By Danielle Buchanan
New clinical trials, especially ASO therapies, are a welcome arrival to the Huntington Disease (HD) community. As a clinical research coordinator, one of my responsibilities is to answer patient and family inquiries about clinical trial participation. I have noticed major misconceptions regarding these upcoming trials. Many consider participation in a trial as a long-term treatment, but of course we truly don’t know the final story, as it can be a long time before a compound has clinical relevance.
As of now, most of the inquiries to our center concern GENERATION-HD (Genentech-Roche) or PRECISION-HD (Wave Life Sciences). The former includes a two-year commitment with a monthly intrathecal injection; the latter is a Phase I study. Participation in either of these trials could exclude you from another ASO clinical trial. Despite these restrictions, I have encountered a frenetic urgency for participation. Most patients equate participation in these studies as an opportunity “to be cured.” I have even encountered some patients wanting to drop out of a study, to get into another.
Months before GENERATIONS-HD was announced, I received calls from patients inquiring about this study, asking to “save their spot.” How, as a community, can we help our patients and families navigate this stressful time of feeling like they may be left out? How do we, as HD coordinators, manage expectations? These are difficult interactions, and I believe we need to discuss as an HD community how to navigate these challenging situations.
When I receive calls from patients, I make sure that each patient has established care in an HD clinic. I still receive calls about studies that have closed enrollment. If coming from another HD center, our policy is to make sure we know the patients clinically before we engage them regarding a research study. We want to meet the patients, understand their HD story, ensure they are getting the proper care, and establish a therapeutic relationship. We set out, at the beginning, to remind patients that we have a limited number of slots for clinical trials, and make extra efforts to explain that clinical trials are not treatments. I think, as a coordinator, you must stress that there is never a guarantee for participation in a clinical trial, but the most important action is making sure the patient is getting good clinical care.
Several months ago, I met a man with HD during an ENROLL-HD appointment. This appointment was made soon after he received the news that he was gene positive. The patient and his wife were devastated, but amazingly had never heard of clinical trials before. I told them about SIGNAL and how it was testing the hypothesis that infusions of this therapy may slow the progression of HD. They were thrilled and ecstatic to think they could possibly be a candidate. When I called to confirm his screening appointment, they had read more about clinical trials in HD, and decided to wait to consider participation in a potential upcoming ASO trial. The man may or may not be a candidate for a future trial, but the potential for receiving a gene-silencing treatment grabbed his attention. Despite the small amount of promising data in humans, it was enough for him to choose to wait to see if he could participate in an ASO study. His story echoes other stories of participants who believe participation in a certain trial will deliver a better outcome for their HD.
I believe we are still early on the therapeutic timeline of HD. We can’t predict the future of gene-silencing studies, as there is no sure thing in clinical research. This is the beginning of an era and a new strategy for clinical trials in HD, but remembering the difference between a trial and a therapy is an important reminder as we speak to our patients. We need to counsel our patients not to jump to conclusions about the potential outcomes of these studies before they are completed. I also think we need to make sure patients understand every aspect of what they are signing up for in the consent. Retention is really important for giving us the best chance to accurately interpret clinical trial data. While a patient may be eager to jump on board a study, it may be challenging for them to commit to two years, and participation in a trial should not be threatened if another trial seems more interesting.
Managing the expectations of our patients is one of the main battles I experience as a coordinator. Until we understand what these treatments can actually do for patients, we must encourage participation to help figure out future treatments—and this is just the beginning. Better treatments are the light at the end of the tunnel. HD is finally getting the societal attention it needs, which means more of an opportunity for coordinators, and the HD team, to take the lead in these discussions.
Danielle Buchanan is a clinical research coordinator at Vanderbilt University Medical Center.