such as clinicaltrials.gov or options recommended by your care team.
Search open clinical trials at St Jude Children’s Research Hospital by visiting clinical trials at St. Jude.
Deciding whether to take part in a clinical trial is a personal choice. You can choose not to join or leave the trial at any time. Take time to learn about the study, understand treatment options, and ask questions to know what may be right for your child.
Questions to ask your research team
- What is a clinical trial in simple terms?
- What kind of clinical trial is this?
- Why is my child a good candidate for this clinical trial?
- What are the other treatment options for my child’s condition?
- What are the possible risks, side effects, or discomforts of this study?
- How long will the research study last?
- What will happen if side effects or problems occur?
- How will my child’s safety and privacy be protected?
- Who can I contact if I have questions or concerns about the study?
Find more questions to ask your research team about clinical trials.
