What Are Clinical Trials?

Clinical trials are research studies that test ways to prevent, detect, and treat cancer and its potential side effects. They may be done to test new treatments or to use treatments we already have in a different way. Years of research are done before a new medicine or other treatment is given to a person to make sure it is a safe treatment that works.

Clinical trials are done in steps called “phases” and in most cases must pass through each phase before moving to the next one.  Clinical trials may also be called clinical studies, research studies, research trials, clinical protocols, and even research protocols.  

Clinical Trial Phases 

  • Phase 0 is not used often and is usually used to speed up the approval process for a new drug. When this phase of a trial is done there is little risk and little benefit to the people in these trials.  

  • Phase I is normally the first phase to test a new drug in humans and involves a small number of people (only a few dozen). Phase I trials are only available at major cancer centers. People in phase I trials are watched very closely. The schedule often includes many appointments (sometimes every day) for blood work and other testing to closely watch for side effects. This phase has the highest risk for the person taking the drug. The benefit of the drug is unknown during this phase and is not the focus of phase I trials, although it is possible some people may see benefit from the treatment.   

  • Phase II looks at how well the new drug works for certain types of cancer. A group of people with the same cancer type participate in these trials. A larger number of people are allowed to participate in this phase, around 25-100. This phase is more accessible.  

  • Phase III trials include more people and are more likely to be offered in more settings and even in different locations worldwide. Some phase III trials compare a new medicine with an inactive medicine, called a placebo or sugar pill. Usually the participants and the doctor do not know if the real treatment or placebo was being given.  

  • Phase IV trials take place after the FDA (Food and Drug Administration) approves a treatment based on the earlier trials. When the treatment approved is a new medicine, the FDA continues to monitor the results and side effects of the treatment over a long period. Phase IV trials may include thousands of patients, and since it is FDA approved, the treatment is available even if you are not involved in the clinical trial.   

  • For more information on the specific types and phases of clinical trials, visit this link

Benefits and risks of participating in a clinical trial

Clinical trials help us understand more about cancer treatments, such as how well they work for specific cancers and what side effects they cause. Some treatments are found to help people live longer with their cancer, slow the growth of their cancer, or improve the quality of their life.

Other benefits of clinical trials may include:  

  • Paving the way for future patients to get better care and more treatment options in the future 

  • Increasing the number of treatment options available to you  

  • Potentially getting a safer or more effective treatment than standard treatment options  

  • Close monitoring by your clinical team during the trial 

  • Some clinical trials pay for part or all the cost of treatment 

There are also risks associated with participating in clinical trials, including: 

  • The treatment may not work for you  

  • You may need to have more frequent doctor's visits  

  • The treatment could have unknown side effects  

  • Your insurance may not cover all the costs of the clinical trial (Ask both your insurance and the trial coordinator about this)  

  • If you are in a randomized trial, part of your treatment may include a placebo, or inactive medicine  

Making an informed decision: Is a clinical trial right for me? 

This can be a difficult decision. Each person is unique and must decide this for themselves. Consider why you would want to take part in a clinical trial.   

  • Do you have all the information you need to make an informed decision?   

  • How does this option compare or affect other treatment decisions?   

  • What does my doctor think about my participation in the trial? Does it align with their recommendations?  

  • What are the benefits and risks?  

How to find available clinical trials

There are many ways to research possible clinical trials. The best place to start is to talk with your cancer doctor about potential trials available to you. Research of clinical trials can also be done online. One important website is www.clinicaltrials.gov.

When searching, be as specific as possible to help you get the best results that apply to you. Within each study description there is a clinical trial study protocol which will describe the following:  

  • Why the trial is being done  

  • Specific information about what kind of treatment is being studied, such as medicine 

  • Information about any similar trials on the same topic that were already done in the past 

  • The phase of the trial   

  • Who qualifies and the number of participants 

  • The timing of the trial  

It is important to remember that clinical trials can have complex qualification criteria. If you think you have found a clinical trial that you qualify for and that interests you, reach out to the contact number listed. The study representative can help further explain the study and give you a detailed list of requirements. 

It is a good idea to also speak with your cancer doctor who is not associated with the trial for another opinion and compare treatment options.  

Questions to ask your doctor about clinical trials 

  • Is now a good time for me to consider a clinical trial?  

  • Are there any clinical trials you would suggest for me?  

  • What do you think about me taking part in this specific clinical trial?  

  • Will participation in a clinical trial delay or have a poor effect on my treatment plan?  

  • Do you think this is the best clinical trial option for me?  

  • Is there anything else about taking part in a clinical trial that you think I should know about?