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.
About the Author
Frieda wachsmann
I have three children all positive for HD, and all between 30 and 40 years old.
I live in Australia, and I couldn’t agree with you more the recruitment process is lacking.
As you say there should be a queue of people wanting to be admitted into trials.
I believe HD positive people want to take part in trials, but don’t know how.
As you can imagine life, is busy atm, but I would be happy to take this conversation further.
With best wishes, Frieda Wachsmann
B.J. Viau
Thanks for the comment, Freida. Sorry to hear about your children's impact with HD. Please reach out whenever you're able if you want to discuss more. -BJ
Jolanda
Ik heb zelf de ziekte van huntington...ik ben een vrouw 57 jaar oud en woon in Nederland.....IK smeek om mee te doen aan bv hersen injecties van unicure ik mail ze zonder resultaat. Of roche tomiseren kwam ik niet in aanmerking....ik doe al 13 jaar mee aan het wetenschappelijk onderzoek in het lUMC...maar daar kun je bellen war je wilt maar ze blijven vaag...dus geen hoop voor mij helaas....ik ben zelf verpleegkundige
Dave Dalenberg
I am my wife's caregiver and we have been to Univ. S. Florida, Univ. of Alabama and Vanderbilt Univ HDSA clinics with the goal of enrolling her in a clinical trial. While she was qualified for expanded access use at Vanderbilt for Pridopidine earlier this year, we have learned that expanded access is in reality no access as only three of the trial sites (Duke, OSU and Mass. Gen'l) are open for this use and so far, none have agreed to allow her to participate in expanded use. Ohio State University sent us an email saying that they are being limited to opening use by the onerous, inflexible, costly and time delayed process of approval by FDA. PCT 518 is another drug that, while in trial in EU, the FDA has suspended trial in the US and no expanded access is allowed by the PCT Therapeutics, presumably until the suspension is lifted. There are no adverse consequences, despite years of trials for either of these two drugs.
As you know, time is working against my wife, as with all patients with HD, and she is discouraged and losing all hope.
Is there anything you can do to help us?
Dave Dalenberg
B.J. Viau
Hi Dave - thanks for the comment bringing to life the sad but true reality of current trials for people currently impacted by HD. Why the HD community hasn't advocated for PTC 518 trial access is something I think about a lot. It's essentially the best shot for someone currently impacted by HD and we have very limited awareness of "why" the FDA decided not to allow the trial in the USA. If I am an HD advocacy organization, i'd be looking in the mirror wondering how they could advocate for trial access vs waiting for a phase 3 study to become open in 2-3 years. We're are losing the war against huntington's disease and I don't feel like the HD army is taking the strategic approach to win the war. I haven't seen much strategic change since I've been in this fight for 20 years. It's time for some change and starts with all of the families impacted by HD.
I did reach out to some folks at Prilenia to figure out if there is any other options for your wife. I'll reach back out when I hear something. Feel free to email me [email protected] . Thanks, Dave.
Edie Morris
I never know what to do regarding signing up for participating in data collected for trials.
My dad was diagnosed with HD at age 70 in 2006 (42 CAG repeats). I think he may have started developing symptoms when he was around age 55. He died in 2009 (age 73). We didn’t know it was in his family since his mom (my grandmother) died at age 36 when he was 3. Their family was “disconnected” from him and his siblings for the most part. The only thing we really knew was that there a number of cousins who had committed suicide when they were young (ages unknown). There was also a tendency for chronic depression in that part of the family.
I suspect my great grandfather developed HD later in life after my grandmother died. I also suspect my great grandfather’s brother may have had either HD or another form of dementia since froze to death after wandering away from home and falling into a creek. He had lived with my great grandmother and great grandfather for a number of years.
I am now 67 years old. I’ve declined to be genetically tested since I’ve never had children with no risk of passing HD on. I am thinking that my risk of developing HD symptoms is decreasing as I get older. I do suffer from chronic depression which manifested when I was a teen. My question is: would I be helpful in any way(s) for any of the HD studies? I want to do all I can to help find a cure or way of preventing this terrible disease.
B.J. Viau
The only study you can participate in without knowing your genetic status is the ENROLL-HD.org study which is a yearly observational study. If you're willing and able I'd highly encourage you to check it out and consider participating! Thanks for the comment! - BJ
J. C.
I think one of the major hurtles is that many patients, while otherwise totally willing, logistically CANT participate in many trials because it involves frequent, often long-distance travel. My own father had to travel three hours (each way) to and from Nashville to Vanderbilt to participate in the trail that just proved Ingrezza/Valbenazine's efficacy for managing Huntington's Chorea.
