For years, people with vitiligo didn’t have many treatment options, but that has changed, thanks to burgeoning research being studied in clinical trials. Current research studies are investigating treatments such as Janus kinase (JAK) inhibitors, a solution of healthy skin cells to repigment skin, and monoclonal antibodies that can help bring melanocytes back to the body. These studies all provide hope for new vitiligo treatments.
To understand more about how these research studies work and what you should know before joining one, MyVitiligoTeam sat down with Dr. Amit Pandya, president of the Global Vitiligo Foundation. We talked to Dr. Pandya during a recent live Q&A event on MyVitiligoTeam, where he and several panelists shared details about participating in research studies.
Dr. Pandya has conducted several clinical trials during the course of his career and shared some of the top facts that people with vitiligo should know about participating in them.
Clinical trials are research studies that test new ways of treating a condition like vitiligo, managing symptoms, or improving quality of life with a condition. During a clinical trial, researchers try experimental treatment options on people who have a particular condition and monitor the effects to determine whether the treatment is effective and safe. Clinical trials may involve lifestyle interventions, such as a specific diet, or a new medication, procedure, or device.
Clinical trials for vitiligo are led by a principal investigator (typically a medical doctor). You can find out about them from your dermatologist, from a local research institution, or on sites like ClinicalTrials.gov. The National Institutes of Health and other federal agencies have developed an array of guidelines and policies to ensure the clinical studies are safe, ethical, and of high quality.
Clinical trials represent the top way that new treatments for skin conditions are developed, Dr. Pandya said. “The reason we don’t see patients with severe acne scars anymore is because a miracle drug that went through clinical trials back in the ’70s and ’80s called isotretinoin is now routinely being used by dermatologists. The biologic [medicines] for psoriasis have changed people’s lives and cleared their psoriasis because of studies started 20 years ago. My hope is that by this time next year, we will have the first medication for vitiligo. … So many agents in dermatology have come out as a result of clinical trials.”
If you are interested in participating in a clinical trial, you’ll want to be armed with information before you actually choose one and get started. That’s why it’s important to know what you should ask the study directors when you first contact them.
“First, you should ask the purpose of the study,” Dr. Pandya said. “Find out if the study is being conducted to determine what concentration or dose of the medicine works and if it’s going to improve [your condition] significantly. Is it going to be just for patients with localized vitiligo? Is it going to be for patients with generalized vitiligo? And then ask why researchers think this approach will be effective, because there should be some scientific basis behind using this.”
Additional questions you’ll want to ask include:
You’ll also want to know how many people have tested this treatment. “It makes a difference if only 50 people have been treated with a drug versus 3,000 people,” Dr. Pandya said. “If a particular treatment has been used in a lot of patients for another condition, you’ll have a clearer vision of how it’s benefited those patients, as well as what their side effects were.”
If you’re considering joining a trial, you’ll also want to ask about the chances that you’ll be on the actual drug versus a placebo, which is an inactive compound. You’ll also want to understand if you’ll eventually be able to get on the actual drug and when that will be.
“In order to do evidence-based scientific studies, we give some of the patients the actual medicine, and some of the patients get a pill that has nothing in it,” Dr. Pandya said. “And the reason we do that is because sometimes just taking a pill with nothing in it can actually be associated with improvement in your condition, and that’s called the placebo effect.”
The study investigator should be able to tell you the percentage of people who will be on the active drug versus the placebo. “For example, perhaps 80 percent of the patients will be on the medicine, but 20 percent will be on the placebo — so you’ve got a four out of five chance of being on the medicine,” Dr. Pandya said. “It’s possible that you’ll be more comfortable participating in a study in which you have a higher chance of being on the actual medicine versus a placebo.”
You should also ask the investigators how long people will stay on the placebo. “Ask if there’s a possibility that people given the placebo will be able to transition to the actual drug later in the study — and this is important,” Dr. Pandya said. “This happens a lot in vitiligo studies — for instance, someone may be on the placebo for six months, and then after that, everyone transitions to the actual medicine.”
Because some vitiligo medications must be applied topically to the skin, you should also ask the study investigators if you’ll need to change any lifestyle habits to take part in the study. For example, you might want to ask if you can wear makeup with the treatment, and if so, whether you’ll need to wait a certain amount of time between applying the topical medication and putting on makeup.
Watch as Rosa explains why she participated in a clinical trial for vitiligo.
You’ll also want to ask about the cost of participating in a clinical trial. While most trials themselves are paid for by the sponsoring organization, there can sometimes be additional costs involved. Dr. Pandya recommends asking the following questions:
Knowing the answers to these questions can help make your decision process easier.
Living with vitiligo can be challenging. The good news is that you don’t have to go it alone. MyVitiligoTeam is the social network for people with vitiligo and their loved ones. Here, members from around the world come together to ask questions, offer support and advice, and meet others who understand life with vitiligo.
Have you participated in a clinical trial? Are you interested in joining one? Share your thoughts in the comments below or by posting on MyVitiligoTeam.
In partnership with the Global Vitiligo Foundation, which strives to improve the quality of life for individuals with vitiligo through education, research, clinical care, and community support.