Frequently Asked Questions About Clinical Trials

A clinical trial is a research study with human volunteers designed to help answer specific health questions. Carefully conducted clinical trials are the safest way to find new treatments that will improve health conditions.

Each clinical trial is different depending on what health problem it’s trying to solve. However, all trials have a clinical team that includes doctors, psychologists, nurses and study coordinators. They check your child’s health at the beginning of the trial, give specific instructions for participating in the trial and monitor your child’s health carefully during the trial.

Side effects are any undesired effects of the study medication. These effects may include headache, nausea, skin irritation or other physical problems.

Based on the focus and goals of the trial, all clinical trials have guidelines about who can participate. There are several factors that allow someone to participate in a clinical trial called "inclusion criteria," while "exclusion criteria" are those that prevent someone from participating. These criteria are based on factors like age, the type and stage of disease, previous treatment history and other medical conditions. The criteria are used to identify the right participants, keep them safe and ensure that researchers are able to answer the trial’s questions.

Before joining a clinical trial, a participant must qualify for the study. Our standard practice is for a study coordinator to have a screening call over the phone with you to figure out whether your child meets the criteria for the study and if the study might benefit your child.

Yes. Our team offers access to more than 300 oncology clinical trials, and you can browse many of these trials on the ClinicalTrials.gov website. You can also learn more about our Experimental Therapeutics Program, which is part of our Center for Cancer and Blood Disorders (CCBD). 

Patients who participate in clinical trials gain access to new research treatments before they are widely available. Often the process of collecting information in the study allows your child’s doctor to find out more about their condition and the effects it has on your child. This may allow them to benefit from better treatment after completing the trial. Many patients and families also feel a sense of satisfaction from knowing they are helping to reduce the suffering of other people with the same condition.

Benefits: Clinical trials that are well-designed and well-executed are the best approach for eligible participants to:

• Gain access to new research treatments before they are widely available.
• Obtain expert medical care during the trial.
• Help others by contributing to medical research.

Risks: There are risks to clinical trials.

• There may be unpleasant, or even serious, side effects to treatment.
• The treatment may not be effective for the participant.
• The study may require more of the patient and family’s time and attention, including trips to the study site, more treatments or complex dosage requirements.

The ethical and legal codes that govern healthcare also apply to clinical trials. In addition, clinical research is federally regulated with built-in safeguards to protect the study participants. All clinical trials follow a carefully controlled protocol, also called a study plan, that details what researchers will do in the study. As a clinical trial progresses, researchers report the results of the trial at scientific meetings, to medical journals and to various governmental agencies. Individual participants’ names will remain anonymous and won’t be mentioned in these reports.

Families should know as much as possible about the clinical trial and feel comfortable asking their child’s care team questions.

Here are some questions to consider discussing with your child’s healthcare team:

• What is the purpose of the study?
• Who is going to be in the study?
• Why do researchers believe the new treatment being tested may be effective?
• Has the treatment been tested before?
• What kinds of tests and treatments are involved?
• How do the possible risks, side effects and benefits in the study compare with my child’s current treatment?
• How might this trial affect my child’s daily life?
• How long will the trial last?
• Will we be reimbursed for other expenses?
• What type of long-term follow up care is part of this study?
• How will we know that the treatment is working?
• Will the results of the trials be provided to me?
• Who will lead my child’s care?

Plan ahead and write down questions you would like to ask before meeting with the doctor. If it would make you more comfortable, ask a friend or relative to come with you for support. It’s also a good idea to bring a notebook or digital device like a phone or laptop for taking notes while meeting with the doctor.

Yes. Most clinical trials provide short-term treatments related to a specific illness or condition, but they don’t replace your child’s primary healthcare needs. Additionally, this ensures that other medications or treatments provided by your child’s primary doctor don’t conflict with the study medication.

Yes. You can leave a clinical trial at any time. When withdrawing from the trial, you should let the research team know and briefly explain your reasons for leaving the study.

Anyone joining a clinical trial in the United States is required to sign an informed consent, or a form indicating that they understand what will happen to them during the study. The doctor will go over the details of the study to help you decide whether your child will participate. You will be given the informed consent document that includes details about the study, such as its purpose, length of time, required procedures and important contacts. Risks and potential benefits are also explained in the informed consent document. Informed consent isn’t a contract; it’s an agreement that you understand the study, and you may withdraw from the trial at any time.

The pharmaceutical company, government research institution or other health organization that funds a clinical trial and designs its protocol.

A protocol is the plan for how to run the clinical trial. The plan is carefully designed to safeguard the health of the participants while answering specific research questions. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications and dosages; and the length of the study.

Every clinical trial in the U.S. must be approved and monitored by an Institutional Review Board (IRB) to make sure the risks are as low as possible and are worth any potential benefits. An IRB is an independent committee of physicians, statisticians, community advocates and people with a diversity of backgrounds and experiences that ensures the clinical trial is ethical and the rights of study participants are protected.

The Food and Drug Administration (FDA) is a government agency that monitors the manufacturing, testing, effectiveness and use of drugs and medical devices. The FDA must first approve a drug before it can be tested on humans in our studies. When the results of the studies are completed, the FDA can then determine the effectiveness of the drug and decide if it should become available to the public.

Clinical trials are conducted in phases. The trials at each phase have a different purpose and help scientists to answer different questions.

• In phase 1 trials, researchers test a new drug in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range and identify any side effects.
• In phase 2 trials, the study drug is given to a larger group of people (100-300) to determine the effectiveness and safety.
• In phase 3 trials, the study drug is given to an even larger group of people (1,000-3,000) to confirm its effectiveness, determine its safety, monitor side effects and compare it to commonly used treatments.
• In phase 4 trials, once a drug is on the market, studies identify any additional information including the drug’s risks, benefits and optimal use.