A lot of the questions posted on the blog are repeated, and, in fact, similar questions are often posed on the Foundation’s Facebook page and through emails and phone calls to our Information and Referral Department.  To ensure that everyone benefits from the responses, I’ll be answering some of these questions via video Q&A blog posts, like this one.

Q: My 7-year-old grandson has cone-rod dystrophy, and it’s difficult to find much information on research about it. Will the research for RP, Usher syndrome and other diseases also benefit him?

Q: I am so excited to hear about all of the discoveries with stem cell research. My son, who is 32 years old, has RP. I am greatly encouraged by this news. How soon will there be clinical trials? Will there be some trials, in close proximity to Tulsa, OK?

Q: My husband has Stargardt disease. We have two small children. What kind of tests do they perform on the children of a blind parent? How much do they cost, and are they covered under health-care plans?

Q: I’m wondering if anyone with RP has had cataract surgery, and how it turned out. I’m scared to do it, but was told it is something I really need to do.

Check out this video for my answers to these four questions, and be sure to check back periodically for similar Q&A posts. And, by all means, if you have a question that relates to one of my posts or my answers in this video post, feel free to post it as a comment and we’ll get you the most informed answer we can.

How do I enroll in, and then keep apprised of, clinical trials?
To stay abreast of clinical trials, visit this Foundation web page, where you’ll need to be registered, if you aren’t already. But that’s quick and painless, and it gets you on the list to receive informative publications and news alerts on retinal research.

Our site is easy to navigate and up-to-date, providing links to informative articles on the emerging treatments entering human studies. Each listing on the site has a link to www.clinicaltrials.gov, the federal government’s website, where you can navigate for additional information. In addition, on clinicaltrials.gov, you can search on your retinal degeneration and get a list of clinical trials. And a new website, NIH Clinical Research Trials and You, explains clinical trials in detail, offering, among other things, basic information, explanations from researchers and first-hand experiences from trial participants.

I’m on the Foundation’s National Registry, but nobody ever calls me. So how does it work?
Researchers often come to us when they’re looking for clinical trial participants. That’s where the Registry comes in. If the treatment is for Stargardt, for example, we’ll search the Registry and let those patients know about recruitment.  We provide you with the researcher’s contact information, and you reach out. This maintains your confidentiality.

If you’re not listed on the Registry, you can go to this page to access an application and FAQ. With all the new clinical trials coming to fruition, the sooner you get registered, the better.

What are the criteria for clinical trials? How do I know if I can participate?
Criteria for participation in a clinical trial vary from trial to trial. With an early safety study, for example, researchers may only want people with advanced disease, those with less vision to lose, in case the treatment has an adverse effect on vision. In some trials that are further along, and known to be safe, young children are treated because of a disease’s early onset.

If you have complications such as glaucoma or cataracts, diabetes, heart disease or cancer, that can exclude you. The only way to know is to check the trial listing first and then talk with the appropriate person at a clinical trial site. Participation in one clinical trial might exclude you from a future study, but that depends on the actual clinical trial, so it is not a hard-and-fast rule.

What’s expected once you’ve made yourself available for a clinical trial?
While everyone with a retinal degeneration wants a treatment or cure as soon as possible, that isn’t the best reason to go into a clinical trial. Clinical trials are human experiments. The main reason to participate is to help advance the science. Even if a treatment doesn’t make it through the trial (i.e.,  the intervention does not have the expected positive result), researchers learn about the treatment and/or the potential of possible alternatives. And while researchers collaborate with the FDA to ensure safety, there are still risks. You should be aware of this before signing on.

Clinical trials often require several or many visits, sometimes over years, to a research center for follow-up. Some visits may last several hours, as you undergo several tests, some of which can be grueling. This can be especially tough if you don’t live near the center. And investigators want you to finish what you started. It’s critical that you comply with the treatment and schedule until the trial’s done. If people drop out or don’t comply, the trial will likely fail.

What could happen to me during or after a clinical trial?
Before you participate in a clinical trial, you will have to read what has become a long consent form thoroughly and weigh your personal along with scientific considerations. Really, the bottom line is: Are you willing to help the field move forward, not only for yourself but for others while understanding that there could be some risk involved?

Here are a couple other things to consider: If a trial is for a drug, you might be given a placebo instead of the active medication; and even if the treatment requires an implant or surgical procedure, you might receive a sham implant or incision. The ability to determine if a potential treatment has an effect requires this comparison.