An oncology clinical trial is a research study that tests new methods of screening, preventing, diagnosing, or treating cancer. These investigations may focus on: the effectiveness of new treatments; new ways of administering approved treatments; new combinations of approved treatments, new surgical techniques, devices, or biological products.

Clinical trials must be conducted before a new drug, biologic, or device may be marketed in the United States. The U.S. Food and Drug Administration (FDA) regulates clinical trials by giving researchers permission to test a new treatment or device under strict regulatory conditions. Once given permission, trials must follow strict rules set by the FDA. These rules make sure that patients who agree to participate are treated as safely as possible.

Prior to enrolling in an oncology clinical trial, qualifying patients are provided with counseling called Informed Consent. This is designed to provide detailed information about:

• Exactly what the trial is studying (the protocol)
• Description of the treatments, if any, that will be administered
• Risks, side effects, and benefits
• What is required of the patient (procedures, time, information, etc.)
• The rights of the patient before, during, and after enrolling in the trial

By the time an oncology investigation reaches the clinical trial stage, it has gone through a rigorous process in a lab. Trials are not just available for patients with progressive disease; there are trials available for all types and stages of pediatric cancer. 

For more information about Informed Consent, visit the FDA’s website.

A protocol describes the rules to follow during the research. For example, a protocol states why it is important to study the research question and how the research team will answer the research question. A protocol also states how the research team will protect the health and wellbeing of the research volunteers. A protocol includes information on the study procedures, medications to be used, possible risks and benefits, possible adverse events, and data analysis methods. 

Oncology clinical trials are conducted for many reasons, such as:

• Determining whether a new treatment or device is safe and effective for people to use.
• Studying different ways to use standard treatments or currently approved treatments so they will be more effective, easier to use, or reduce side effects.
• Asking a question that hasn't been answered yet such as, "Does a certain medicine cure a disease, or make some of the symptoms go away?"
• Learning how to safely use a treatment in a population for which the treatment was not previously tested, such as children or adults.

There are 3 main types of research studies: Interventional, Observational, and Expanded Access.

• Interventional studies are Clinical Trials in which an intervention/treatment is being investigated to be properly tested before becoming publicly available.
• Observational studies assess investigations to look for health outcomes rather than providing treatment.
• Expanded Access (also called Compassionate Use) is a way for patients with advancing conditions to receive treatment from a clinical trial despite being ineligible to enroll in the trial.

Oncology clinical trials are organized by five types of phases. Classification depends on the purpose of the study and determines the total number of patients who may receive treatment on that clinical trial.

• Phase 0 usually has less than 15 patients participating. After rigorous testing in the lab, this phase investigates whether the treatment is safe in human subjects.
• Phase 1 has 20-80 patients participating and is determining the highest dose that can be given without serious side effects. The FDA estimates that 70% of these trials make it to the next phase.
• Phase 2 trials have more than 100 patients and are now monitoring the treatment’s effectiveness, side effects, and fine-tuning methods in anticipation of phase 3. According to the FDA, about 33% of these trials make it to phase 3.
• Phase 3 trials are usually very large– up to 3,000 patients participating. Now that the safety and effectiveness have been established, this phase investigates how this new treatment compares to existing treatment options. This is usually done through a double-blind randomization process, which means the investigator and the patient do not know which treatment they are receiving. If the results from this phase are successful, the FDA typically approves the treatment and it becomes widely available.
• Phase 4 trials are also very large and span many years. This is a monitoring phase after FDA approval has been given to track long-term safety, effectiveness, and other relevant data.

Placebos are rarely used in pediatric oncology clinical trials. If a placebo is part of the trial you’re considering, you will be fully informed of that fact ahead of time. You would only be considered for such a trial when it’s safe and medically appropriate. A placebo in cancer treatment would only be used if there is no standard treatment for that diagnosis.

Clinical trials can be sponsored by organizations (such as a pharmaceutical company), Federal offices and agencies (such as the National Institutes of Health or the U.S. Department of Veterans Affairs), or individuals (such as doctors or healthcare providers). The sponsor determines the location(s) of the trials, which are usually conducted at universities, medical centers, clinics, hospitals, and other federally or industry-funded research sites.

