Media reports of two newly approved drugs for treating alopecia areata brought good news to many people living with the disease. After years of being told there were no treatment options, there’s now hope. However, most news stories about new drugs or indications (uses) usually focus on the researchers and clinical trial results. Rarely do they mention the thousands of clinical trial volunteers — like Maureen McGettigan. Maureen, a 61-year-old from the Philadelphia, PA, area, has lived with alopecia areata since she was 16. Without volunteers like her, there could be no approvals by the U.S. Food & Drug Administration (FDA). Given how crucial clinical trial volunteers are, how can you or someone you know who has alopecia areata find a study and be part of the very important clinical trial community? Three stages before drugs are FDA-approved Maureen, who is also a NAAF board member, encourages people to consider entering a clinical trial if the opportunity arises. She said it took quite a while before there was an alopecia areata drug trial in her area, but when a phase 3 trial became available, she jumped at the chance to participate. “I thought, literally, what do I have to lose? I’d had alopecia areata for such a long time, it’s worth a shot,” she said. “If my hair grows back, that’s fabulous. If not, then I gave it a shot.” If there had been a local phase 2 trial, Maureen said she would have volunteered for that one. There are three stages of clinical trials before a drug is approved. When one phase is successful, researchers can move on to the next. However, trials can be stopped at any time in any phase if the researchers find dangers to the volunteers or there’s no apparent benefit to the drug. (Information on the differences between the clinical stages follows below the article. In addition, NAAF has a webinar, Patient Guide to Clinical Trials and Drug Development: Phases and Terminology Explained, which goes into more detail.) Like many clinical trial volunteers, Maureen pointed out that while she hoped she’d benefit from taking the study drug, there was another reason. “The clinical trial is an opportunity to help myself but also to help our community to advance the science,” she explained. She added that having yet one more patient enrolled in the study would help provide more data for the scientists. Benefits of being a clinical trial volunteer In addition to having access to a possibly new medication, clinical trial volunteers have other advantages through their participation, such as having someone keeping a close eye on their health. This includes regular contact with research nurses, along with blood tests and other medical tests, depending on the drug and its potential side effects. Of course, there may be some disadvantages to participating in a clinical trial as well. If it’s a trial that compares a new drug with a placebo (an inactive medication), there is a chance you may not get the study drug at first. But Maryanne Senna, MD, Director of the Lahey Hair Loss Center of Excellence and Research Unit at Beth Israel Lahey Health and member of NAAF’s Scientific & Medical Advisory Task Force, pointed out that some studies have what is called cross-over portions. “This is where patients who were first randomized to placebo eventually get treated with the active therapy. This is worth asking if you are considering participating in a clinical trial.” The advantages of regular contact and tests could become a disadvantage if the clinic visits become too frequent or inconvenient for you, though, so you have to consider how much of your time is needed. Finally, you could experience side effects, or the study drug may not work. In Maureen’s case, although she hasn’t had full hair regrowth since she entered the trial, she said she’s happy anyway. “My lashes and eyebrows have come back!” she explained. “I have more hair than I did two and a half years ago. I’m not wearing my false eyelashes anymore and I can put the makeup on my eyebrows to expand them a little bit.” But why be in a trial if there already are approved treatments? Many in the alopecia areata community welcomed the FDA approval for Olumiant and LITFULO over the past year. Still, research continues into pediatric use of these drugs, and other medications. Expanding the number of available treatments is essential because not all drugs work for or are appropriate for everyone. According to Dr. Senna, response rates, where patients see good results, hover around 40% in most clinical trials when the researchers look at about a year’s worth of data. “This means that many people with alopecia areata still have less than clinically meaningful results. I believe that these first FDA approvals in alopecia areata are the tip of the iceberg,” she said. “As we learn more about alopecia areata and continue to study emerging therapies, we will hopefully increase these response rates and have improved patient outcomes.” In addition, more treatments may become available for children as well. How to find a trial If volunteering for an alopecia areata clinical trial interests you, speak with your dermatologist. There may be a trial that is not local but close enough that you’re willing to travel. NAAF has a downloadable worksheet to help you discuss the issue with your doctor and a guide that explains how to use the U.S. government’s clinical trials website. NAAF also posts information about active alopecia areata clinical trials on its website. Researchers vet and prepare their clinical trial volunteers carefully because they want the trial to be as fair and accurate as possible – and safe for the volunteers. Before trial admission, the volunteers are carefully screened to ensure they are likely to complete the trial (to minimize drop-outs) and don’t have any health issues that could affect how the study drug works. Part of the screening looks for inclusion and exclusion criteria. Inclusion criteria may include things like: Having the condition the drug is meant for Being in a particular demographic or geographic area Being in a specific age group Exclusion criteria can include things like: Not being able to give informed consent Undergoing treatment for an unrelated disease Not being in the trial’s target age group “Participating in a trial doesn’t hurt and you’re helping the next generation,” Maureen said. There’s the hope that when other 16-year-old girls lose their hair there will be options, and they don’t have to go through all the challenges and the pain and the suffering that that many of us did.” Dr. Senna agreed. “Some current treatments are less effective for people who have had alopecia areata for a long time. That is understandably disappointing. But it doesn’t always have to be this way,” she says. “Now is our moment! We are at the tip of the iceberg in understanding this condition and the best treatments for it. As physicians, patients, and advocates, we must keep this wonderful momentum. To continue to move our knowledge forward – and that includes developing treatments for patients who have had the disease for a longer time – clinical trials in alopecia areata are critical.” Clinical Trial Phases Explained: Phase 1 is like a fact-finding mission. A small group of up to 100 clinical trial volunteers take the new drug for a few months so researchers can look at the drug’s safety and dose levels. Most often, the volunteers are healthy, although in some cases, they may have the disease or condition the drug is meant to treat. About 70% of these studies move on to phase 2 trials. Phase 2 trials need volunteers with the disease or condition. The researchers now observe up to several hundred people, how they react to the drug, and whether they have side effects. This phase can last from several months to a couple of years. Only about one-third of phase 2 trials move on to phase 3. Phase 3 trials take the longest and need the most volunteers, up to 3000 and sometimes more. This is when researchers are looking more closely at the safety data. Because there are more volunteers and the study goes for an extended period, the researchers may find more uncommon side effects or some that only occur after someone takes the drug for longer. About one-quarter of drugs move on to apply for FDA approval after a phase 3 trial.
