Clinical Trials at the CU Cancer Center

Clinical trials offer patients the most cutting-edge and promising treatments to treat and prevent cancer.

Many advances in medicine are the result of clinical trials — studies of new medications or treatments in humans before they are approved for widespread use.

Clinical trials are the final step in a yearslong process that begins with laboratory research and animal models. Many treatments used today are the result of past clinical trials. Clinical cancer trials are designed to answer questions about new treatments or new ways of using old treatments and how well they work. These trials may test drugs or vaccines, ways to perform surgery or deliver radiation therapy, or combinations of many treatments. A doctor may offer patients the option to join a clinical trial as part of their treatment plan.

Clinical cancer trials follow very strict rules, called protocols, that are overseen by a governing scientific and ethical body. The protocol determines who can participate in the trial and help makes sure the trial is safe and has accurate and meaningful results.

What is a clinical trial?

A clinical research study, often known as a clinical trial, is a way to find answers to difficult scientific or health questions. For example, clinical trials can help us learn how best to screen for cancer so the disease can be diagnosed earlier, when the cancer is more likely to be treatable. Results from these studies can lead to the approval of new ways to detect and treat diseases such as cancer. There are no additional costs for patients who choose to take part in a clinical trial.

Clinical trials can range from small, locally based research studies to worldwide efforts that enroll thousands of participants. People who participate might have a specific disease or condition, or they may be healthy volunteers.

What are the phases of a clinical trial? 

Clinical trials are completed in phases. If a new treatment does not pass any phase, then it cannot move to the next. Each phase generally increases the number of people participating and asks different questions.

The goal of Phase 1 clinical trials is to test a treatment’s safety. In this phase, doctors can also learn more about how much to give a patient, what the side effects are, and how the cancer responds to the treatment. Typically, there are anywhere from 10 to 30 people in a Phase 1 clinical trial.

Phase 2 clinical trials determine whether a treatment is effective or not. Sometimes participants are separated into two or more groups. This is done to see how a new therapy compares to the standard of care or to try different amounts of the treatment. Participants in a Phase 2 clinical trial may be asked to keep logs about how they are feeling. These trials typically have fewer than 100 patients.

Phase 3 clinical trials compare the new therapy to the current standard of care, or what is currently being used in clinics to treat the disease. In this phase, the trial evaluates side effects and effectiveness and tries to define whether one treatment works better than the other. In many cases, people on Phase 3 clinical trials are chosen randomly to either be treated with the new therapy or with the standard of care (most cancer clinical trials do not give people a placebo, or sugar pill). The Food and Drug Administration will typically not approve a drug to be used by the general public unless it passes a Phase 3 clinical trial. Phase 3 clinical trials include more than 100 people.

The last phase of clinical trials is Phase 4. During this phase, drugs that are newly approved by the FDA are tested in hundreds or thousands of participants. This allows doctors to collect more information about the treatment over time and in a large group to watch for longer-term side effects.

What do I need to know before participating?

Be sure you understand what happens during the study, the type of health care you will receive, any related costs, and the benefits and risks of participating.

A clinical trial is different from regular medical care. Unlike medical care, when you and your doctors make decisions about the goal of treating your disease or condition the best they can, research seeks to answer a research question. The researchers might not be able to treat you as they would if you were their patient.

In some studies, researchers need to compare healthy volunteers with people who have a specific disease or condition. Research with healthy volunteers is designed to develop new knowledge, not to provide direct benefit to study participants.

Don't be afraid to ask the research team for more information and talk with them about your concerns. It is their job to provide you with the information you need to make your decision.

Why are clinical trials important?

Clinical trials allow doctors and researchers to test treatments that will extend lives and even lead to cures for some forms of cancer. Some patients will benefit from some clinical trials. Other patients understand that their involvement contributes to helping future patients. In these essential tests, it is the bravery and hope of human patients that allows cancer science to truly reach its goal of providing new treatments. 

It is important that people from all backgrounds participate in clinical trials. If a treatment is only tested in one type of person, doctors are unable to determine how the treatment works for all different types of people.

What is an investigator-initiated trial (IIT)?

Investigator-initiated trials are “homegrown” ideas from researchers and investigators who are active in lab and clinical settings. The sponsor investigator of the trial is not only sponsoring the study but also conducting it. This means that they will be in charge of creating, coordinating, and carrying out the IIT. This may be done alone or with a team supporting the sponsor investigator.