All clinical trials have guidelines called eligibility criteria. These may include age, gender, the type and stage of your disease, previous treatment history, and other medical conditions. Following eligibility criteria helps keep patients safe and ensures that researchers learn the information they need. Your child’s medical team or a Clinical Trials Navigator can help find trials for your child that match with eligibility criteria.

There are many resources available for explaining clinical trials to children, but that can take on different meaning when trying to explain to your own child the potential of enrolling in an oncology clinical trial.

Your child’s doctor, nurse, or social worker is a great source of support and ideas for how to explain this process. These resources may be useful, too:

• https://www.nih.gov/health-information/nih-clinical-research-trials-you/parents-children
• https://www.webmd.com/a-to-z-guides/video/clinical-trials-explained

The following risk factors are standard across any treatment protocol and clinical trial, and each trial will indicate specific risk factors in their Informed Consent paperwork.

• The treatment might not work.
• The treatment might cause serious side effects.
• Even if a new approach helps some patients, it may not help all patients.

• Access to new drugs and other treatments, sometimes years before they are widely available.
• Close monitoring for any side effects and more acute care.
• Making a valuable contribution to childhood cancer research.
• The chance to take an active role in your child’s healthcare.

Every clinical trial 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.

The FDA has regulations and guidelines for clinical research to protect participants from unreasonable risks. Although efforts are made to control the risks to participants, some may be unavoidable because the treatment is still under investigation.

Patients do not have to pay to participate in an oncology clinical trial, but there may be out-of-pocket costs associated with participating in the trial. These costs may include traveling to the trial and insurance deductibles and copays.

Oncology clinical trials do not pay participants, but may cover the costs directly associated with getting to the trial site like travel, parking, meals, lodging, etc. Each trial is different, so this is an important question to ask of any trial you are considering.

Insurance companies are required by the Affordable Care Act to cover the same routine treatments, procedures, and services that would be covered under standard treatment.  

The trial coordinator can help clarify what the out-of-pocket costs would be under the trial, and works with the oncology social worker and a financial counselor at the hospital to help reduce those costs.

• What are the risks?
• What is involved and required?
• Will I be able to afford the out-of-pocket costs associated with the trial?
• What impact will participating in this trial have on my immediate family?
• How can I end my participation if I change my mind?
• What will happen when the study is over? Will I be told the results?
• Whom do I contact to express concerns or obtain information?

A clinical trial can sometimes require more time and medical attention than normal care. This may include doctor visits, phone calls, more treatments, a hospital stay, or a more complicated treatment regimen.

Informed consent is the process of learning about the clinical trial before you decide to take part in it. The research team running the trial will explain the trial’s purpose, how long it will take, what will happen, all potential risks and benefits, and information on the privacy of your child’s medical records. If you decide to participate, you will sign a consent form that agrees to all of the information that has been discussed with the research team. A copy of the form will be provided so you can refer to it at any time.

Even after you have signed the consent form, you still have the right to leave the study at any time and for any reason. Informed consent continues throughout the trial. This will give the research team an opportunity to update you on the progress of the trial as well as any side effects or other risks that have been identified.

In the U.S., the age of informed consent is 18, so children who are old enough to understand are asked to give assent. The age may vary, but generally, starting around age 7, children are asked to agree to the therapeutic treatment that will be given to them. This is not required by law, but many trials require it for ethical purposes.

Children may also dissent (disagree), so parents and researchers may need to talk through the reasons the child does not agree. Doctors and parents need to work together to help the child understand what a study involves. The researcher will consider your child's age and maturity level when presenting information. This may take time and numerous meetings to determine if the child has a clear understanding of what the trial involves.

Both of the child’s parents much give permission in order to participate in the trial. Exceptions to this are if one of the parents has died, is unknown, is legally incompetent, or does not have custodial rights.

Sometimes assent is not required. In situations where the research may be the only way to help a patient with a life-threatening illnesses, assent is not required if the research is expected to be beneficial to the individual. Even so, researchers are encouraged to discuss the research with the individual patient.

• You have the right to not take part in a research study.
• You have the right to drop out at any time.
• You have the right to be given new information about the study.
• You have the right to ask questions at any time and have them answered as soon as possible.

You also have the responsibility to stay informed during your participation in a study. You should ask questions about anything you do not understand or simply want to know.

Participation in clinical trials is completely voluntary. You may remove your child from the clinical trial at any time.