About Clinical Trials

Clinical trials are research studies that involve people. All clinical trial participants are volunteers. Clinical trials are needed to answer specific scientific questions to find new ways to prevent, diagnose or treat cancer. All new advances in cancer care are first tested in a clinical trial. This guide is for patients who may join a cancer clinical trial.

There are many different types of clinical trials. Some clinical trials look at promising new drugs, combinations of drugs, new approaches to surgery or radiation therapy, tailoring therapy using precision medicine approaches, or advances in new areas such as gene therapy.

Cancer clinical trials are part of a long, careful process to develop new ways to prevent, diagnose or treat cancer. This involves many steps and may take many years. For example, researchers test new drugs in the laboratory and animals for many years before the new drug is tested in people.

Your doctor may offer you a clinical trial as a treatment option. You do not need to take part in a clinical trial to receive care at UNC – participation in a trial is voluntary. Use this guide to help make the best decision for you and your family.

Learn more about and find clinical trials at UNC Lineberger

The UNC Lineberger Comprehensive Cancer Center is a cancer research and treatment center at the University of North Carolina at Chapel Hill. The National Institutes of Health (NIH)-designated comprehensive cancer centers are those that deliver excellent care for their patients but also innovative research in order to improve care in the future through trials. UNC Lineberger is the only public comprehensive cancer center in the state of North Carolina. UNC Lineberger is committed to using clinical trials to find better ways to treat cancer.

Learn more about UNC Lineberger

UNC Linebergers‘s most important job is to provide care for patients with cancer. UNC Lineberger protects patients in clinical trials by carefully following a clear plan described in a “protocol.”

A protocol:

• Explains what and how treatments will be given
• Lists medical tests and procedures that patients will receive during the trial
• Gives the number of patients who can take part in the clinical trial
• Lists eligibility criteria, which are the guidelines to decide who may be able to join the clinical trial
• Explains safety information related to the treatment
• Explains how patient information is protected

UNC Lineberger protects patients by using an informed consent process. Informed consent is the process of educating a person about a clinical trial before he or she decides whether to join the trial. During the informed consent process, patients are told what will happen to them if they agree to join the clinical trial. They are also given a chance to ask questions.

All clinical trials are reviewed by UNC’s Institutional Review Board (IRB). The IRB is made up of doctors, nurses, chaplains, social workers, lawyers, and patients. The IRB protects patients by reviewing plans for clinical trials and making sure trials follow federal laws. They also monitor trials to make sure they are conducted in the safest way possible.

• Ask your doctor
• View UNC Lineberger clinical trials database
• Call the UNC Lineberger Clinical Trials Office at (919) 966-4432
• Visit ClinicalTrials.gov, a database of privately and publicly funded clinical studies conducted around the world

Treatment options may include:

• Standard treatment — treatment accepted by medical experts as proper treatment for a certain type of cancer. Standard treatments change over time as doctors learn from experience and research. In some cases, the standard treatment may be no treatment at all. For example, standard treatment may be to watch the cancer and wait to see if it grows. Also, there may not be available standard treatments for you if your cancer comes back after you have received all available standard treatments.
• Clinical trials — many clinical trials are for experimental medicines or treatments that the FDA has not yet approved. However, some trials study treatments that the FDA has already approved. In these trials, doctors may look at new ways to give the treatments, try the treatment in a different type of cancer or study different doses of the treatment.

Both standard treatment and clinical trials may involve:

• Chemotherapy
• Immunotherapy
• Hormonal therapy
• Surgery
• Radiation
• Combined treatments (more than one kind of treatment used together)
• No treatment or delayed treatment

Your prognosis is what may happen with your cancer and how your cancer might respond to treatment. It is important to ask your doctor about your prognosis. This may affect your choices about treatment.

• How might standard treatment affect my prognosis?
• How might the clinical trial affect my prognosis?

A new treatment goes through several phases. Most of the time, when you take part in a clinical trial, you will only be in that one phase of the clinical trials process. Treatments move through the phases, but patients do not. Each phase has a different purpose:

Phase I Trials

• Test if an experimental treatment is safe
• Find the best doses and ways to give the experimental treatment, such as by mouth or by IV infusion
• See what changes the experimental treatment has on the cancer.
• Usually include 15 to 30 patients who are divided into small groups, called cohorts. The first cohort gets a dose of the experimental medicine. Doctors may collect blood or urine samples to measure medicine levels in these patients. If the first cohort does not have any severe side effects, then a new cohort gets a higher dose of the same medicine. The dose increases with each new cohort until doctors find the best dose for future patients. With each increasing dose, doctors test each patient to see if he or she is responding to the treatment. It is not known if the medicine will help treat the cancer.

If doctors find that the treatment is safe, then it may move forward to be studied in a Phase II trial if patients have also benefited from the treatment.

Phase II Trials

• Test if a new treatment works in one type of cancer.
• Fewer than 100 patients usually join a Phase II trial. Even though the main goal is to see if the treatment works, doctors still closely watch patients’ side effects. If the new treatment works, doctors may go on to study it in a Phase III trial.

