Clinical trials for children and teens

Clinical trials are conducted worldwide under rigorous guidelines to help doctors find out whether new cancer treatments are safe and effective or better than the standard treatment. 

Before a trial begins, a new therapy is often developed and tested in a laboratory. It's then thoroughly tested in animals. If this early research (the preclinical testing) shows promise that the therapy may be safe and effective, a carefully planned and monitored clinical trial of the drug or treatment will be conducted in people. 

Doctors who specialize in cancer research develop and lead cancer clinical trials. They determine the study's protocol (procedures and design), including: 

• The disease to be treated
• The treatment to be tested
• The study goal(s) (sometimes called endpoints)
• The type of patient who will be an appropriate participant
• The methods to be used to protect the participants' safety
• How much medicine or other treatment will be given to patients
• How long the treatment will be studied 

A trial can take place at a few specific locations or be conducted from many venues across the United States. In many cases, participants can be treated at various locations, which may include a large cancer center, a university hospital, a clinic, a local medical center, or the doctor's office. 

A cancer clinical trial is divided into four parts, called phases, each with a specific purpose. As each phase is successfully completed, the trial moves into the next phase. 

Phase I 

Treatment is tested in a very small group of patients to determine: 

• Its safety
• The appropriate dose (amount)
• The best way to administer the treatment 

Researchers watch patients closely for possible side effects. 

Phase II 

Treatment is tested in a larger group of patients to determine: 

• Whether the treatment works
• How well the treatment works 

Researchers continue to monitor patient safety in Phase II and throughout the trial. Phase II studies with positive results will move into Phase III.

Phase III 

Phase III trials are "randomized." This means a "reatment group" is compared to a "control group." In a randomized trial: 

• The treatment group is made up of large numbers of patients who receive the study treatment
• The control group is made up of large numbers of patients being treated with the best standard treatment
• The treatment outcomes for the two groups are compared at specific time intervals 

The FDA approves a treatment if it passes Phase III testing. The treatment must meet safety requirements, and either be more effective than standard treatment or equally as effective as standard treatment but with less toxic side effects. 

Phase IV 

By the time a clinical trial enters phase IV, the FDA has already approved the treatment. Phase IV studies are often performed to: 

• Identify an additional use for an already approved drug or other treatment
• Gather additional information on safety and effectiveness from a larger group of patients
• Establish effectiveness in a subgroup of patients—for example, patients over age 65 

A clinical trial team is made up of doctors, nurses, social workers, and other healthcare professionals. The team members: 

• Check each participant's health at the beginning of the trial
• Give specific instructions for taking part in the trial
• Monitor each participant's health throughout the trial
• In some cases, follow up with patients after the trial is over  

Your regular doctor or healthcare provider coordinates with the research team to ensure that other ongoing drugs or treatments you're receiving won't interfere with the study treatment.

U.S. clinical trials are designed to give patients the safest, potentially most effective clinical therapies. Clinical trials are conducted once researchers have shown in the laboratory and in animal research that a particular study treatment has a good chance of offering better outcomes for people with a specific disease. 

Patients enrolled in cancer clinical trials are never treated as “guinea pigs.” In fact, patients are given either: 

• The best treatment currently available
• A new and possibly more effective therapy 

Patients in clinical trials are watched closely by their doctor, and other members of their medical team to ensure their safety. Every trial has a precise treatment plan called a “protocol,” which must be followed. Patients receive a lot of attention and get excellent cancer care. The trial can be changed or stopped if there is a problem. Patients who take part in a clinical trial also have the option to leave the trial at any time. 

In a cancer clinical trial, placebos (simple pills with no medication in them, often called “sugar pills”) are not used in place of a proven effective therapy. 

If you're interested in the possibility of a clinical trial as a treatment option, talk with your doctor first. They can help you find an eligible trial.

Watch our video series, Communicating With Your Doctor and Talking About Clinical Trials.

Learn more about communicating with your blood cancer specialist or find a list of suggested questions to ask your healthcare providers.  

You can also contact one of our Clinical Trial Nurse Navigators for help in finding an appropriate clinical trial. We can help you identify the information you need about your diagnosis and treatment history to determine eligible trials. We can also help you develop a list of questions to ask your doctor or the trial team about participating in a trial. 

If your doctor agrees that a clinical trial is a good option, they can contact the trial team, who will ask many questions related to your medical and treatment history to determine if the trial is right for you. 

When you contact a member of a trial team, ask to speak with the study coordinator, the referral coordinator, or the protocol assistant. 

The study coordinator answers your and your doctor's questions. They will make a preliminary assessment of your eligibility for the trial. After your initial appointment with the coordinator, they decide whether you'll be accepted to participate in the clinical trial. 

At times, you may need to contact a trial team yourself about participating if your doctor is unable to. If this is the case, one of our Clinical Trial Nurse Navigators can help guide you and suggest what to ask the trial team. 

