A clinical trial is a research study that involves human volunteers with specific health conditions. The trial evaluates a proposed drug or therapy in a controlled environment to determine its safety and effectiveness in treating patients with certain conditions. Patients must give their “consent” to participate in a clinical trial, and they should do so only after fully understanding the potential benefits and risks involved.
In the majority of clinical trials, one group of patients will be given a new investigational drug that has shown positive indications in previous phases of testing, while patients known as the control group are given the current accepted standard-of-care treatment. Comparing the two groups allows researchers to evaluate the effectiveness of the study drug or therapy. In "blind" or "masked" studies, patients and sometimes the study staff do not know if they are receiving the experimental or standard treatment.
A placebo is a pill or other substance that has no therapeutic effect. Many people think that clinical trials involve placebos, but the fact is that only a small fraction of cancer clinical trials use them. 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.
Early access to treatments. By participating in a clinical trial, you can gain early access to promising new drugs and other treatments, sometimes years before they are widely available to patients.
Consistency of care. Each clinical trial has a detailed protocol, or care plan, that specifies how and when patients are administered their medication and monitored with diagnostic tests. Patients participating in clinical trials interact closely with a dedicated care team. This close monitoring and strict adherence to the treatment protocol ensures the highest consistency of care.
Aggressive treatment options. For patients with late-stage and advanced cancer, and for those who have not responded to standard lines of therapy, clinical trials can offer some of the most aggressive options for treatment. This is especially true for early-phase clinical trials, which are designed to test the newest experimental drugs.
Advancing cancer care and finding a cure. When you participate in a clinical trial, you not just taking an active role in the management of your own care, but you are making a valuable contribution for all patient by helping to advance cancer medicine, improve outcomes, and find a cure.
Risks vary for each clinical trial and should be explored fully with your physician and study coordinator before you consent to participate. Risks may include unpleasant, serious, or life-threatening side effects, and the possibility that the treatment is not effective in treating your cancer. Even if a new approach helps some patients, it might not help you.
For a treatment to become standard (i.e., widely available and used), it must first go through a series of developmental steps, called phases. The early phases of development focus on making sure the experimental treatment is safe. The later phases focus on whether the experimental treatment is more effective than the current standard treatment. The phase you might participate in depends on several factors, including the stage and progression of your cancer and how you have responded to prior therapy.
Phase I Trials
A Phase I trial is designed to evaluate a new investigational agent that has not previously been tested in humans. The trial looks at the best way to administer the new treatment, to determine the dose that can be given safely, and to identify side effects. Typically, only about 15-30 people take part in a Phase I trial.
Phase II Trials
A Phase II trial builds on the results of Phase I testing and is designed to determine whether a new treatment has an effect on a certain cancer. While patients are generally required to be previously untreated, participation in a Phase II trial doesn't usually preclude the patient from getting the standard treatment after they have received the investigational agent or study drug/therapy. Typically, less than 100 people take part in a Phase II trial.
Phase III Trials
If an investigational drug shows activity against a particular cancer in Phase I and Phase II trials, it will often advance to a Phase III clinical trial. At this stage, the safety and effectiveness of the new treatment is compared against the current standard treatment. Because doctors do not yet know which treatment is more effective, study participants are often assigned at random (called randomized) to get either the standard treatment or the new treatment. Phase III trials typically involve larger groups of patients, from hundreds to several thousands, and often occur at multiple sites.
Other Types of Trials
Other types of trials are less common. Phase 0 trials are very small trials that help researchers decide if a new investigational agent should proceed to a Phase I testing. Phase IV trials look at long-term safety and effectiveness and take place after a new drug has been approved by the FDA.
When you participate in a clinical trial, you generally do not have to pay extra out-of-pocket costs for expenses related to the research part of a trial. Every trial is different, but the clinical trial sponsor usually pays for all research-related costs and any special testing. Typically, the patient or his or her insurance company is asked to pay for any routine tests, treatments, or procedures that would be required as part of standard cancer treatment. Before you join a clinical trial, you will receive an informed consent document that spells out exactly what you’ll have to pay for and what you won’t.
The Stephenson Cancer Center has financial counselors who can help you manage any insurance and financial questions related to clinical trials. Your physician or research trial coordinator will be able to connect you with this resource.
Yes. You can leave a clinical trial at any time for any reason.
You will receive a clinical trial “informed consent” from the physician or nurse that should explain the major elements of the trial. Questions you may wish to ask include:
- Why is the trial being done?
- Why do investigators think that the proposed treatment may be better than the standard treatment?
- How long will I be in the trial?
- What kinds of tests and treatments are involved?
- How often will I need to come to the cancer center?
- What are the possible risks?
- What are the possible benefits?
- How will we know if the treatment is working?
- What other treatment alternatives do I have?
- Are there any costs involved in participating in the study?
- Will I receive any compensation for participating in the study?
- What do I do if I need emergency care during the study?
- What are my rights as a participant in the study?
- What will be done with the information gathered from the study?
- Will I be able to get information about the results of the study?
- Who can I call with questions or problems?
If you are currently being treated at the Stephenson Cancer Center, you are encouraged to talk to your physician or nurse about clinical trials opportunities.
You can also call the Clinical Trials Office at 405-271-8777 to find out more information.
Please keep in mind that all clinical trials have specific guidelines about who can participate based on such factors as age, type of disease, medical history and current medical condition.