Even with my dad still in relatively early stages of the disease AND Vanderbilt paying the travel costs, he still finds it physically and mentally exhausting. I was with him at his last appointment when they mentioned the possibility of extending the trial and I could see him seriously considering dropping out. He VERY much wants to help any way he can, but travel is hard on HD patients, and unless someone just happens to live right next door to a major research hospital with a world-class neurology center, travel is exactly what they're going to have to do.
B.J. Viau
Great thought. I 100% agree. There needs to be more consideration for people's time and effort to attend. Even more so as trials recruit young participants who are still working and have children/pets. Many studies dont include enough $$ to offset the costs to leave home and take off from work. There has to be better options to do some of the visits remotely. Appreciate your comment! - BJ
Vicky Albrecht
My husband is currently involved in a drug study and his doctor is the one who told us about it!
Several months ago, HDSA sent out an email that included a list of trials and studies with all of the contact information. I found that to be very helpful and was able to sign him up for a study through that info. Why doesn't a list like that come out on a regular basis?
B.J. Viau
More of this would be great! I don't see as much proactive outreach about specific trials as I'd like to see from many of the HD advocacy organizations. THANK YOU to you and your husband for participating!!
Jody Digregorio
I was wondering where the nearest clinical trial center would be close to Kingston ontario Canada,
B.J. Viau
There are three Enroll-HD trial sites that look like equal distance from you. Enroll-HD is an observational study, but most locations that have enroll also have clinical trials. Here are the three: 1) Toronto, North York General Hospital 2) Ottawa, Parkinson's and Movement Disorders Clinic, C2 and 3) Montreal, CHUM Centre de Recherche du Centre Hospitalier de l’université de Montréal. The Ottawa Hospital . If you use this link you can get direct contact details for these study sites. Best idea is to email/call them and ask your questions to a study coordinator. https://www.enroll-hd.org/study-sites-and-locations-browse-the-map/#find-a-platform-clinic-browse-map/?view_449_per_page=1000&view_449_page=1 Hope this helps, but reach out if I can provide additional info.
Alvin Hippolyte
My friend was diagnosed in with the disease in 2016. Her condition has deteriorated where she can hardly walk (more like stumbling along) short distances. Her speech is garbled and her head is in constant side to side motion. She would do anything to take part in a clinical trial. She lives in New York and would be more than willing to be guided to a trial. She turns 50 years in November and is not hopeful of living beyond that age. Help!!!
B.J. Viau
Hi Alvin - sorry to hear about your friend. Do you know if she currently receives care for HD? There are a handful of clinical trial sites throughout New York state and also great places to receive care if a trial isn't an option. Feel free to send me an email and I can try to better direct you. [email protected]
Alvin Hippolyte
Thanks for your response. Right now her only care us the prescribed medication which is geared towards preventing depression. I take care of her, taking her to physical therapy etc; A while back she was prescribed a medication to help her chores, but once she started taking it, it triggered the reaction of her crying daily. She stopped taking it. We’re thinking now of trying Ingreeza and see what the response might be. Will email you
Jiří (George)
Hi BJ Viau.
My beloved friend Lence was diagnosed with Huntington's disease 3 years ago. Now Lence is 44 years old and is in the early stages of the disease, where he is starting to have problems walking and speaking. Lenka is registered in the Enroll-HD observational study.
We actively monitor the development of drugs for HD patients. Lenka is very interested in participating in clinical trials conducted by UniQure or PTC Therapeutics for AMT-130 or PTC518. Unfortunately, no such study has been opened in the Czech Republic so far.
We wrote emails abroad, where clinical studies of AMT-130 and PTC518 have opened, but they are not interested in patients from the Czech Republic. Either they did not respond at all to our request to participate in the clinical trial, or they politely wrote to us that they have a sufficient number of their adepts for the experimental treatment of HD and thus do not need to recruit patients for the clinical trial from abroad.
It's strange that they have enough of their patients, but when I look at some clinics they are still recruiting people for the AMT-130 phase 2 treatment group and it's been 9 months and the places are still not filled, so there is something poorly.
Dear BJ Viau, can you please do something for us? Can you please advise me where I should write or whom I should contact with a big request so that my beloved Lenka is accepted into the AMT-130 or PTC518 clinical trial?
Thank you very much for your reply,
Jiří (George) from Brno, Czech Republic (Europe)
B.J. Viau
Thanks for the message, Jiri. Frustrating probably doesn't even describe your feelings. I sent you an email to discuss specifics on possible steps to move forward. Thank you for reading and commenting! -BJ