Medically reviewed by Dr. Amanda F. Marsch, dermatologist at UC San Diego Health.
What is a clinical trial? Can I participate in a clinical trial? And if so, how do I find one? If you’re curious about the answers to these questions, then we’ve got you covered.
In order to help you navigate the decision of whether a clinical trial is right for you, we’re going to explain what a clinical trial is, how they work, the pros and cons of participating, how to find one near you, and potential questions to ask before participating.
Let’s dive in.
What is a clinical trial and how do they work?
Clinical trials are carefully designed research studies that evaluate a medical intervention or treatment. Most clinical trials are created to determine if a new, experimental treatment for a disease is safe and effective, and if relevant, how it fares in comparison to existing treatments.
In the United States, clinical trials are categorized into five phases. Here’s why each phase exists and how it works.
Phase 0 clinical trials aim to understand how a drug acts within the body, and how it is processed in the body, by giving the drug to a small group of healthy individuals (10-15 people).
Phase I clinical trials aim to determine the maximally effective dosage of the drug with the fewest side effects. This phase occurs in a group of 15-30 individuals. If the experimental treatment is deemed safe, it can then progress to be tested in a Phase II clinical trial.
Phase II clinical trials test the experimental treatment in a group of 25 to 100 people with a disease to assess for effectiveness, as well as to continue to monitor for safety. Once again, if the drug proves to be effective in treatment of the disease as well as safe for use, it can be used in a Phase III clinical trial.
Phase III clinical trials compare the safety and effectiveness of the experimental drug to existing treatments for the disease. Most Phase III clinical trials include several hundred patients and are often randomized, meaning that each participant is randomly assigned to receive the experimental treatment, or assigned to the control group, in which they will receive one of the existing treatments for the disease or a placebo drug. If the physicians leading the trial find the drug to be both safe and effective in a Phase III trial, they will submit the results of the trial to the Food and Drug Administration (FDA) for approval of the experimental drug as a treatment option for the disease.
The FDA is a U.S. government agency tasked with protecting public health through supervision over the safety of food, medications, and biological products. If the FDA finds the results of this trial to be positive, it will approve the experimental treatment as an option for treatment of the disease in the U.S.
Following FDA approval, Phase IV clinical trials deliver the treatment to thousands of people and monitor the long-term safety and effectiveness of the drug.
Wondering which type of trial you might be looking for? If you have vitiligo or another condition and participate in a clinical trial, you will most likely participate in a Phase II or Phase III trial.
Participating in a clinical trial
Now that you know how each phase of a clinical trial works, let’s dive into the details. What is it like to participate in a clinical trial? Most Phase II or Phase III clinical trials in the U.S. follow a similar set of steps. Here’s how it works.
First, trial staff provide information about the study and answer any questions from potential participants. Next, participants sign a consent form, a legal document indicating that he or she understands all of the research-related information, and is willing to participate in the study.
The participant will then attend a “screening visit” during which they will undergo baseline evaluations – for example, a physical exam – to determine eligibility for inclusion in the study. If the participant meets eligibility criteria, he or she will move forward to being the trial. In a Phase II trial, this means the participant will be given the experimental treatment. In a Phase III trial, the participant will be randomly assigned to receive the experimental treatment, one of the existing treatments for the disease or a placebo drug.
Once assigned to a trial, participants will attend regular study visits to the research site – a clinic or hospital – where they will be seen by the doctor leading the clinical trial. At each visit, participants will be evaluated to assess the safety and effectiveness of the treatment. Participants can discontinue participation, or withdraw, from a clinical trial at any time.
Benefits of participating in a vitiligo clinical trial
There are many benefits of participating in a clinical trial. First and foremost, you become a partner in scientific discovery by helping researchers learn more about the condition at hand and bringing new treatments to the masses.
For those who have not had success with existing vitiligo treatments, clinical trials offer a new treatment option with the potential to provide more success in repigmentation. Finally, participating in a clinical trial can make people feel that they are taking a more active role in their health care by seeking out all options – even new ones.
However, clinical trials are not ideal for everyone. Here’s why.
Considerations when participating in a vitiligo clinical trial
There are some aspects to consider before opting into a clinical trial. First, participants could experience worsening of disease symptoms or unwanted side effects from either the experimental treatment, existing treatment or even the placebo. Also, depending on the number of required research visits and research site location, participation can be time-consuming and costly.
Clinical trials can also require participants to limit their use of other medications.
How to find a vitiligo clinical trial
Whether you’re interested in learning more or signing up for a trial, there are a number of ways to identify trials that are coming up or currently underway. To find ongoing trials near you, visit clinicaltrials.gov. You can also ask your primary care physician about ongoing clinical trials in vitiligo, reach out to local support groups and check vitiligo social media pages to hear from others in the vitiligo community about their experiences with clinical trials.
Questions to ask before signing up for a clinical trial
When researching clinical trials for vitiligo, you’ll have a chance to speak with clinical trial staff and ask questions. This is the time to get answers. Here are some questions you might want to ask:
What is the purpose of this study?
Will I be compensated to participate in this study?
What are the chances I will receive the experimental treatment versus an existing treatment/placebo?
What are possible risks/side effects of this experimental treatment?
Can I take my regular medications while on the trial?
How often will I be required to attend in-person appointments?
Where is the research site located?
Will I be reimbursed for expenses such as travel and parking?
How will this study impact my day-to-day life?
Will I continue receiving the study treatment after the trial ends?
How will I be kept informed about the results of the study?
Have you ever participated in a clinical trial? Are you interested in being a part of a clinical trial? Share your experience below.
Meera is a medical student from San Diego, California who plans to pursue a career as a dermatologist. She hopes to help navigate her patients through challenging and uncertain times through support, education and empathy. In her free time, she enjoys traveling, cooking and spending time with friends and family.