Recruiting patients, especially those with rare conditions, for clinical trials is really, really hard, and that difficulty can delay research. There are a number of reasons why patients are reluctant to participate, some valid, some myth-based, which need to be overcome. And then there’s the basic challenge that most patients don’t even know the research is happening.
The researchers themselves are subject to a whole bunch of restrictions on how they can go about recruitment, and pharma has its own restrictions. Advertising is almost worthless. A scattershot approach (on tv/streaming, mainstream media, internet) is not effective for rare disorders (too low odds of the ad reaching an affected patient), but there’s no good/legal way to target the advertising, because the whole problem is that the advertiser doesn’t know how to identify the patients, or else they could just contact the patient more directly.
That’s where patients themselves can take action to benefit the whole community — we can help to ensure that clinical trials (and other research) get filled as quickly as possible. Thanks to various online resources (patient groups and social media), we tend to know other patients and we may even know which patients in our circle are potentially eligible for a clinical trial.
So this month, I’m asking you to think about whether you know any adults with XLH (must be XLH, not autosomal or TIO unfortunately), who are NOT on burosumab/Crysvita. And if you do, please pass this link along to them: https://clinicaltrials.gov/study/NCT06525636?cond=xlh&aggFilters=status:not%20rec&rank=5
Just a quick, “Hey, did you know there’s some research happening that needs volunteers for a possible new treatment for XLH? Here’s the link with the basic information and the locations, in case you’re interested.” That’s all you need to do. If you want, you could also send them a link to this post (or the newsletter if you get it by email). You don’t need to advise them, and really, they should do their own due diligence anyway, because they know their risk/benefit factors better than anyone else could.
If you want to know more, this research is basically looking into a burosumab-like antibody that will work essentially the same way, but last longer, so patients will need fewer injections. There are research sites in California, Connecticut, Indiana, Tennessee, Paris, Germany, and Spain, although not all are recruiting yet. Travel/lodging expenses will be covered (but sadly, no, you can’t opt to participate in Paris if you live in the U.S. — wouldn’t that be great though if we could?). When I was in a clinical trial, I was assigned a travel agent who specializes in medical research travel to make all the arrangements, based on my preferences, and it made things so simple. It’s fairly standard practice to use a travel agent, so I assume they’ll do the same thing for this trial. It’s about a one-year commitment, with an option to continue for a second year. I don’t know how many clinic visits will be needed, but that’s an important question to ask the contact person, along with any other risks that may be involved.
Participating in a clinical trial has a number of benefits, both to the patient/caregiver community as a whole and to the individual patients. Obviously, the community benefits if a clinical trial is successful, and so do the individual participants who get treatment earlier than they would otherwise. But participation is also, in my experience, beneficial to the participants, in other valuable ways. For one thing, it’s a chance to interact with health care professionals who truly understand XLH, an experience that many — possibly most — XLHers have never had and are unlikely to have outside of a clinical trial. It varies from researcher to researcher, but in my experience, they’re often willing and even happy to answer patients’ questions about XLH, beyond the scope of the trial itself, time permitting. It’s also really validating to work with, say, a phlebotomist who, when you apologize for not being able to straighten your arm fully to make the blood draw easier, just shrugs and says, “Yeah, I know,” because she’s seen other patients like us!
So, please, if you know any adults with XLH who are not on burosumab at present (or in the last several months — they need to be off it for seven months before the screening appointment), let them know that if they’re thinking about treatment, there’s a clinical trial that might be of interest to them. And feel free to refer them to me if they have questions you can’t answer, but they’re not quite ready to reach out to the research contact person.
How about it? Ready to take action in 2025? LFG!
And if you do send the link to someone, I’d love to know. Not the private details, just that you did indeed take the action, so I can brag about how many of you (no names) are stepping up for the community!
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Please note that the author is a well-read patient, not a doctor, and is not offering medical or legal advice.
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