Helping Huntington’s disease clinical trials recruit participants
The oft-confusing process can go faster and attract more candidates
Note: This column was updated Sept. 1, 2023, to correct that phase 1 clinical trials do involve human participants.
I’ve always felt that the most impactful way for me to accelerate the development of new treatments for Huntington’s disease is to quicken recruitment for clinical trials. When such a trial opens for participants, every day that goes by without a full complement of recruits is a day wasted when we could be closer to knowing whether the medicine works.
Clinical trials typically have two phases that involve people with Huntington’s. Phase 2 trials typically strive to figure out if the drug is safe in humans and what dosage is most effective.
If the Phase 2 trial goes well and the pharmaceutical company decides to go further, a Phase 3 trial is scheduled. That larger study, which also involves humans, typically wants to discern the treatment’s effectiveness in context with its risk of side effects; if all seems well, approval is typically sought from the U.S. Food and Drug Administration (FDA) or other countries’ regulatory bodies. This month, for instance, the FDA approved an existing drug, Ingrezza (valbenazine), to treat chorea in adults with Huntington’s disease.
Many in the Huntington’s community might remember when the pharmaceutical giant Roche had a Phase 3 study of tominersen that opened after recruiting over 790 participants from 18 countries. Unfortunately, that trial was halted in March 2021 because the committee that analyzed the data concluded the drug wasn’t effective.
Several clinical trials are recruiting in the U.S. to study treatments for Huntington’s, according to ClinicalTrials.gov. All have been recruiting for many months, and I’ve been hearing from many sources that the trials are struggling to find potential participants. I don’t exactly know what’s meant by “struggling,” but I keep asking this question: Why isn’t there a long list of people waiting for a phone call to join a study? There has to be a better way!
Hearing that studies are struggling to recruit pains me because I know willing participants are out there. When I launched HD Genetics last year, our first priority was to provide a best-in-class genetic testing experience, but at our core, we wanted to bridge the gap so people could learn about opportunities to participate in clinical trials.
Improving the recruitment process
Having had the opportunity to analyze all the HD Genetics data and combining that with insights from conversations with many people affected by Huntington’s, I see a few areas for potential improvement.
For one, we can’t assume that laypeople have any understanding about the basics of clinical trials; many people aren’t aware of what they’d be signing up for. We need more creative and general education in the Huntington’s community about these basics. Many potential recruits are new to the disease and have little to no knowledge of its past, present, or future.
We also must be clearer and more direct about how people can sign up for trials. I attend most educational conferences on Huntington’s, along with educated and engaged families. Many times, the pharmaceutical companies that are holding trials attend, but people stopping by their booths typically get only a basic flyer and are told to contact the clinical trial site. I believe most people get lost at this stage. Why not have them sign up for the trials right on the spot? I’ve seen some clinical trial locations participate in conferences, which makes sign-up easier. We need more of that.
Further, we need clearer education about what’s available in trials. I’m not trying to downplay what’s available to that end, but I think there’s room for improvement. A lot of trial information gets confusing, especially in trying to understand which trials are still recruiting, where they’re located, and what’s the first step to participate.
Lastly, some people need their hands held in these matters. That’s OK! Even some of my longtime friends who are Huntington’s advocates don’t know where to start. Participation in a clinical trial can be a confusing, emotional decision and a major commitment. Some of the most gratifying work I can do is to help provide a nonbiased list of current opportunities, allowing those interested to ask questions and ultimately choose for themselves. Based on that decision, I can then make a warm introduction to a clinical trial location so they can start the screening process.
Not every candidate will be accepted for a clinical or observational trial, but I know many more people would offer themselves for selection if they knew more. I hope to make that happen to accelerate the development of treatments.
Do you want to learn more about clinical or observational research opportunities, but have no idea where to start? Please reach out to me in the comments and I’ll be happy to talk you through some options.
Note: Huntington’s Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Huntington’s Disease News or its parent company, BioNews, and are intended to spark discussion about issues pertaining to Huntington’s disease.