Informed consent process 

When you first express interest in a clinical trial, a process called informed consent begins. Informed consent is the term used to describe the ongoing sharing of information by the trial team before, during, and after the clinical trial. 

To start the process, the clinical trial team gives you an informed consent document with detailed written information about the trial, and the doctors and nurses involved in the trial can explain the study to help you decide if you want to participate. You'll be encouraged to ask questions so that you have all the information you need to make a decision about participating. (A language interpreter is provided for people who request one.) 

You will be given time to review the clinical trial details in the informed consent document. 

Once you and the trial team are satisfied that you understand the clinical trial protocol (procedures), you must agree to sign an informed consent document before you can begin. It affirms that you fully understand the nature of the study. The informed consent document is not a contract; you're free to leave the study if any new information leads you to want to do so. In fact, you're free to leave the study at any time, for any reason. 

New information may become available to the research team as the trial goes along. The clinical trial protocol may also change over time. The informed consent process requires that members of the research team update you as such changes are made. You may also be asked to sign a new informed consent document.

Once you've identified a clinical trial you may want to join, you'll need to consider how your care will be paid for. Insurance coverage for clinical trials varies depending on the treatment, the insurance company, and the health insurance policy. The study sponsor may cover some of the clinical trial costs. Most studies provide the drug or treatment free of charge. Other costs may or may not be covered by health insurance plans. 

Speak with the clinical trial sponsor and your insurance provider to learn who'll be responsible for costs during the study's duration. Find out whether your treatment costs will be paid by the clinical trial sponsor, your insurance provider, or by you, especially if you're uninsured or don't have adequate medical insurance. 

Generally, the study sponsor covers the costs of: 

• The research doctors' and nurses' time
• The treatment being studied

Routine costs that you'd have whether you were in a clinical trial or not may not be covered by the sponsor or insurer.  

These costs can include:

• Doctor's visits
• Hospitalizations
• Laboratory tests
• Drugs that aren't part of the study design 

Try these strategies to determine the costs you'll be responsible for: 

• Talk with your doctor, nurse, social worker, or the study contact person to find out whether the study drug is provided free of charge or low-cost from the drug company.
• Ask your healthcare providers and your insurance representative to tell you which expenses are covered by your insurance plan.
• If your insurance company won't pay treatment costs or denies your claims, contact the drug manufacturer or advocacy groups for assistance. Your healthcare provider can contact them as well. Organizations that may have information and suggestions to help you appeal denied insurance claims include:
• The National Coalition for Cancer Survivorship, (888) 650-9127
• The Patient Advocate Foundation, (800) 532-5274
• Ask your own doctor or the research study contact to send information to your insurance company about the benefits of the study. They may also point out to your insurer that other companies have paid for such treatment. You may need to provide more information once the study begins.
• If you have Medicare, find out what clinical trial costs are covered. If you're enrolled in a federally funded clinical trial, Medicare pays for routine care costs (such as office visits and regular medical tests). Learn more about this coverage online at Centers for Medicare and Medicaid Service website or call (800) 633-4227. 

Another type of study that blood cancer patients and their relatives can participate in is a nontherapeutic study. Nontherapeutic studies aren't considered clinical trials because they don't involve the study of new diagnostics or treatments for diseases. Instead, they're developed to gain a better understanding of disease-incidence patterns or the consequences of specific treatments for a specific disease. 

In all cases, you should be asked to sign a consent form that defines the study's purpose and what's expected from the participants. 

If you'd like to consider taking part in a nontherapeutic disease-specific study, talk with your doctor about the benefits of participating. 

We provide a list of nontherapeutic studies that have statements of compliance with Health Insurance Portability and Accountability Act (HIPAA) regulations and that require informed consent from participants. The information provided on specific studies doesn't represent a Blood Cancer United endorsement of a particular study, group, company, health institution, or product.  

Disease registries 

Another type of study you can participate in is a disease-specific registry. Disease-specific registries are not clinical trials. Instead, they are observational studies that follow patterns in disease diagnosis, treatment, and/or survival patterns over time. Unlike clinical trials, they don't involve the study of new diagnostics or treatments for diseases.  

Disease registries follow patterns of medical history, diagnostics, and/or treatment in real practice settings in a large number of patients (with a specific disease) and observe outcome and/or survival. Participation in some disease- specific registries is available through a doctor's office, while other registries are offered directly to patients. In either case, it's important to discuss with your doctor the benefits of participating and your intent to participate in a disease-specific registry.  

In all cases, you should be asked to sign a consent form that defines the purpose of the study and what is expected from the participants. 

We provide information about disease registries that have statements of compliance with Health Insurance Portability and Accountability Act (HIPAA) regulations and that require informed consent from participants. The information provided on specific registries does not represent a Blood Cancer United endorsement of a particular registry, group, company, or product.

Visit the disease registries page to access disease registries and nontherapeutic studies.