Phase III Trials

• Test if an experimental treatment is better than a standard treatment. They compare people on standard treatment with those getting the experimental treatment.
• May include hundreds to thousands of patients around the country or world. Each patient enrolled in a Phase III clinical trial has a chance of being in one of the following random, computer-generated groups:
  • Control group – the group that gets the standard treatment
  • Study group – the group that gets the new treatment being tested
• In single blind studies, you do not know whether you are in the control or study group, but the doctor knows. In double blind studies, neither you nor the doctors know which patients are in each group. (In case of an emergency, doctors can find this information in the study file.)
• Doctors do not know if the new treatment is better than the standard treatment. They believe it is as good and may be better.

Phase IV Trials

• Find more information about long-term benefits and side effects and are conducted after the treatment is approved by the FDA.

A placebo is something that looks like medicine, but is not. If a placebo is used, it is given together with the best standard treatment. This allows doctors to compare standard treatment alone to standard treatment with a new medicine. If there isn’t a standard treatment, then the placebo may be given alone, but this is not common in cancer trials.

Each clinical trial has its own benefits and risks. You may benefit from joining a clinical trial in one of the following ways:

• If the new treatment works, you may be one of the first people to benefit
• You may be able to help future cancer patients
• You may receive additional expert medical care

Some possible risks include:

• Expected side effects
• Side effects that the doctor does not expect may occur
• New treatments do not always turn out to be better than, or as good as, standard treatment
• As with standard treatment, the new treatment may not work for you even if it works for other patients

All trials are different. Your health insurance should pay for tests and doctors visits that you would need even if you were not on the trial. The NCI has great resources to explain the difference between patient costs, which are costs that you would pay even if you weren’t in a trial and that are often paid for by insurance and research costs that are related to taking part in a trial and often covered by the clinical trial sponsor. 

Learn more about who pays for clinical trials from the NCI

Ask your patient access specialist to explain how your insurance benefits apply to clinical trials and to learn more about what you might have to pay.

Learn more about patient resources

Not all clinical trials are right for all patients. A trial may be safe for one patient to join but not safe for another. Each protocol has strict rules which doctors must follow to decide who may join the clinical trial. These rules are called eligibility criteria. This protects you from getting treatment that may harm you. Eligibility criteria include information about:

You and Your Overall Health

• Age and gender
• Results of medical tests
• Medicines you take
• Any other health problems

Your Cancer

• Cancer type and stage
• Other treatments you may have had
• How long it has been since you were last treated

If you have found a clinical trial that you think you want to join, talk with your doctor to see if you are eligible to take part.

If your doctor offers you a clinical trial, you will first go through a process called informed consent. The goal of informed consent is to make sure you understand the clinical trial’s plan. The research team reviews the informed consent form with you. This form explains the clinical trial’s purpose, plan, risks and benefits.

This is a great time to ask questions. Try to bring a family member or friend to help you ask questions and write down answers. You may also want to record this meeting so that later you can listen to what was said. Take time to make your decision. You may take the informed consent form home with you to review before signing it.

Look for the following information on your informed consent form:

Treatment

• The reason for the clinical trial (what the doctors hope to learn)
• Who is eligible to take part in the clinical trial
• What is known about the type of treatment being studied
• Possible risks and benefits (based on what is known so far)
• Other treatment options

Tests

• Types of tests
• How often you need tests
• How often you meet with the doctor

Costs

• Who pays for the clinical trial
• If the clinical trial causes you to need more medical care, who pays for those costs

Other

• A statement about conflicts of interest (any direct financial benefit to UNC or your doctor from the sponsor of the trial)
• A statement about how your privacy is protected
• Who to call if you have more questions

If you decide to join the clinical trial, you are asked to sign an informed consent form. The doctor also signs the form and gives you a copy. The informed consent process does not end once you sign the informed consent form. For example, your doctor must tell you if new risks or side effects of the treatment are found during the trial. Ask any questions you may have at any time during the trial.

• What will I need to do if I join this trial?
• Where will I receive treatment? How often will I have to come to UNC? How does this compare with standard treatment?
• How often and for how long will I receive treatment?
• How long will each treatment appointment last?
• How will this clinical trial change or affect my daily activities?
• Can I get in touch with other people who are in this clinical trial?

Your research team will still care for you. In a clinical trial, you will also have a:

Principal Investigator (PI): The PI is usually a doctor. He or she runs the clinical trial and makes sure the health care team follows the plan.

Study Coordinator: The study coordinator teaches you about the trial and collects data from patients on the trial. The study coordinator is a good contact if you have questions during a clinical trial.

Yes, you continue to see your research team, if necessary, for treatment and follow-up care.

All patients in clinical trials are volunteers. You can choose to quit a clinical trial at any time, but talk with your doctor first. Your doctor can tell you how quitting the trial might affect your health and if there are other treatment options. Your doctor can also tell you the safest way to stop taking any clinical trial medicines. Your relationship with your health care team is not changed by your decision.