Are Pre-symptomatic Huntington’s Disease Patients Ready for Clinical Trials?

As I’ve shared in previous columns, several Huntington’s disease (HD) advocates, including myself, were granted a patient listening session with the U.S. Food and Drug Administration (FDA), focused on people who are gene-positive for HD but have not yet developed symptoms.

In preparation for the session, which took place on Monday, we conducted an online community survey to better understand how this population feels about clinical trials. Specifically, we wanted to know what benefits people would look for, and what risks they’d be willing to take.

We obtained some great insight from over 200 participants and were excited to share their thoughts with the FDA. This is just the beginning of many important conversations that need to take place between patients, advocates, pharmaceutical companies, clinicians, and government regulators.

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I’ll share more about the survey results in future columns, but today I want to focus on a few key data points.

As I wrote last month, many pre-symptomatic HD patients struggle to stay up to date on the status and availability of all clinical trials. According to ClinicalTrials.gov, about 50 HD-related studies are currently active or recruiting. This is great news, but it can be hard for anyone, even clinicians, to constantly be aware of all possible opportunities to participate.

ClinicalTrials.gov is an important website, but you really have to understand what you’re looking for to determine if you might qualify for a study. Then you have to reach out to a trial site, hope researchers respond, and go through a long list of inclusion and exclusion criteria. The time commitment can be overwhelming.

One of our survey questions was, “I would be willing to accept ___% risk of mortality to receive a highly effective gene therapy that slows disease progression by 90%.” We asked people to consider this question at three different disease stages: pre-symptomatic, experiencing early or subtle symptoms, and experiencing moderate symptoms.

The answers revealed that, on average, patients would accept a 30% risk of mortality when pre-symptomatic, a 36% risk when showing early or subtle symptoms, and a 42% risk when displaying moderate symptoms. I believe these data demonstrate the large risks people are willing to take to delay the onset of HD.

More interesting survey results were that 65% of respondents shared that they do not see a healthcare professional for HD, and 60% are “open to” or “highly motivated” to participate in a clinical trial.

It seems there’s a problem that requires a solution: A number of clinical trials are recruiting patients who are pre-symptomatic or early in their disease progression, and many of these patients are willing to participate in trials and take risks. But how will researchers meet their aggressive recruitment timeframes when much of their target population doesn’t regularly see a doctor for HD and is unable to keep track of clinical trial opportunities?

I’m not sure there’s a perfect answer for the whole community, but it’s an issue I continue to think about.

If you are interested in our survey data, please reach out; I’m happy to share our learnings. We plan to submit the data to this year’s annual Huntington Study Group meeting, which will take place in Tampa, Florida, in November. I also plan to write more columns about the survey results and the FDA patient listening session.

Thanks for reading!

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.